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Sample records for clinical trial registries

  1. Clinical trial registries: a practical guide for sponsors and researchers of medicinal products

    National Research Council Canada - National Science Library

    Foote, MaryAnn

    2006-01-01

    ... Industry perspective on public clinical trial registries and results databases . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . ...

  2. Clinical trials in dentistry in India: Analysis from trial registry.

    Science.gov (United States)

    Gowri, S; Kannan, Sridharan

    2017-01-01

    Evidence-based practice requires clinical trials to be performed. In India, if any clinical trial has to be performed, it has to be registered with clinical trial registry of India. Studies have shown that the report of clinical trials is poor in dentistry. Hence, the present study has been conducted to assess the type and trends of clinical trials being undertaken in dentistry in India over a span of 6 years. All the clinical trials which were registered with the Central Trial Registry of India (CTRI) (www.ctri.nic.in) from January 1, 2007 to March 3, 2014 were evaluated using the keyword "dental." Following information were collected for each of the clinical trials obtained from the search; number of centres (single center/multicentric), type of the institution undertaking the research (government/private/combined), study (observational/interventional), study design (randomized/single blinded/double-blinded), type of health condition, type of participants (healthy/patients), sponsors (academia/commercial), phase of clinical trial (Phase 1/2/3/4), publication details (published/not published), whether it was a postgraduate thesis or not and prospective or retrospective registration of clinical trials, methodological quality (method of randomization, allocation concealment). Descriptive statistics was used for analysis of various categories. Trend analysis was done to assess the changes over a period of time. The search yielded a total of 84 trials of which majority of them were single centered. Considering the study design more than half of the registered clinical trials were double-blinded (47/84 [56%]). With regard to the place of conducting a trial, most of the trials were planned to be performed in private hospitals (56/84 [66.7%]). Most (79/84, 94.1%) of the clinical trials were interventional while only 5/84 (5.9%) were observational. Majority (65/84, 77.4%) of the registered clinical trials were recruiting patients while the rest were being done in healthy

  3. Clinical trial registries: a practical guide for sponsors and researchers of medicinal products

    National Research Council Canada - National Science Library

    Foote, MaryAnn

    2006-01-01

    ... . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . ix MaryAnn Foote Clinical trial registries and publication of results - a primer . . . . . . . . . . . . . . . . . . 1 Ana Marušić and Charlotte Haug The journal...

  4. Clinical Trials in Dentistry: A Cross-sectional Analysis of World Health Organization-International Clinical Trial Registry Platform.

    Science.gov (United States)

    Sivaramakrishnan, Gowri; Sridharan, Kannan

    2016-06-01

    Clinical trials are the back bone for evidence-based practice (EBP) and recently EBP has been considered the best source of treatment strategies available. Clinical trial registries serve as databases of clinical trials. As regards to dentistry in specific data on the number of clinical trials and their quality is lacking. Hence, the present study was envisaged. Clinical trials registered in WHO-ICTRP (http://apps.who.int/trialsearch/AdvSearch.aspx) in dental specialties were considered. The details assessed from the collected trials include: Type of sponsors; Health condition; Recruitment status; Study design; randomization, method of randomization and allocation concealment; Single or multi-centric; Retrospective or prospective registration; and Publication status in case of completed studies. A total of 197 trials were identified. Maximum trials were from United States (n = 30) and United Kingdom (n = 38). Seventy six trials were registered in Clinical Trials.gov, 54 from International Standards of Reporting Clinical Trials, 13 each from Australia and New Zealand Trial Register and Iranian Registry of Clinical Trials, 10 from German Clinical Trial Registry, eight each from Brazilian Clinical Trial Registry and Nederland's Trial Register, seven from Japan Clinical Trial Registry, six from Clinical Trial Registry of India and two from Hong Kong Clinical Trial Registry. A total of 78.7% studies were investigator-initiated and 64% were completed while 3% were terminated. Nearly four-fifths of the registered trials (81.7%) were interventional studies of which randomized were the large majority (94.4%) with 63.2% being open label, 20.4% using single blinding technique and 16.4% were doubled blinded. The number, methodology and the characteristics of clinical trials in dentistry have been noted to be poor especially in terms of being conducted multi-centrically, employing blinding and the method for randomization and allocation concealment. More emphasis has to be

  5. Comparing the Effectiveness of a Clinical Registry and a Clinical Data Warehouse for Supporting Clinical Trial Recruitment: A Case Study

    Science.gov (United States)

    Weng, Chunhua; Bigger, J Thomas; Busacca, Linda; Wilcox, Adam; Getaneh, Asqual

    2010-01-01

    This paper reports a case study comparing the relative efficiency of using a Diabetes Registry or a Clinical Data Warehouse to recruit participants for a diabetes clinical trial, TECOS. The Clinical Data Warehouse generated higher positive predictive accuracy (31% vs. 6.6%) and higher participant recruitment than the Registry (30 vs. 14 participants) in a shorter time period (59 vs. 74 working days). We identify important factors that increase clinical trial recruitment efficiency and lower cost. PMID:21347102

  6. Optimal use of MRI in clinical trials, clinical care and clinical registries of patients with rheumatoid arthritis

    DEFF Research Database (Denmark)

    Østergaard, Mikkel; Møller-Bisgaard, Signe

    2014-01-01

    the benefits of including MRI in treat-to-target strategies. The benefits of incorporating MRI into clinical registries are not yet known, but may include improved knowledge about the real-life advantages of MRI, as well as opportunities to develop better clinical and laboratory composite measures to monitor......Magnetic resonance imaging (MRI) clearly is more sensitive than clinical examination and conventional radiography (x-ray) for detection of inflammation (synovitis, bone marrow oedema (osteitis) and tenosynovitis) and damage (bone erosion and cartilage loss/joint space narrowing) in patients...... with rheumatoid arthritis (RA). The question is when and how MRI should be used. The present article reviews our knowledge about, and provides suggestions for, the use of MRI in clinical trials, clinical care and clinical registries. In clinical trials, the OMERACT RA MRI scoring system (RAMRIS) is a thoroughly...

  7. Differences in Investigator-Initiated Trials between Japan and Other Countries: Analyses of Clinical Trials Sponsored by Academia and Government in the ClinicalTrials.gov Registry and in the Three Japanese Registries.

    Directory of Open Access Journals (Sweden)

    Tatsuya Ito

    Full Text Available Following the amendment of the Pharmaceutical Affairs Law in Japan in 2003 researchers were permitted to begin investigator-initiated trials (IITs. In subsequent years, however, the number of IITs remained low. In other countries in Asia as well as in Europe, North America, and South Africa, the number of IITs has increased over the past decade. The differences in the characteristics of IITs between Japan and other countries are unknown. Some studies have analyzed the characteristics of all clinical trials according to registry databases, but there has been less research focusing on IITs.The purpose of this study is to analyze the characteristics of IITs in the ClinicalTrials.gov registry and in the three Japanese registries, to identify differences in IITs between Japan and other countries.Using Thomson Reuters Pharma™, trials sponsored by academia and government as IITs in 2010 and registered in ClinicalTrials.gov were identified. IITs from 2004 to 2012 in Japan were identified in the three Japanese registries: the University Hospital Medical Information Network Clinical Trials Registry, the Japan Pharmaceutical Information Center Clinical Trials Information, and the Japan Medical Association Center for Clinical Trials, Clinical Trials Registry. Characterization was made of the trial purposes, phases, participants, masking, arms, design, controls, and other data.New and revised IITs registered in ClinicalTrials.gov during 2010 averaged about 40% of all sponsor-identified trials. IITs were nearly all early-phase studies with small numbers of participants. A total of 56 Japanese IITs were found over a period of 8 years, and these were also almost nearly all early-phase studies with small numbers of participants.There appear to be no great differences between Japan and other countries in terms of characteristics of IITs. These results should prompt a new review of the IIT environment in Japan.

  8. Clinical Case Registries (CCR)

    Data.gov (United States)

    Department of Veterans Affairs — The Clinical Case Registries (CCR) replaced the former Immunology Case Registry and the Hepatitis C Case Registry with local and national databases. The CCR:HIV and...

  9. Do clinical trials conducted in India match its healthcare needs? An audit of the Clinical Trials Registry of India

    Directory of Open Access Journals (Sweden)

    Mansi Chaturvedi

    2017-01-01

    Full Text Available Background: India continues to contribute disproportionately to the global burden of disease and public health research output from India is also known to be not commensurate with her healthcare needs. We carried out the present study to assess if clinical trials were in line with the health care needs of the country by auditing the clinical trials registry of India. Materials and Methods: All the clinical studies registered in CTRI between July 20, 2007 and December 31, 2015 were searched in the “Trial Search” section. The total number of studies, their phases of development, and therapeutic areas were assessed. Trials in each therapeutic area was compared with the disease burden (DALYs in that area taken from Global Health Estimates [2014] Summary Tables of the WHO. The number of trials conducted per state in India was also compared with the population of that state [Census 2011]. Results: A total of 6474 studies were registered of which 3325 (51.4% were clinical trials. The state of Maharashtra had the highest number trials [16.4%] followed by Karnataka ( 11.6% and Tamil Nadu (10%. Populous states like Uttar Pradesh (5.3% and Bihar (1.4% had far fewer trials. The largest number of trials was in the area of cancer (16.4%, followed by diabetes (12.1% and cardiovascular diseases (10.1%. Infectious and parasitic diseases had the highest DALYs (82,681 and ranked first in disease burden but accounted for only 5% of the total trials and ranked 7th according to number of trials. Cancer ranked first in the number of trials (16.4%, but ranked 6th based on DALYs. Conclusion: Clinical trials conducted in India are not in consonance with her health care needs. Strengthening the capacity for conducting trials in the populous states and the north-eastern part of the country is necessary to allow a more equitable selection of participants. The government should introduce policies to encourage new drug development in areas where needed the most.

  10. Systematic Reviews Published in Emergency Medicine Journals Do Not Routinely Search Clinical Trials Registries: A Cross-Sectional Analysis.

    Science.gov (United States)

    Keil, Lukas G; Platts-Mills, Timothy F; Jones, Christopher W

    2015-10-01

    Publication bias compromises the validity of systematic reviews. This problem can be addressed in part through searching clinical trials registries to identify unpublished studies. This study aims to determine how often systematic reviews published in emergency medicine journals include clinical trials registry searches. We identified all systematic reviews published in the 6 highest-impact emergency medicine journals between January 1 and December 31, 2013. Systematic reviews that assessed the effects of an intervention were further examined to determine whether the authors described searching a clinical trials registry and whether this search identified relevant unpublished studies. Of 191 articles identified through PubMed search, 80 were confirmed to be systematic reviews. Our sample consisted of 41 systematic reviews that assessed a specific intervention. Eight of these 41 (20%) searched a clinical trials registry. For 4 of these 8 reviews, the registry search identified at least 1 relevant unpublished study. Systematic reviews published in emergency medicine journals do not routinely include searches of clinical trials registries. By helping authors identify unpublished trial data, the addition of registry searches may improve the validity of systematic reviews. Copyright © 2014 American College of Emergency Physicians. Published by Elsevier Inc. All rights reserved.

  11. Clinical trials in allied medical fields: A cross-sectional analysis of World Health Organization International Clinical Trial Registry Platform

    Directory of Open Access Journals (Sweden)

    S. Kannan

    2016-03-01

    Conclusion: The number of clinical trials done in allied fields of medicine other than the allopathic system has lowered down, and furthermore focus is required regarding the methodological quality of these trials and more support from various organizations.

  12. Pediatric Clinical Trials Conducted in South Korea from 2006 to 2015: An Analysis of the South Korean Clinical Research Information Service, US ClinicalTrials.gov and European Clinical Trials Registries.

    Science.gov (United States)

    Choi, Sheung-Nyoung; Lee, Ji-Hyun; Song, In-Kyung; Kim, Eun-Hee; Kim, Jin-Tae; Kim, Hee-Soo

    2017-12-01

    The status of pediatric clinical trials performed in South Korea in the last decade, including clinical trials of drugs with unapproved indications for children, has not been previously examined. The aim was to provide information regarding the current state of pediatric clinical trials and create a basis for future trials performed in South Korea by reviewing three databases of clinical trials registrations. We searched for pediatric clinical studies (participants South Korea between 2006 and 2015 registered on the Clinical Research Information Service (CRIS), ClinicalTrials.gov, and the European Clinical Trials Registry (EuCTR). Additionally, we reviewed whether unapproved indications were involved in each trial by comparing the trials with a list of authorized trials provided by the Ministry of Food and Drug Safety (MFDS). The primary and secondary outcomes were to determine the change in number of pediatric clinical trials with unapproved indications over time and to assess the status of unauthorized pediatric clinical trials from the MFDS and the publication of articles after these clinical trials, respectively. We identified 342 clinical studies registered in the CRIS (n = 81), ClinicalTrials.gov (n = 225), and EuCTR (n = 36), of which 306 were reviewed after excluding duplicate registrations. Among them, 181 studies were interventional trials dealing with drugs and biological agents, of which 129 (71.3%) involved unapproved drugs. Of these 129 trials, 107 (82.9%) were authorized by the MFDS. Pediatric clinical trials in South Korea aiming to establish the safety and efficacy of drugs in children are increasing; however, non-MFDS-authorized studies remain an issue.

  13. Overview of registered studies in orthodontics: Evaluation of the ClinicalTrials.gov registry.

    Science.gov (United States)

    Allareddy, Veerasathpurush; Rampa, Sankeerth; Masoud, Mohamed I; Lee, Min Kyeong; Nalliah, Romesh; Allareddy, Veerajalandhar

    2014-11-01

    The Food and Drug Administration Modernization Act of 1997 made it mandatory for all phase II through IV trials regulated by this Act to be registered. After this, the National Institutes of Health created ClinicalTrials.gov, which is a registry of publicly and privately supported clinical studies of human participants. The objective of this study was to examine the characteristics of registered studies in orthodontics. The ClinicalTrials.gov Web site was used to query all registered orthodontic studies. The search term used was "orthodontics." No limitations were placed for the time period. All registered studies regardless of their recruitment status, study results, and study type were selected for analysis. A total of 64 orthodontic studies were registered as of January 1, 2014. Of these, 52 were interventional, and 12 were observational. Close to 60% of the interventional studies and 66.7% of the observational studies had sample sizes of 50 or fewer subjects. About 21.2% of the interventional studies and 16.7% of the observational studies had sample sizes greater than 100. Only 1 study was funded by the National Institutes of Health, and the rest were funded by "other" or "industry" sources. Close to 87.7% of the interventional studies were randomized. Interventional model assignments included factorial assignment (3.9%), parallel assignments (74.5%), crossover assignment (7.8%), and single-group assignment (13.7%). Most studies were treatment oriented (80.4%). The types of masking used by the interventional studies included open label (28.9%), single blind (44.2%), and double blind (26.9%). Outcome assessors were blinded in only 6 studies. Orthodontic studies registered in ClinicalTrials.gov are dominated by small single-center studies. There are wide variations with regard to treatment allocation approaches and randomization methods in the studies. These results also indicate the need for multicenter clinical studies in orthodontics. Copyright © 2014

  14. The evaluation of complex clinical trial protocols: resources available to research ethics committees and the use of clinical trial registries--a case study.

    Science.gov (United States)

    Homedes, Núria; Ugalde, Antonio

    2015-06-01

    To assess the potential role of clinical trial (CT) registries and other resources available to research ethics committees (RECs) in the evaluation of complex CT protocols in low-income and middle-income countries. Using a case study approach, the authors examined the decision-making process of a REC in Argentina and its efforts to use available resources to decide on a complex protocol. We also analysed the information in the USA and other CT registries and consulted 24 CT experts in seven countries. Information requested by the Argentinean REC from other national RECs and ethics' experts was not useful to verify the adequacy of the REC's decision whether or not to approve the CT. The responses from the national regulatory agency and the sponsor were not helpful either. The identification of international resources that could assist was beyond the REC's capability. The information in the USA and other CT registries is limited, and at times misleading; and its accuracy is not verified by register keepers. RECs have limited access to experts and institutions that could assist them in their deliberations. Sponsors do not always answer RECs' request for information to properly conduct the ethical and methodological assessment of CT protocols. The usefulness of the CT registries is curtailed by the lack of appropriate codes and by data errors. Information about reasons for rejection, withdrawal or suspension of the trial should be included in the registries. Establishing formal channels of communication among national and foreign RECs and with independent international reference centres could strengthen the ethical review of CT protocols. Published by the BMJ Publishing Group Limited. For permission to use (where not already granted under a licence) please go to http://group.bmj.com/group/rights-licensing/permissions.

  15. A quality analysis of clinical anaesthesia study protocols from the Chinese clinical trials registry according to the SPIRIT statement.

    Science.gov (United States)

    Yang, Lei; Chen, Shouming; Yang, Di; Li, Jiajin; Wu, Taixiang; Zuo, Yunxia

    2018-05-15

    To learn about the overall quality of clinical anaesthesia study protocols from the Chinese Clinical Trials Registry and to discuss the way to improve study protocol quality. We defined completeness of each sub-item in SPIRIT as N/A (not applicable) or with a score of 0, 1, or 2. For each protocol, we calculated the proportion of adequately reported items (score = 2 and N/A) and unreported items (score = 0). Protocol quality was determined according to the proportion of reported items, with values >50% indicating high quality. Protocol quality was determined according to the proportion of reported items. For each sub-item in SPIRIT, we calculated the adequately reported rate (percentage of all protocols with score 2 and NA on one sub-item) as well as the unreported rate (percentage of all protocols with score 0 on one sub-item). Total 126 study protocols were available for assessment. Among these, 88.1% were assessed as being of low quality. By comparison, the percentage of low-quality protocols was 88.9% after the publication of the SPIRIT statement. Among the 51 SPIRIT sub-items, 18 sub-items had an unreported rate above 90% while 16 had a higher adequately reported rate than an unreported rate. The overall quality of clinical anaesthesia study protocols registered in the ChiCTR was poor. A mandatory protocol upload and self-check based on the SPIRIT statement during the trial registration process may improve protocol quality in the future.

  16. Clinical trials for vaccine development in registry of Korea Food and Drug Administration.

    Science.gov (United States)

    Kang, Seog-Youn

    2013-01-01

    Based on the action plan "Ensuring a stable supply of National Immunization Program vaccines and sovereignty of biopharmaceutical products," Korea Food and Drug Administration (KFDA) has made efforts to develop vaccines in the context of self reliance and to protect public health. Along with the recognized infrastructures for clinical trials, clinical trials for vaccines have also gradually been conducted at multinational sites as well as at local sites. KFDA will support to expand six to eleven kinds of vaccines by 2017. In accordance with integrated regulatory system, KFDA has promoted clinical trials, established national lot release procedure, and strengthened good manufacturing practices inspection and post marketing surveillance. Against this backdrop, KFDA will support the vaccine development and promote excellent public health protection.

  17. Clinical disease registries in acute myocardial infarction.

    Science.gov (United States)

    Ashrafi, Reza; Hussain, Hussain; Brisk, Robert; Boardman, Leanne; Weston, Clive

    2014-06-26

    Disease registries, containing systematic records of cases, have for nearly 100 years been valuable in exploring and understanding various aspects of cardiology. This is particularly true for myocardial infarction, where such registries have provided both epidemiological and clinical information that was not readily available from randomised controlled trials in highly-selected populations. Registries, whether mandated or voluntary, prospective or retrospective in their analysis, have at their core a common study population and common data definitions. In this review we highlight how registries have diversified to offer information on epidemiology, risk modelling, quality assurance/improvement and original research-through data mining, transnational comparisons and the facilitation of enrolment in, and follow-up during registry-based randomised clinical trials.

  18. How does a clinical trial fit into the real world? The RELAX-AHF study population into the EAHFE registry.

    Science.gov (United States)

    Miró, Òscar; Gil, Víctor; Müller, Christian; Mebazaa, Alexander; Bueno, Héctor; Martín-Sánchez, Francisco Javier; Herrero, Pablo; Jacob, Javier; Llorens, Pere

    2015-10-01

    To test how accurate the recently published RELAX-AHF trial was in recruiting real-world patients with acute-decompensated heart failure (ADHF). We compared clinical and outcome data of patients receiving serelaxin in the RELAX-AHF trial (RELAX group, n = 581) with patients included in the EAHFE registry [5497 ADHF from 29 Spanish emergency departments (EDs)]. The EAHFE registry was split into two groups: EAHFE-non-RELAX (patients not fulfilling the RELAX-AHF inclusion criteria; n = 3205, 58.3 %) and EAHFE-RELAX A (patients fulfilling RELAX-AHF inclusion criteria; n = 2292, 41.7 %). The latter group was further refined by also applying exclusion criteria (EAHFE-RELAX B; n = 964, 17.4 %). Both EAHFE-RELAX groups differed from the EAHFE-non-RELAX group in multiple aspects, with the lower the proportion of patients with implantable cardiac defibrillator and with pulmonary diseases the greater the differences found. The RELAX group, compared with the EAHFE-RELAX groups, significantly included fewer females, younger patients, less in NYHA class I/II, less with implantable cardiac defibrillator and on beta-blocker treatment, and patients had lower systolic blood pressure and cardiac and respiratory rates at ED arrival. The EAHFE-RELAX groups had a significantly lower all-cause mortality than EAHFE-non-RELAX group, and qualitative analysis suggested that EAHFE-RELAX groups had a higher mortality than the RELAX group. Patients included in the RELAX-AHF trial showed unanticipated differences when compared with a population from the EAHFE registry fulfilling very similar inclusion and exclusion criteria.

  19. Instantaneous Wave-Free Ratio versus Fractional Flow Reserve guided intervention (iFR-SWEDEHEART): Rationale and design of a multicenter, prospective, registry-based randomized clinical trial.

    Science.gov (United States)

    Götberg, Matthias; Christiansen, Evald H; Gudmundsdottir, Ingibjörg; Sandhall, Lennart; Omerovic, Elmir; James, Stefan K; Erlinge, David; Fröbert, Ole

    2015-11-01

    Instantaneous wave-free ratio (iFR) is a new hemodynamic resting index for assessment of coronary artery stenosis severity. iFR uses high frequency sampling to calculate a gradient across a coronary lesion during a period of diastole. The index has been tested against fractional flow reserve (FFR) and found to have an overall classification agreement of 80% to 85%. Whether the level of disagreement is clinically relevant is unknown. Clinical outcome data on iFR are scarce. This study is a registry-based randomized clinical trial, which is a novel strategy using health quality registries as on-line platforms for randomization, case record forms, and follow-up. iFR-SWEDEHEART is a multicenter, prospective, randomized, controlled, clinical open-label clinical trial. Two thousand patients with stable angina or acute coronary syndrome and an indication for physiology-guided assessment of one or more coronary stenoses will be randomized 1:1 to either iFR- or FFR-guided intervention. The randomization will be conducted online in the Swedish web-based system for enhancement and development of evidence-based care in heart disease evaluated according to recommended therapies (SWEDEHEART) registry. The trial has a non-inferiority design, with a primary combined end point of all-cause death, non-fatal myocardial infarction, and unplanned revascularization at 12 months. End points will be identified through national registries and undergo central blind adjudication to ensure data quality. The iFR-SWEDEHEART trial is an registry-based randomized clinical trial evaluating the safety and efficacy of the diagnostic method iFR compared to FFR. Copyright © 2015 Elsevier Inc. All rights reserved.

  20. Clinical Trials

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    Full Text Available ... Clinical Trials About Clinical Trials Clinical trials are research studies that explore whether a medical strategy, treatment, or ... humans. What Are Clinical Trials? Clinical trials are research studies that explore whether a medical strategy, treatment, or ...

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  3. Clinical Trials

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  4. Can rheumatoid arthritis (RA) registries provide contextual safety data for modern RA clinical trials? The case for mortality and cardiovascular disease.

    Science.gov (United States)

    Michaud, Kaleb; Berglind, Niklas; Franzén, Stefan; Frisell, Thomas; Garwood, Christopher; Greenberg, Jeffrey D; Ho, Meilien; Holmqvist, Marie; Horne, Laura; Inoue, Eisuke; Nyberg, Fredrik; Pappas, Dimitrios A; Reed, George; Symmons, Deborah; Tanaka, Eiichi; Tran, Trung N; Verstappen, Suzanne M M; Wesby-van Swaay, Eveline; Yamanaka, Hisashi; Askling, Johan

    2016-10-01

    We implemented a novel method for providing contextual adverse event rates for a randomised controlled trial (RCT) programme through coordinated analyses of five RA registries, focusing here on cardiovascular disease (CVD) and mortality. Each participating registry (Consortium of Rheumatology Researchers of North America (CORRONA) (USA), Swedish Rheumatology Quality of Care Register (SRR) (Sweden), Norfolk Arthritis Register (NOAR) (UK), CORRONA International (East Europe, Latin America, India) and Institute of Rheumatology, Rheumatoid Arthritis (IORRA) (Japan)) defined a main cohort from January 2000 onwards. To address comparability and potential bias, we harmonised event definitions and defined several subcohorts for sensitivity analyses based on disease activity, treatment, calendar time, duration of follow-up and RCT exclusions. Rates were standardised for age, sex and, in one sensitivity analysis, also HAQ. The combined registry cohorts included 57 251 patients with RA (234 089 person-years)-24.5% men, mean (SD) baseline age 58.2 (13.8) and RA duration 8.2 (11.7) years. Standardised registry mortality rates (per 100 person-years) varied from 0.42 (CORRONA) to 0.80 (NOAR), with 0.60 for RCT patients. Myocardial infarction and major adverse cardiovascular events (MACE) rates ranged from 0.09 and 0.31 (IORRA) to 0.39 and 0.77 (SRR), with RCT rates intermediate (0.18 and 0.42), respectively. Additional subcohort analyses showed small and mostly consistent changes across registries, retaining reasonable consistency in rates across the Western registries. Additional standardisation for HAQ returned higher mortality and MACE registry rates. This coordinated approach to contextualising RA RCT safety data demonstrated reasonable differences and consistency in rates for mortality and CVD across registries, and comparable RCT rates, and may serve as a model method to supplement clinical trial analyses for drug development programmes. Published by the BMJ Publishing

  5. Clinical Trials

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  20. Clinical Trials

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    Full Text Available ... of clinical trials contribute to medical knowledge and practice. Why Clinical Trials Are Important Clinical trials are a key ... Enterprise NHLBI has a strong tradition of supporting clinical trials that have not only shaped medical practice around the world, but have improved the health ...

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    Full Text Available ... Working at the NHLBI Contact and FAQs Accessible Search Form Search the NHLBI, use the drop down list to ... to learn more about clinical research and to search for clinical trials: NHLBI Clinical Trials Browse a ...

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    Full Text Available ... resources to the strategies and treatments that work best. How Clinical Trials Work If you take part in a clinical trial, you may get tests or treatments in a hospital, clinic, or doctor's office. In some ways, taking part in a clinical trial is different ...

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    Full Text Available ... Health Topics / About Clinical Trials About Clinical Trials Clinical trials are research studies that explore whether a medical strategy, treatment, ... tool for advancing medical knowledge and patient care. Clinical research is done only if doctors don't know ...

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    Full Text Available ... about your health or fill out forms about how you feel. Some people will need to travel or stay in hospitals to take part in clinical trials. For example, the National Institutes of Health Clinical Center in Bethesda, Maryland, runs clinical trials. Many other clinical trials take place ...

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    Full Text Available ... child to enroll. Also, children aged 7 and older often must agree (assent) to take part in clinical trials. Clinical trials for children have the same scientific safeguards as clinical trials for adults. For more information, go to "How Do Clinical ...

  9. Clinical Trials

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    Full Text Available ... part in a clinical trial is your decision. Talk with your doctor about all of your treatment options. Together, you can make the ... more about, or taking part in, clinical trials, talk with your doctor. He or she may know about ... clinical trials. NIH Clinical Research Studies ...

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    Full Text Available ... any clinical trial before you agree to take part in the trial. Talk with your doctor about specific trials you're interested in. For a list of questions to ask your doctor and the ...

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    Full Text Available ... medical knowledge and practice. Why Clinical Trials Are Important Clinical trials are a key research tool for ... other for moderate persistent asthma. The results provided important treatment information for doctors and patients. The results ...

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    Full Text Available ... Entire Site NHLBI Entire Site Health Topics News & Resources Intramural Research ... or device is safe and effective for humans. What Are Clinical Trials? Clinical trials are research ...

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    Full Text Available ... sponsor clinical trials. Many other groups, companies, and organizations also sponsor clinical trials. Examples include Government Agencies, ... and Veterans Affairs; private companies; universities; and nonprofit organizations. NIH Institutes and Centers (including the NHLBI) usually ...

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    Full Text Available ... decisionmaking. The purpose of clinical trials is research, so the studies follow strict scientific standards. These standards ... otherwise. The purpose of clinical trials is research, so the studies follow strict scientific standards. These standards ...

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    Full Text Available ... needed. For safety purposes, clinical trials start with small groups of patients to find out whether a ... phase I clinical trials test new treatments in small groups of people for safety and side effects. ...

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    Full Text Available ... organizations also sponsor clinical trials. Examples include Government Agencies, such as the U.S. Departments of Defense and ... to Expect During a clinical trial, doctors, nurses, social workers, and other health care providers might be ...

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    Full Text Available ... risks that outweigh any possible benefits. Clinical Trial Phases Clinical trials of new medicines or medical devices are done in phases. These phases have different purposes and help researchers ...

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    Full Text Available ... whether a new approach causes any harm. In later phases of clinical trials, researchers learn more about ... other National Institutes of Health (NIH) Institutes and Centers sponsor clinical trials. Many other groups, companies, and ...

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    Full Text Available ... are research studies that explore whether a medical strategy, treatment, or device is safe and effective for ... a Clinical Trial If you're interested in learning more about, or taking part in, clinical trials, ...

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    Full Text Available ... best data available for health care decisionmaking. The purpose of clinical trials is research, so the studies ... Thus, research in humans is needed. For safety purposes, clinical trials start with small groups of patients ...

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    Full Text Available ... Events About NHLBI About NHLBI Home Mission and Strategic Vision Leadership Scientific Divisions Operations and Administration Advisory ... a Clinical Trial If you're interested in learning more about, or taking part in, clinical trials, ...

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    Full Text Available ... Some companies and groups sponsor clinical trials that test the safety of products, such as medicines, and how well they work. The U.S. Food and Drug Administration (FDA) oversees these clinical trials. ...

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    Full Text Available ... What to Expect During a clinical trial, doctors, nurses, social workers, and other health care providers might ... enroll in a clinical trial, a doctor or nurse will give you an informed consent form that ...

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    Full Text Available ... and doctors' offices around the country. Benefits and Risks Possible Benefits Taking part in a clinical trial ... volunteer because they want to help others. Possible Risks Clinical trials do have risks and some downsides, ...

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    Full Text Available ... providers don't always cover all patient care costs for clinical trials. If you're thinking about ... clinical trial, find out ahead of time about costs and coverage. You should learn about the risks ...

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    Full Text Available ... and treatments that work best. How Clinical Trials Work If you take part in a clinical trial, ... Learn More Connect With Us Contact Us Directly Policies Privacy Policy Freedom of Information Act (FOIA) Accessibility ...

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    Full Text Available ... and treatments that work best. How Clinical Trials Work If you take part in a clinical trial, ... your doctor about all of your treatment options. Together, you can make the best choice for you. ...

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    Full Text Available ... medical knowledge and practice. Why Clinical Trials Are Important Clinical trials are a key research tool for ... and Usage No FEAR Act Grants and Funding Customer Service/Center for Health Information Email Alerts Jobs ...

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    Full Text Available ... give permission for their child to enroll. Also, children aged 7 and older often must agree (assent) to take part in clinical trials. Find a Clinical Trial If you're interested in learning more about, or taking part in, clinical trials, ...

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    Full Text Available ... more screening tests to see which test produces the best results. Some companies and groups sponsor clinical trials that test the ... and Drug Administration (FDA) oversees these clinical trials. The NIH may partner with these companies or groups to help sponsor some trials. All ...

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    Full Text Available ... you to explore NIH Clinical Center for patient recruitment and clinical trial information. For more information, please email the NIH Clinical Center Office of Patient Recruitment at cc-prpl@cc.nih.gov or call ...

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    Full Text Available ... the same scientific safeguards as clinical trials for adults. For more information, go to "How Do Clinical ... based on what is known to work in adults. To improve clinical care of children, more studies ...

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    Full Text Available ... groups, companies, and organizations also sponsor clinical trials. Examples include Government Agencies, such as the U.S. Departments ... sponsor trials that test principles or strategies. For example, one NHLBI study explored whether the benefits of ...

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    Full Text Available ... Departments of Defense and Veterans Affairs; private companies; universities; and nonprofit organizations. NIH Institutes and Centers (including ... our campus or trials NIH has sponsored at universities, medical centers, and hospitals. ClinicalTrials.gov View a ...

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    Full Text Available ... identified earlier than they would be in general medical practice. This is because late-phase trials have large ... supporting clinical trials that have not only shaped medical practice around the world, but have improved the health ...

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    Full Text Available ... the past, clinical trial participants often were White men. Researchers assumed that trial results were valid for ... different ethnic groups sometimes respond differently than White men to the same medical approach. As a result, ...

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    Full Text Available ... or strategies work best for certain illnesses or groups of people. Some clinical trials show a positive result. For example, the National Heart, Lung, and Blood Institute (NHLBI) sponsored a trial of two different ...

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    Full Text Available ... healthy people to test new approaches to prevention, diagnosis, or screening. In the past, clinical trial participants ... DSMBs for large trials comparing alternative strategies for diagnosis or treatment. In addition, the NIH requires DSMBs ...

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    Full Text Available ... well they work. The U.S. Food and Drug Administration (FDA) oversees these clinical trials. The NIH may partner with these companies or groups to help sponsor some trials. All ...

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    Full Text Available ... trials produce the best data available for health care decisionmaking. The purpose of clinical trials is research, ... they advance medical knowledge and help improve patient care. Sponsorship and Funding The National Heart, Lung, and ...

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    Full Text Available ... including the NHLBI) usually sponsor trials that test principles or strategies. For example, one NHLBI study explored ... risks. Other examples of clinical trials that test principles or strategies include studies that explore whether surgery ...

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    Full Text Available ... benefits of lowering high blood pressure in the elderly outweighed the risks. Other examples of clinical trials ... child to enroll. Also, children aged 7 and older often must agree (assent) to ... as clinical trials for adults. For more information, go to "How Do Clinical ...

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    Full Text Available ... part. Randomization Most clinical trials that have comparison groups use randomization. This involves assigning patients to different comparison groups by chance, rather than choice. This ...

  17. Glocal clinical registries: pacemaker registry design and implementation for global and local integration--methodology and case study.

    Directory of Open Access Journals (Sweden)

    Kátia Regina da Silva

    Full Text Available The ability to apply standard and interoperable solutions for implementing and managing medical registries as well as aggregate, reproduce, and access data sets from legacy formats and platforms to advanced standard formats and operating systems are crucial for both clinical healthcare and biomedical research settings.Our study describes a reproducible, highly scalable, standard framework for a device registry implementation addressing both local data quality components and global linking problems.We developed a device registry framework involving the following steps: (1 Data standards definition and representation of the research workflow, (2 Development of electronic case report forms using REDCap (Research Electronic Data Capture, (3 Data collection according to the clinical research workflow and, (4 Data augmentation by enriching the registry database with local electronic health records, governmental database and linked open data collections, (5 Data quality control and (6 Data dissemination through the registry Web site. Our registry adopted all applicable standardized data elements proposed by American College Cardiology / American Heart Association Clinical Data Standards, as well as variables derived from cardiac devices randomized trials and Clinical Data Interchange Standards Consortium. Local interoperability was performed between REDCap and data derived from Electronic Health Record system. The original data set was also augmented by incorporating the reimbursed values paid by the Brazilian government during a hospitalization for pacemaker implantation. By linking our registry to the open data collection repository Linked Clinical Trials (LinkedCT we found 130 clinical trials which are potentially correlated with our pacemaker registry.This study demonstrates how standard and reproducible solutions can be applied in the implementation of medical registries to constitute a re-usable framework. Such approach has the potential to

  18. Glocal Clinical Registries: Pacemaker Registry Design and Implementation for Global and Local Integration – Methodology and Case Study

    Science.gov (United States)

    da Silva, Kátia Regina; Costa, Roberto; Crevelari, Elizabeth Sartori; Lacerda, Marianna Sobral; de Moraes Albertini, Caio Marcos; Filho, Martino Martinelli; Santana, José Eduardo; Vissoci, João Ricardo Nickenig; Pietrobon, Ricardo; Barros, Jacson V.

    2013-01-01

    Background The ability to apply standard and interoperable solutions for implementing and managing medical registries as well as aggregate, reproduce, and access data sets from legacy formats and platforms to advanced standard formats and operating systems are crucial for both clinical healthcare and biomedical research settings. Purpose Our study describes a reproducible, highly scalable, standard framework for a device registry implementation addressing both local data quality components and global linking problems. Methods and Results We developed a device registry framework involving the following steps: (1) Data standards definition and representation of the research workflow, (2) Development of electronic case report forms using REDCap (Research Electronic Data Capture), (3) Data collection according to the clinical research workflow and, (4) Data augmentation by enriching the registry database with local electronic health records, governmental database and linked open data collections, (5) Data quality control and (6) Data dissemination through the registry Web site. Our registry adopted all applicable standardized data elements proposed by American College Cardiology / American Heart Association Clinical Data Standards, as well as variables derived from cardiac devices randomized trials and Clinical Data Interchange Standards Consortium. Local interoperability was performed between REDCap and data derived from Electronic Health Record system. The original data set was also augmented by incorporating the reimbursed values paid by the Brazilian government during a hospitalization for pacemaker implantation. By linking our registry to the open data collection repository Linked Clinical Trials (LinkedCT) we found 130 clinical trials which are potentially correlated with our pacemaker registry. Conclusion This study demonstrates how standard and reproducible solutions can be applied in the implementation of medical registries to constitute a re-usable framework

  19. Patient registries: useful tools for clinical research in myasthenia gravis.

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    Baggi, Fulvio; Mantegazza, Renato; Antozzi, Carlo; Sanders, Donald

    2012-12-01

    Clinical registries may facilitate research on myasthenia gravis (MG) in several ways: as a source of demographic, clinical, biological, and immunological data on large numbers of patients with this rare disease; as a source of referrals for clinical trials; and by allowing rapid identification of MG patients with specific features. Physician-derived registries have the added advantage of incorporating diagnostic and treatment data that may allow comparison of outcomes from different therapeutic approaches, which can be supplemented with patient self-reported data. We report the demographic analysis of MG patients in two large physician-derived registries, the Duke MG Patient Registry, at the Duke University Medical Center, and the INNCB MG Registry, at the Istituto Neurologico Carlo Besta, as a preliminary study to assess the consistency of the two data sets. These registries share a common structure, with an inner core of common data elements (CDE) that facilitate data analysis. The CDEs are concordant with the MG-specific CDEs developed under the National Institute of Neurological Disorders and Stroke Common Data Elements Project. © 2012 New York Academy of Sciences.

  20. Clinical Trials

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    Full Text Available ... Children and Clinical Studies Program has been successfully developed and evaluated to fill an important gap in ... Possible Benefits Taking part in a clinical trial can have many benefits. For example, you may gain ...

  1. Clinical Trials

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    Full Text Available ... you may get tests or treatments in a hospital, clinic, or doctor's office. In some ways, taking ... people will need to travel or stay in hospitals to take part in clinical trials. For example, ...

  2. Bridging the gap between the randomised clinical trial world and the real world by combination of population-based registry and electronic health record data: A case study in haemato-oncology.

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    Kibbelaar, R E; Oortgiesen, B E; van der Wal-Oost, A M; Boslooper, K; Coebergh, J W; Veeger, N J G M; Joosten, P; Storm, H; van Roon, E N; Hoogendoorn, M

    2017-11-01

    Randomised clinical trials (RCTs) are considered the basis of evidence-based medicine. It is recognised more and more that application of RCT results in daily practice of clinical decision-making is limited because the RCT world does not correspond with the clinical real world. Recent strategies aiming at substitution of RCT databases by improved population-based registries (PBRs) or by improved electronic health record (EHR) systems to provide significant data for clinical science are discussed. A novel approach exemplified by the HemoBase haemato-oncology project is presented. In this approach, a PBR is combined with an advanced EHR, providing high-quality data for observational studies and support of best practice development. This PBR + EHR approach opens a perspective on randomised registry trials. Copyright © 2017 Elsevier Ltd. All rights reserved.

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    Full Text Available ... the clinical trial you take part in, the information gathered can help others and add to scientific knowledge. People who take part in clinical trials are vital to the process of improving medical care. Many people volunteer because ...

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    Full Text Available ... from other clinical trials show what doesn't work or may cause harm. For example, the NHLBI Women's Health Initiative ... safe a treatment is or how well it works. Children (aged 18 and younger) get ... legal consent for their child to take part in a clinical trial. When ...

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    Full Text Available ... Expect During a clinical trial, doctors, nurses, social workers, and other health care providers might be part of your treatment ... phase II clinical trials. The risk of side effects might be even greater for ... treatments. Health insurance and health care providers don't always ...

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    Full Text Available ... a Clinical Trial If you're interested in learning more about, or taking part in, clinical trials, talk with your doctor. He or she may know about studies going on in your area. You can visit the following website to learn more about ...

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    Full Text Available ... products, such as medicines, and how well they work. The U.S. Food and Drug Administration (FDA) oversees these clinical trials. ... cancer also increased. As a result, the U.S. Food and Drug Administration now recommends never using HT ... Clinical Trials Work If you take ...

  9. Evaluating the Efficacy of a Registry linked to a Consent to Re-Contact Program and Communication Strategies for Recruiting and Enrolling Participants into Clinical Trials.

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    Flood-Grady, Elizabeth; Clark, Virginia C; Bauer, Angie; Morelli, Lauren; Horne, Patrick; Krieger, Janice L; Nelson, David R

    2017-12-01

    Although registries can rapidly identify clinical study participants, it is unknown which follow up methods for recruiting are most effective. Our goal is to examine the efficacy of three communication strategies for recruiting and enrolling patients who were identified via a contact registry (i.e., registry linked to a consent to re-contact program). Patients who met the study criteria were identified via the contact registry and targeted for recruitment. In condition 1, patients established in the university hepatology specialty clinics were contacted one time via phone call by the study coordinator and asked to participate (C1). In condition 2, non-established specialty clinic patients were mailed an IRB-approved letter with study information and instructions for calling the study coordinator to participate (C2). Condition 2A included patients who called within two weeks of receiving the letter (C2A); condition 2B included patients who did not call after receiving the letter but were subsequently contacted via phone call. A registry identified 1,060 patients, of which 661were eligible and targeted for recruiting. All 37 patients were reached in C1 and 17 (45.9%) were recruited. Nineteen of the 624 patients in C2A were reached and 10 were recruited whereas 120 of the 605 patients in C2B were reached and 53 (8.7%) were recruited. Seventy patients enrolled with C2B being the most effective (total, cost) recruitment strategy ( n = 50) ( p recruiting.

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    Full Text Available ... trial found that one of the combinations worked much better than the other for moderate persistent asthma. The results provided important treatment information for doctors and patients. The results from other clinical trials show what doesn't work or may cause harm. For example, the NHLBI ...

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    Full Text Available ... patients to find out whether a new approach causes any harm. In later phases of clinical trials, ... device improves patient outcomes; offers no benefit; or causes unexpected harm All of these results are important ...

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    Full Text Available ... and compare new treatments with other available treatments. Steps To Avoid Bias The researchers doing clinical trials take steps to avoid bias. "Bias" means that human choices ...

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    Full Text Available ... gathered can help others and add to scientific knowledge. People who take part in clinical trials are vital to the process of improving medical care. Many people volunteer because they want to help others. ...

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    Full Text Available ... materials, and offer advice on research-related issues. Data Safety Monitoring Board Every National Institutes of Health ( ... III clinical trial is required to have a Data and Safety Monitoring Board (DSMB). This board consists ...

  17. Clinical Trials

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    ... of Personal Stories Peers Celebrating Art Peers Celebrating Music Be Vocal Support Locator DBSA In-Person Support ... by participating in a clinical trial is to science first and to the patient second. More About ...

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    Full Text Available ... to Expect During a clinical trial, doctors, nurses, social workers, and other health care providers might be ... the new approach. You also will have the support of a team of health care providers, who ...

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    Full Text Available ... final stages of a long and careful research process. The process often begins in a laboratory (lab), where scientists ... part in clinical trials are vital to the process of improving medical care. Many people volunteer because ...

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    Full Text Available ... as gene therapy) or vulnerable patients (such as children). A DSMB's role is to review data from a clinical trial for safety problems or differences in results among different groups. The DSMB also reviews research results ...

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    Full Text Available ... or treatment is having harmful effects. Food and Drug Administration In the United States, the Food and Drug Administration (FDA) provides oversight for clinical trials that ...

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    Full Text Available ... Usually, a computer program makes the group assignments. Masking The term "masking" refers to not telling the clinical trial participants which treatment they're getting. Masking, or "blinding," helps avoid bias. For this reason, ...

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    Full Text Available ... organizations also sponsor clinical trials. Examples include Government Agencies, such as the U.S. Departments of Defense and ... how you feel. Some people will need to travel or stay in hospitals to take part in ...

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    Full Text Available ... clinical trials are required to have an IRB. Office for Human Research Protections The U.S. Department of Health and Human Services’ (HHS’) Office for Human Research Protections (OHRP) oversees all research ...

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    Full Text Available ... successfully developed and evaluated to fill an important gap in information and education for parents, clinicians, researchers, ... gathered can help others and add to scientific knowledge. People who take part in clinical trials are ...

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    Full Text Available ... studies. View funding information for clinical trials optimization . Building 31 31 Center Drive Bethesda, MD 20892 Learn ... and Usage No FEAR Act Grants and Funding Building 31 31 Center Drive Bethesda, MD 20892 Learn ...

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    Full Text Available Skip to main content U.S. Department of Health & Human Services Health Topics Health Topics A-Z Clinical Trials Publications and Resources Health Education and Awareness The Science Science Home Blood Disorders and ...

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    Full Text Available ... results. Clinical trials are one of the final stages of a long and careful research process. The ... a patient's age and gender, the type and stage of disease, and whether the patient has had ...

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    Full Text Available ... Clinical Trials Publications and Resources Health Education and Awareness The Science Science Home Blood Disorders and Blood ... of estrogen and progestin, the risk of breast cancer also increased. As a result, the U.S. Food ...

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    Full Text Available ... issues arise. Participation and Eligibility Each clinical trial defines who is eligible to take part in the ... the strategy or treatment is having harmful effects. Food and Drug Administration In the United States, the ...

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    Full Text Available Skip to main content U.S. Department of Health & Human Services Health Topics Health Topics A-Z Clinical Trials Publications and Resources Health Education and Awareness The Science Science Home Blood Disorders ...

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    Full Text Available ... A-Z Clinical Trials Publications and Resources Health Education and Awareness The Science Science Home Blood Disorders ... to fill an important gap in information and education for parents, clinicians, researchers, children, and the general ...

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    Full Text Available ... clinical care of children, more studies are needed focusing on children's health with the goal to develop ... study? How might this trial affect my daily life? Will I have to be in the hospital? ...

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    Full Text Available ... work best for certain illnesses or groups of people. Clinical trials produce the best data available for ... or animals doesn't always work well in people. Thus, research in humans is needed. For safety ...

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    Full Text Available ... sponsor clinical trials. Examples include Government Agencies, such as the U.S. Departments of Defense and Veterans Affairs; ... age and frequency for doing screening tests, such as mammography; and compare two or more screening tests ...

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    Full Text Available ... harm. In later phases of clinical trials, researchers learn more about the new approach's risks and benefits. ... explore whether surgery or other medical treatments produce better results for certain illnesses or groups of people; ...

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    Full Text Available ... offer a variety of funding mechanisms tailored to planning and conducting clinical trials at all phases, including ... Center for Health Information Email Alerts Jobs and Careers Site Index About NHLBI National Institute of Health ...

  20. Clinical Trials

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    Full Text Available ... patients. Usually, a computer program makes the group assignments. Masking The term "masking" refers to not telling ... questions to ask your doctor and the research staff, go to "How Do Clinical Trials Protect Participants?" ...

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    Full Text Available ... study explored whether the benefits of lowering high blood pressure in the elderly outweighed the risks. Other examples of clinical trials that test principles or strategies include studies that explore whether ...

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    Full Text Available ... treatment is having harmful effects. Food and Drug Administration In the United States, the Food and Drug Administration (FDA) provides oversight for clinical trials that are ...

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    Full Text Available ... questions to ask your doctor and the research staff, go to "How Do Clinical Trials Protect Participants?" ... in Bethesda, Maryland. The physicians, nurses, scientists and staff of the NHLBI encourage you to explore NIH ...

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    Full Text Available ... Science Science Home Blood Disorders and Blood Safety Sleep Science and Sleep Disorders Lung Diseases Heart and Vascular Diseases Precision ... women and that are ethnically diverse. Children also need clinical trials that focus on them, as medical ...

  5. Clinical Trials

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    Full Text Available ... and evaluated to fill an important gap in information and education for parents, clinicians, researchers, children, and the general public. What to Expect During a clinical trial, ...

  6. Clinical Trials

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    Full Text Available ... small groups of people for safety and side effects. Phase II clinical trials look at how well ... confirm how well treatments work, further examine side effects, and compare new treatments with other available treatments. ...

  7. Clinical Trials

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    Full Text Available ... Events About NHLBI About NHLBI Home Mission and Strategic Vision Leadership Scientific Divisions Operations and Administration Advisory ... offer a variety of funding mechanisms tailored to planning and conducting clinical trials at all phases, including ...

  8. Clinical Trials

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    Full Text Available ... NHLBI About NHLBI Home Mission and Strategic Vision Leadership Scientific Divisions Operations and Administration Advisory Committees Budget ... always, parents must give legal consent for their child to take part in a clinical trial. When ...

  9. Clinical Trials

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    Full Text Available ... and organizations also sponsor clinical trials. Examples include Government Agencies, such as the U.S. Departments of Defense and Veterans Affairs; private companies; universities; and nonprofit organizations. NIH Institutes and ...

  10. Clinical Trials

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    Full Text Available ... records can quickly show this information if safety issues arise. Participation and Eligibility Each clinical trial defines ... and materials, and offer advice on research-related issues. Data Safety Monitoring Board Every National Institutes of ...

  11. Clinical Trials

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    Full Text Available ... women and that are ethnically diverse. Children also need clinical trials that focus on them, as medical ... often differ for children. For example, children may need lower doses of certain medicines or smaller medical ...

  12. Clinical Trials

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    Full Text Available ... care providers might be part of your treatment team. They will monitor your health closely. You may ... taking part in a clinical trial. Your treatment team also may ask you to do other tasks. ...

  13. Clinical Trials

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    Full Text Available ... new treatments in small groups of people for safety and side effects. Phase II clinical trials look at how well treatments work and further review these treatments for safety. Phase ...

  14. Clinical Trials

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    Full Text Available ... Clinical Trials Publications and Resources Health Education and Awareness The Science Science Home Blood Disorders and Blood ... these results are important because they advance medical knowledge and help improve patient care. Sponsorship and Funding ...

  15. Clinical Trials

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    Full Text Available ... and organizations also sponsor clinical trials. Examples include Government Agencies, such as the U.S. Departments of Defense ... FOIA) Accessibility Copyright and Usage No FEAR Act Grants and Funding Building 31 31 Center Drive Bethesda, ...

  16. Clinical Trials

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    Full Text Available ... as the U.S. Departments of Defense and Veterans Affairs; private companies; universities; and nonprofit organizations. NIH Institutes ... for parents, clinicians, researchers, children, and the general public. What to Expect During a clinical trial, doctors, ...

  17. Clinical Trials

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    Full Text Available ... combination of estrogen and progestin, the risk of breast cancer also increased. As a result, the U.S. Food ... to test new approaches to prevention, diagnosis, or screening. In the past, clinical trial participants often were ...

  18. Clinical Trials

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    Full Text Available ... from other clinical trials show what doesn't work or may cause harm. For example, the NHLBI Women's Health Initiative tested whether hormone therapy (HT) reduced the risk of heart disease in postmenopausal women. ( ...

  19. Clinical Trials

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    Full Text Available ... always, parents must give legal consent for their child to take part in a clinical trial. When ... minimal, both parents must give permission for their child to enroll. Also, children aged 7 and older ...

  20. Clinical Trials

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    Full Text Available ... Wide Range of Audiences The Children and Clinical Studies Program has been successfully developed and evaluated to fill an important gap in information and education for parents, clinicians, researchers, children, and the general public. What to Expect During a clinical trial, doctors, ...

  1. Clinical Trials

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    Full Text Available ... an important gap in information and education for parents, clinicians, researchers, children, and the general public. What to Expect During a clinical trial, doctors, nurses, social workers, and other health care providers might be part of your treatment team. ...

  2. Clinical Trials

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    Full Text Available Skip to main content U.S. Department of Health & Human Services Health Topics Health Topics A-Z Clinical Trials Publications and Resources Health Education and Awareness The Science Science Home Blood Disorders and Blood Safety Sleep ...

  3. Clinical Trials

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    Full Text Available ... treatments produce better results for certain illnesses or groups of people; look at the best age and frequency for doing screening tests, such as mammography; and compare two or more screening tests to see which test ... Some companies and groups sponsor clinical trials that test the safety of ...

  4. Clinical Trials

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    Full Text Available ... patient has had certain treatments or has other health problems. Eligibility criteria ensure that new approaches are tested ... public. What to Expect During a clinical trial, doctors, nurses, social workers, and other health care providers might be part of your treatment ...

  5. Clinical Trials

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    Full Text Available ... This shows how the approach affects a living body and whether it's harmful. However, an approach that works well in the lab or animals doesn't always work well in people. Thus, research in humans is needed. For safety purposes, clinical trials start ...

  6. Clinical Trials

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    Full Text Available ... to main content U.S. Department of Health & Human Services Health Topics Health Topics A-Z Clinical Trials Publications and Resources Health Education and Awareness The Science Science Home Blood Disorders and Blood Safety Sleep Science and ...

  7. Clinical Trials

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    Full Text Available ... protect patients and help produce reliable study results. Clinical trials are one of the final stages of a long and careful research process. The process often begins in a laboratory (lab), where scientists first develop and test new ...

  8. Clinical Trials

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    Full Text Available ... benefits of lowering high blood pressure in the elderly outweighed the risks. Other examples of clinical trials ... child to enroll. Also, children aged 7 and older often must agree (assent) to take part ... about how you feel. Some people will need to travel or stay in hospitals ...

  9. Clinical Trials

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    Full Text Available ... safe a treatment is or how well it works. Children (aged 18 and younger) get special protection as research subjects. Almost always, parents must give legal consent for their child to take part in a clinical trial. When ...

  10. Clinical Trials

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    Full Text Available ... As a result, the U.S. Food and Drug Administration now recommends never using HT to prevent heart disease. When HT is used for menopausal symptoms, it should be taken only at the smallest dose and for the shortest time possible. Clinical trials, like the two described above, ...

  11. Clinical Trials

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    Full Text Available ... approach that works well in the lab or animals doesn't always work well in people. Thus, research in humans is needed. For safety purposes, clinical trials start with small groups of patients to find out whether a ...

  12. Information on blinding in registered records of clinical trials

    Directory of Open Access Journals (Sweden)

    Viergever Roderik F

    2012-11-01

    Full Text Available Abstract Information on blinding is part of the data that should be provided upon registration of a trial at a clinical trials registry. Reporting of blinding is often absent or of low quality in published articles of clinical trials. This study researched the presence and quality of information on blinding in registered records of clinical trials and highlights the important role of data-recording formats at clinical trial registries in ensuring high-quality registration.

  13. Textbook of clinical trials

    National Research Council Canada - National Science Library

    Day, Simon; Machin, David; Green, Sylvan B

    2006-01-01

    ... . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . xix INTRODUCTION . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 1 1 The Development of Clinical Trials Simon...

  14. Clinical Trials

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    Full Text Available ... criteria differ from trial to trial. They include factors such as a patient's age and gender, the ... bias. "Bias" means that human choices or other factors not related to the protocol affect the trial's ...

  15. Clinical Trials

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    Full Text Available ... under way. For example, some trials are stopped early if benefits from a strategy or treatment are ... stop a trial, or part of a trial, early if the strategy or treatment is having harmful ...

  16. Hotlines and clinical trial updates presented at the German Cardiac Society Meeting 2010: FAIR-HF, CIPAMI, LIPSIA-NSTEMI, Handheld-BNP, PEPCAD III, remote ischaemic conditioning, CERTIFY, PreSCD-II, German Myocardial Infarction Registry, DiaRegis.

    Science.gov (United States)

    Pöss, Janine; Jacobshagen, Claudius; Ukena, Christian; Böhm, Michael

    2010-07-01

    This article summarizes the results of a number of clinical trials and registries in the field of cardiovascular medicine which were presented during the Hotline Sessions at the annual meeting of the German Cardiac Society, held in Mannheim, Germany, from 8th to 10th April 2010. The data were presented by leading experts in the field with relevant positions in the trials. It is important to note that unpublished reports should be considered as preliminary data, as the analysis may change in the final publications. The comprehensive summaries have been generated from the oral presentation and should provide the readers with the most comprehensive information on diagnostic and therapeutic development in cardiovascular medicine similar as previously reported (Maier et al. in Clin Res Cardiol 98:345-352, 2009; 98:413-419, 2009).

  17. Analysis of Existing Guidelines for the Systematic Planning Process of Clinical Registries.

    Science.gov (United States)

    Löpprich, Martin; Knaup, Petra

    2016-01-01

    Clinical registries are a powerful method to observe the clinical practice and natural disease history. In contrast to clinical trials, where guidelines and standardized methods exist and are mandatory, only a few initiatives have published methodological guidelines for clinical registries. The objective of this paper was to review these guidelines and systematically assess their completeness, usability and feasibility according to a SWOT analysis. The results show that each guideline has its own strengths and weaknesses. While one supports the systematic planning process, the other discusses clinical registries in great detail. However, the feasibility was mostly limited and the special requirements of clinical registries, their flexible, expandable and adaptable technological structure was not addressed consistently.

  18. Clinical Trials

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    Full Text Available ... people who fit the patient traits for that study (the eligibility criteria). Eligibility criteria differ from trial to trial. They include factors such as a patient's age and gender, the type and stage of disease, and whether ...

  19. Clinical Trials

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    Full Text Available ... trials are research studies that explore whether a medical strategy, treatment, or device is safe and effective ... trials are research studies that explore whether a medical strategy, treatment, or device is safe and effective ...

  20. Clinical Trials

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    Full Text Available ... trials show what doesn't work or may cause harm. For example, the NHLBI Women's Health Initiative tested whether hormone therapy (HT) reduced the risk of heart disease in postmenopausal women. (When the trial began, HT ...

  1. Clinical Trials

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    Full Text Available ... for trials with cutting-edge approaches, such as gene therapy or new biological treatments. Health insurance and ... trials that involve high-risk procedures (such as gene therapy) or vulnerable patients (such as children). A ...

  2. Clinical Trials

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    Full Text Available ... sponsored a trial of two different combinations of asthma treatments. The trial found that one of the ... much better than the other for moderate persistent asthma. The results provided important treatment information for doctors ...

  3. Clinical Trials

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    Full Text Available ... Sponsors also may stop a trial, or part of a trial, early if the strategy or treatment is having harmful effects. Food and Drug Administration In the United States, the Food and Drug Administration (FDA) provides oversight ...

  4. Constructing a Local Potential Participant Registry to Improve Alzheimer's Disease Clinical Research Recruitment.

    Science.gov (United States)

    Grill, Joshua D; Hoang, Dan; Gillen, Daniel L; Cox, Chelsea G; Gombosev, Adrijana; Klein, Kirsten; O'Leary, Steve; Witbracht, Megan; Pierce, Aimee

    2018-01-01

    Potential participant registries are tools to address the challenge of slow recruitment to clinical research. In particular, registries may aid recruitment to secondary prevention clinical trials for Alzheimer's disease (AD), which enroll cognitively normal older individuals meeting specific genetic or biomarker criteria. Evidence of registry effectiveness is sparse, as is guidance on optimal designs or methods of conduct. We report our experiences of developing a novel local potential participant registry that implemented online enrollment and data collection. In the first year of operation, 957 individuals submitted email addresses to the registry, of whom 592 self-reported demographic, family history, and medical data. In addition, registrants provided information related to their interest and willingness to be contacted about studies. Local earned media and community education were the most effective methods of recruitment into the registry. Seventy-six (26%) of 298 registrants contacted about studies in the first year enrolled in those studies. One hundred twenty-nine registrants were invited to enroll in a preclinical AD trial, of whom 25 (18%) screened and 6 were randomized. These results indicate that registries can aid recruitment and provide needed guidance for investigators initiating new local registries.

  5. Clinical Trials

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    Full Text Available ... the NHLBI's Children and Clinical Studies Web page. Children and Clinical Studies Learn more about Children and Clinical Studies Importance of Children in Clinical Studies Children have often had to ...

  6. Clinical Trials

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    Full Text Available ... Studies Learn more about Children and Clinical Studies Importance of Children in Clinical Studies Children have often ... participants. Children and Clinical Studies Learn about the importance of children in clinical studies and get answers ...

  7. Clinical Trials

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    Full Text Available ... for trials with cutting-edge approaches, such as gene therapy or new biological treatments. Health insurance and health ... trials that involve high-risk procedures (such as gene therapy) or vulnerable patients (such as children). A DSMB's ...

  8. Clinical Trials

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    Full Text Available ... include factors such as a patient's age and gender, the type and stage of disease, and whether ... How long will the trial last? Who will pay for the tests and treatments I receive? Will ...

  9. Clinical Trials

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    Full Text Available ... medicines, and how well they work. The U.S. Food and Drug Administration (FDA) oversees these ... trials are a key research tool for advancing medical knowledge and patient care. ...

  10. Clinical Trials

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    Full Text Available ... Masking, or "blinding," helps avoid bias. For this reason, researchers also may not be told which treatments ... from a study at any time, for any reason. Also, during the trial, you have the right ...

  11. Clinical Trials

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    Full Text Available ... get special protection as research subjects. Almost always, parents must give legal consent for their child to ... trial's potential risks are greater than minimal, both parents must give permission for their child to enroll. ...

  12. Clinical Trials

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    Full Text Available ... risk of heart disease in the first few years, and HT also increased the risk of stroke ... a safety measure. They ensure a trial excludes any people for whom the protocol has known risks ...

  13. Clinical Trials

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    Full Text Available ... Initiative tested whether hormone therapy (HT) reduced the risk of heart disease in postmenopausal women. (When the trial began, HT was already in common use for the treatment of menopausal symptoms. It also ...

  14. Clinical Trials

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    Full Text Available ... risk of heart disease in the first few years, and HT also increased the risk of stroke ... master plan called a protocol (PRO-to-kol). This plan explains how the trial will work. The ...

  15. Clinical Trials

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    Full Text Available ... treatment is having harmful effects. Food and Drug Administration In the United States, the Food and Drug ... life? Will I have to be in the hospital? How long will the trial last? Who will ...

  16. Clinical Trials

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    Full Text Available ... procedures painful? What are the possible risks, side effects, and benefits of taking part in the study? How might this trial affect my daily life? Will I have to be in the hospital? ...

  17. Clinical Trials

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    Full Text Available ... Blood Safety Sleep Science and Sleep Disorders Lung Diseases Heart and Vascular Diseases Precision Medicine Activities Obesity, Nutrition, ... whether hormone therapy (HT) reduced the risk of heart disease in postmenopausal women. (When the trial began, HT ...

  18. Clinical Trials

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    Full Text Available ... are ethical and that the participants' rights are protected. The IRB reviews the trial's protocol before the ... may know about studies going on in your area. You can visit the following website to learn ...

  19. Clinical Trials

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    Full Text Available ... a laboratory (lab), where scientists first develop and test new ideas. If an approach seems promising, the ... Centers (including the NHLBI) usually sponsor trials that test principles or strategies. For example, one NHLBI study ...

  20. Clinical Trials

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    Full Text Available ... whether hormone therapy (HT) reduced the risk of heart disease in postmenopausal women. (When the trial began, HT ... also was increasingly being used for prevention of heart disease.) The study found that HT increased the risk ...

  1. Clinical Trials

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    Full Text Available ... trials optimization . Building 31 31 Center Drive Bethesda, MD 20892 Learn more about getting to NIH Get ... and Funding Building 31 31 Center Drive Bethesda, MD 20892 Learn more about getting to NIH Connect ...

  2. Clinical Trials

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    Full Text Available ... that the participants' rights are protected. The IRB reviews the trial's protocol before the study begins. An IRB will only approve research that deals with medically important questions ...

  3. Clinical Trials

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    Full Text Available ... to preexisting differences between the patients. Usually, a computer program makes the group assignments. Masking The term " ... under way. For example, some trials are stopped early if benefits from a strategy or treatment are ...

  4. Clinical Trials

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    Full Text Available ... treatment of menopausal symptoms. It also was increasingly being used for prevention of heart disease.) The study ... a trial are due to the different strategies being used, not to preexisting differences between the patients. ...

  5. Clinical Trials

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    Full Text Available ... Precision Medicine Activities Obesity, Nutrition, and Physical Activity Population and Epidemiology Studies Women’s Health All Science A- ... assumed that trial results were valid for other populations as well. Researchers now realize that women and ...

  6. Understanding Clinical Trials

    Science.gov (United States)

    Watch these videos to learn about some basic aspects of cancer clinical trials such as the different phases of clinical trials, methods used to protect patient safety, and how the costs of clinical trials are covered.

  7. Clinical Trials

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    Full Text Available ... Diseases Heart and Vascular Diseases Precision Medicine Activities Obesity, Nutrition, and Physical Activity Population and Epidemiology Studies ... the NHLBI's Children and Clinical Studies Web page. Children and Clinical Studies Learn more about Children and ...

  8. Clinical Trials

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    Full Text Available ... go to the NHLBI's Children and Clinical Studies Web page. Children and Clinical Studies Learn more about ... Protections The U.S. Department of Health and Human Services’ (HHS’) Office for Human Research Protections (OHRP) oversees ...

  9. Clinical Trials

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    Full Text Available ... Studies Learn more about Children and Clinical Studies Importance of Children in Clinical Studies Children have often ... rights that help protect them. Scientific Oversight Institutional Review Board Institutional review boards (IRBs) help provide scientific ...

  10. Clinical Trials

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    Full Text Available ... taking the same treatment the same way. These patients are closely watched by Data and Safety Monitoring Boards. Even if you don't directly ... risk procedures (such as gene therapy) or vulnerable patients (such as ... trial for safety problems or differences in results among different groups. ...

  11. Clinical Trials

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    Full Text Available ... edge approaches, such as gene therapy or new biological treatments. Health insurance and health care providers don't ... of a trial, early if the strategy or treatment is having harmful effects. Food and Drug Administration In the United States, the Food and ...

  12. Clinical Trials

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    Full Text Available ... U.S. Departments of Defense and Veterans Affairs; private companies; universities; and nonprofit organizations. NIH Institutes and Centers (including the NHLBI) usually sponsor trials that test principles or strategies. For example, one NHLBI study explored whether the ...

  13. Clinical Trials

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    Full Text Available ... Trials Publications and Resources Health Education and Awareness The Science Science Home Blood Disorders and Blood Safety Sleep ... Activity Population and Epidemiology Studies Women’s Health All Science A-Z Grants ... in the Press Research Features All Events Past Events Upcoming ...

  14. Clinical Trials

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    Full Text Available ... Diseases Heart and Vascular Diseases Precision Medicine Activities Obesity, Nutrition, and Physical Activity Population and Epidemiology Studies ... include factors such as a patient's age and gender, the type and stage of disease, ... helps ensure that any differences observed during a trial are due to the ...

  15. Clinical Trials

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    Full Text Available ... an important gap in information and education for parents, clinicians, researchers, children, and the general public. What to Expect During ... trial's potential risks are greater than minimal, both parents must give permission for their child to enroll. Also, children aged 7 and older ...

  16. Clinical Case Registries: Simultaneous Local and National Disease Registries for Population Quality Management

    Science.gov (United States)

    Backus, Lisa I.; Gavrilov, Sergey; Loomis, Timothy P.; Halloran, James P.; Phillips, Barbara R.; Belperio, Pamela S.; Mole, Larry A.

    2009-01-01

    The Department of Veterans Affairs (VA) has a system-wide, patient-centric electronic medical record system (EMR) within which the authors developed the Clinical Case Registries (CCR) to support population-centric delivery and evaluation of VA medical care. To date, the authors have applied the CCR to populations with human immunodeficiency virus (HIV) and hepatitis C virus (HCV). Local components use diagnosis codes and laboratory test results to identify patients who may have HIV or HCV and support queries on local care delivery with customizable reports. For each patient in a local registry, key EMR data are transferred via HL7 messaging to a single national registry. From 128 local registry systems, over 60,000 and 320,000 veterans in VA care have been identified as having HIV and HCV, respectively, and entered in the national database. Local and national reports covering demographics, resource usage, quality of care metrics and medication safety issues have been generated. PMID:19717794

  17. Clinical Trials

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    Full Text Available ... and useful results, which in turn will improve public health. We offer a variety of funding mechanisms tailored to planning and conducting clinical ... Privacy Policy Freedom of Information Act (FOIA) Accessibility ...

  18. Clinical Trials

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    Full Text Available ... and devices specific to children. Resources for a Wide Range of Audiences The Children and Clinical Studies ... have not only shaped medical practice around the world, but have improved the health of millions of ...

  19. Clinical Trials

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    Full Text Available ... the strategy or treatment is having harmful effects. Food and Drug Administration In the United States, the Food and Drug Administration (FDA) provides oversight for clinical ...

  20. Clinical Trials

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    Full Text Available ... harmful effects. Food and Drug Administration In the United States, the Food and Drug Administration (FDA) provides oversight ... of research studies at the NIH Clinical Center, America's research hospital, located on the NIH campus in ...

  1. Clinical Trials

    Medline Plus

    Full Text Available ... seems promising, the next step may involve animal testing. This shows how the approach affects a living body and whether it's harmful. However, an approach that works well in the lab or animals doesn't always work well in people. Thus, research in humans is needed. For safety purposes, clinical ...

  2. Clinical Trials

    Medline Plus

    Full Text Available ... the final stages of a long and careful research process. The process often begins in a laboratory (lab), where scientists first develop and test new ideas. If an approach seems ... Thus, research in humans is needed. For safety purposes, clinical ...

  3. Implementation of a prospective pregnancy registry for antiretroviral based HIV prevention trials.

    Science.gov (United States)

    Mhlanga, Felix G; Noguchi, Lisa; Balkus, Jennifer E; Kabwigu, Samuel; Scheckter, Rachel; Piper, Jeanna; Watts, Heather; O'Rourke, Colin; Torjesen, Kristine; Brown, Elizabeth R; Hillier, Sharon L; Beigi, Richard

    2018-02-01

    Safety data on pregnancy and fetal outcomes among women in HIV prevention trials are urgently needed to inform use of effective antiretroviral agents for HIV prevention. We describe an effective, efficient, and novel method to prospectively collect perinatal safety data concurrent with on-going parent clinical trials. The Microbicide Trials Network (MTN)-016 study is a multinational prospective pregnancy exposure registry designed to capture pregnancy and neonatal outcomes. Studies currently contributing data to this registry included phase I and II safety trials with planned exposures to candidate HIV prevention agents, as well as phase IIB and III efficacy trials capturing data on pregnancy and infant outcomes following inadvertent fetal exposure during study participation. To date, participants from two phase I studies and two effectiveness trials have participated in MTN-016, resulting in 420 pregnant women and 381 infants enrolled. Infant retention has been high, with 329 of 381 (86%) infants completing the 12-month follow-up visit. In a research setting context, it is feasible to establish and implement a prospective, multinational HIV chemoprophylaxis pregnancy registry that will generate pregnancy exposure data in a robust fashion.

  4. Clinical trial methodology

    National Research Council Canada - National Science Library

    Peace, Karl E; Chen, Ding-Geng

    2011-01-01

    ... in the pharmaceutical industry, Clinical trial methodology emphasizes the importance of statistical thinking in clinical research and presents the methodology as a key component of clinical research...

  5. Research Areas - Clinical Trials

    Science.gov (United States)

    Information about NCI programs and initiatives that sponsor, conduct, develop, or support clinical trials, including NCI’s Clinical Trial Network (NCTN) and NCI Community Oncology Research Program (NCORP) initiatives.

  6. Clinical trial methodology

    National Research Council Canada - National Science Library

    Peace, Karl E; Chen, Ding-Geng

    2011-01-01

    "Now viewed as its own scientific discipline, clinical trial methodology encompasses the methods required for the protection of participants in a clinical trial and the methods necessary to provide...

  7. Impact of clinical registries on quality of patient care and clinical outcomes: A systematic review.

    Directory of Open Access Journals (Sweden)

    Dewan Md Emdadul Hoque

    Full Text Available Clinical quality registries (CQRs are playing an increasingly important role in improving health outcomes and reducing health care costs. CQRs are established with the purpose of monitoring quality of care, providing feedback, benchmarking performance, describing pattern of treatment, reducing variation and as a tool for conducting research.To synthesise the impact of clinical quality registries (CQRs as an 'intervention' on (I mortality/survival; (II measures of outcome that reflect a process or outcome of health care; (III health care utilisation; and (IV healthcare-related costs.The following electronic databases were searched: MEDLINE, EMBASE, CENTRAL, CINAHL and Google Scholar. In addition, a review of the grey literature and a reference check of citations and reference lists within articles was undertaken to identify relevant studies in English covering the period January 1980 to December 2016. The PRISMA-P methodology, checklist and standard search strategy using pre-defined inclusion and exclusion criteria and structured data extraction tools were used. Data on study design and methods, participant characteristics attributes of included registries and impact of the registry on outcome measures and/or processes of care were extracted.We identified 30102 abstracts from which 75 full text articles were assessed and finally 17 articles were selected for synthesis. Out of 17 studies, six focused on diabetes care, two on cardiac diseases, two on lung diseases and others on organ transplantations, rheumatoid arthritis, ulcer healing, surgical complications and kidney disease. The majority of studies were "before after" design (#11 followed by cohort design (#2, randomised controlled trial (#2, experimental non randomised study and one cross sectional comparison. The measures of impact of registries were multifarious and included change in processes of care, quality of care, treatment outcomes, adherence to guidelines and survival. Sixteen of 17

  8. Construction of ethics in clinical research: clinical trials registration

    Directory of Open Access Journals (Sweden)

    C. A. Caramori

    2007-01-01

    Full Text Available Scientific development that has been achieved through decades finds in clinical research a great possibility of translating findings to human health application. Evidence given by clinical trials allows everyone to have access to the best health services. However, the millionaire world of pharmaceutical industries has stained clinical research with doubt and improbability. Study results (fruits of controlled clinical trials and scientific publications (selective, manipulated and with wrong conclusions led to an inappropriate clinical practice, favoring the involved economic aspect. In 2005, the International Committee of Medical Journal Editors (ICMJE, supported by the World Association of Medical Editors, started demanding as a requisite for publication that all clinical trials be registered at the database ClinicalTrials.gov. In 2006, the World Health Organization (WHO created the International Clinical Trial Registry Platform (ICTRP, which gathers several registry centers from all over the world, and required that all researchers and pharmaceutical industries register clinical trials. Such obligatory registration has progressed and will extend to all scientific journals indexed in all worldwide databases. Registration of clinical trials means another step of clinical research towards transparency, ethics and impartiality, resulting in real evidence to the forthcoming changes in clinical practice as well as in the health situation.

  9. Linking ClinicalTrials.gov and PubMed to track results of interventional human clinical trials.

    Directory of Open Access Journals (Sweden)

    Vojtech Huser

    Full Text Available OBJECTIVE: In an effort to understand how results of human clinical trials are made public, we analyze a large set of clinical trials registered at ClinicalTrials.gov, the world's largest clinical trial registry. MATERIALS AND METHODS: We considered two trial result artifacts: (1 existence of a trial result journal article that is formally linked to a registered trial or (2 the deposition of a trial's basic summary results within the registry. RESULTS: The study sample consisted of 8907 completed, interventional, phase 2-or-higher clinical trials that were completed in 2006-2009. The majority of trials (72.2% had no structured trial-article link present. A total of 2367 trials (26.6% deposited basic summary results within the registry. Of those, 969 trials (10.9% were classified as trials with extended results and 1398 trials (15.7% were classified as trials with only required basic results. The majority of the trials (54.8% had no evidence of results, based on either linked result articles or basic summary results (silent trials, while a minimal number (9.2% report results through both registry deposition and publication. DISCUSSION: Our study analyzes the body of linked knowledge around clinical trials (which we refer to as the "trialome". Our results show that most trials do not report results and, for those that do, there is minimal overlap in the types of reporting. We identify several mechanisms by which the linkages between trials and their published results can be increased. CONCLUSION: Our study shows that even when combining publications and registry results, and despite availability of several information channels, trial sponsors do not sufficiently meet the mandate to inform the public either via a linked result publication or basic results submission.

  10. Hepatitis C: Clinical Trials

    Science.gov (United States)

    ... and Public Home » Hepatitis C » Treatment Decisions Viral Hepatitis Menu Menu Viral Hepatitis Viral Hepatitis Home For ... can I find out about participating in a hepatitis C clinical trial? Many trials are being conducted ...

  11. Managing clinical trials

    Directory of Open Access Journals (Sweden)

    Kenyon Sara

    2010-07-01

    Full Text Available Abstract Managing clinical trials, of whatever size and complexity, requires efficient trial management. Trials fail because tried and tested systems handed down through apprenticeships have not been documented, evaluated or published to guide new trialists starting out in this important field. For the past three decades, trialists have invented and reinvented the trial management wheel. We suggest that to improve the successful, timely delivery of important clinical trials for patient benefit, it is time to produce standard trial management guidelines and develop robust methods of evaluation.

  12. [The user´s reporting from the national registry of catheter aortic valve implantations (Czech TAVI Registry): the possibilities of the analytical reports based on the database system TrialDB2].

    Science.gov (United States)

    Bláha, Milan; Kala, Petr; Klimeš, Daniel; Bernat, Ivo; Branny, Marian; Cervinka, Pavel; Horák, Jan; Kočka, Viktor; Mates, Martin; Němec, Petr; Pešl, Ladislav; Stípal, Roman; Sťásek, Josef; Zelízko, Michael

    2014-10-01

    Assessment of the treatment procedures and their results is increasingly important in current medicine. The emphasis is put on an effective use of the health technologies (HTA). Unlike randomised studies, which involve strictly selected groups of patients who meet inclusion and exclusion criterias, the multicentre clinical registries provide a real-life picture of the treatment safety and effectiveness. Well prepared registries involve both research database and a friendly user interface enabling collection of parametric and easily analyzable data. Although there are some technological aspects aiming to ensure a maximum quality of entered data, cooperation with the users and data managers is essential. Such a registry, otherwise meaningful, must provide answers to previously defined medical hypotheses. Regular feedback to users (so called benchmarking or reporting) is considered to be of key importance. The Czech TAVI Registry (CTR) is a good example of reaching all of the above defined criterias. This registry contains data of approximately 95 % of all transcatheter aortic valve implantations (TAVI) performed in the Czech Republic. It is based on a general system aimed at the design of clinical trials, namely the TrialDB2 (a database system for clinical registries developed by the Institute of Biostatistics and Analyses at the Masaryk University (IBA MU). CTR has been run as an English-language version under the auspices of the Czech Society of Cardiology and represents one of the top-quality registries maintained by IBA MU. This paper presents the currently available database systems and some reports from this particular registry.

  13. Types of Cancer Clinical Trials

    Science.gov (United States)

    Information about the several types of cancer clinical trials, including treatment trials, prevention trials, screening trials, supportive and palliative care trials. Each type of trial is designed to answer different research questions.

  14. Clinical trials of homoeopathy.

    Science.gov (United States)

    Kleijnen, J; Knipschild, P; ter Riet, G

    1991-01-01

    OBJECTIVE--To establish whether there is evidence of the efficacy of homoeopathy from controlled trials in humans. DESIGN--Criteria based meta-analysis. Assessment of the methodological quality of 107 controlled trials in 96 published reports found after an extensive search. Trials were scored using a list of predefined criteria of good methodology, and the outcome of the trials was interpreted in relation to their quality. SETTING--Controlled trials published world wide. MAIN OUTCOME MEASURES--Results of the trials with the best methodological quality. Trials of classical homoeopathy and several modern varieties were considered separately. RESULTS--In 14 trials some form of classical homoeopathy was tested and in 58 trials the same single homoeopathic treatment was given to patients with comparable conventional diagnosis. Combinations of several homoeopathic treatments were tested in 26 trials; isopathy was tested in nine trials. Most trials seemed to be of very low quality, but there were many exceptions. The results showed a positive trend regardless of the quality of the trial or the variety of homeopathy used. Overall, of the 105 trials with interpretable results, 81 trials indicated positive results whereas in 24 trials no positive effects of homoeopathy were found. The results of the review may be complicated by publication bias, especially in such a controversial subject as homoeopathy. CONCLUSIONS--At the moment the evidence of clinical trials is positive but not sufficient to draw definitive conclusions because most trials are of low methodological quality and because of the unknown role of publication bias. This indicates that there is a legitimate case for further evaluation of homoeopathy, but only by means of well performed trials. PMID:1825800

  15. Fundamentals of clinical trials

    CERN Document Server

    Friedman, Lawrence M; DeMets, David L; Reboussin, David M; Granger, Christopher B

    2015-01-01

    This is the fifth edition of a very successful textbook on clinical trials methodology, written by recognized leaders who have long and extensive experience in all areas of clinical trials. The three authors of the first four editions have been joined by two others who add great expertise.  Most chapters have been revised considerably from the fourth edition.  A chapter on regulatory issues has been included and the chapter on data monitoring has been split into two and expanded.  Many contemporary clinical trial examples have been added.  There is much new material on adverse events, adherence, issues in analysis, electronic data, data sharing, and international trials.  This book is intended for the clinical researcher who is interested in designing a clinical trial and developing a protocol. It is also of value to researchers and practitioners who must critically evaluate the literature of published clinical trials and assess the merits of each trial and the implications for the care and treatment of ...

  16. Bridging the gap between the randomised clinical trial world and the real world by combination of population-based registry and electronic health record data : A case study in haemato-oncology

    NARCIS (Netherlands)

    Kibbelaar, R E; Oortgiesen, B E; van der Wal-Oost, A M; Boslooper, K; Coebergh, J W; Veeger, N J G M; Joosten, P; Storm, H; van Roon, E N; Hoogendoorn, M

    2017-01-01

    Randomised clinical trials (RCTs) are considered the basis of evidence-based medicine. It is recognised more and more that application of RCT results in daily practice of clinical decision-making is limited because the RCT world does not correspond with the clinical real world. Recent strategies

  17. ClinicalTrials.gov

    Data.gov (United States)

    U.S. Department of Health & Human Services — Provides patients, family members, health care professionals, and members of the public easy access to information on clinical trials for a wide range of diseases...

  18. Cancer clinical trials

    International Nuclear Information System (INIS)

    Scheurlen, A.; Kay, R.; Baum, M.

    1988-01-01

    This book contains the proceedings on Cancer clinical trials: A critical appraisal. Topics covered include: Scientific fundamentals; Heterogeneous treatment effects; On combining information: Historical controls, overviews, and comprehensive cohort studies; and assessment of quality of life

  19. Clinical registry for rheumatoid arthritis; a preliminary analysis

    International Nuclear Information System (INIS)

    Fakhr, A.; Hakim, F.; Zaidi, S.K.; Sharif, A.

    2017-01-01

    To establish a clinical registry for Rheumatoid Arthritis and delineate the most common symptoms that rheumatoid arthritis (RA) patients experience in our set up. Study Design: Cross sectional study. Place and Duration of Study: Study was carried out at Military Hospital (MH) Rawalpindi at Rheumatology Department during the period of Jan 2013 to Jun 2015. Material and Methods: A clinical registry for Rheumatoid Arthritis was developed as per criteria jointly developed by American College of Rheumatology (ACR) along with European League against Rheumatism (EULAR) (2010). Fifty-eight patients were registered after their informed consent and approval by Military Hospital (MH) Rawalpindi ethical committee. Age, gender and relevant clinical parameters of RA patients were recorded on case report forms and stored for analysis in the RA registry in Excel 2010. The figures were reported in frequencies and percentages. Results: Multiple joint pains (48.28%), fever (24.14%), morning stiffness of joints (22.41%) were the most common symptoms in RA patients. Other clinical manifestations included painful bilateral swollen joints (13.79%), pain in different parts of the body (10.34%), Raynaud's phenomenon (10.34%), malaise (8.62%), swollen body parts (8.62%), ulcers (8.62%), fatigue (6.90%), nodules on skin/elbow/interphalangeal joints (6.90%), deformities of fingers/ hand (3.45%), redness of eyes (3.45%), body rash (3.45%), inability to walk (3.45%), cervical lymphadenopathy (1.72%), stiffness of spine (1.72%) and myalgias (1.72%). Conclusion: It is concluded that multiple joint pains, fever and morning stiffness of joints are the most common symptoms of RA patients. (author)

  20. Falsificationism and clinical trials.

    Science.gov (United States)

    Senn, S J

    1991-11-01

    The relevance of the philosophy of Sir Karl Popper to the planning, conduct and analysis of clinical trials is examined. It is shown that blinding and randomization can only be regarded as valuable for the purpose of refuting universal hypotheses. The purpose of inclusion criteria is also examined. It is concluded that a misplaced belief in induction is responsible for many false notions regarding clinical trials.

  1. Methodology series module 4: Clinical trials

    Directory of Open Access Journals (Sweden)

    Maninder Singh Setia

    2016-01-01

    Full Text Available In a clinical trial, study participants are (usually divided into two groups. One group is then given the intervention and the other group is not given the intervention (or may be given some existing standard of care. We compare the outcomes in these groups and assess the role of intervention. Some of the trial designs are (1 parallel study design, (2 cross-over design, (3 factorial design, and (4 withdrawal group design. The trials can also be classified according to the stage of the trial (Phase I, II, III, and IV or the nature of the trial (efficacy vs. effectiveness trials, superiority vs. equivalence trials. Randomization is one of the procedures by which we allocate different interventions to the groups. It ensures that all the included participants have a specified probability of being allocated to either of the groups in the intervention study. If participants and the investigator know about the allocation of the intervention, then it is called an "open trial." However, many of the trials are not open - they are blinded. Blinding is useful to minimize bias in clinical trials. The researcher should familiarize themselves with the CONSORT statement and the appropriate Clinical Trials Registry of India.

  2. Methodology Series Module 4: Clinical Trials.

    Science.gov (United States)

    Setia, Maninder Singh

    2016-01-01

    In a clinical trial, study participants are (usually) divided into two groups. One group is then given the intervention and the other group is not given the intervention (or may be given some existing standard of care). We compare the outcomes in these groups and assess the role of intervention. Some of the trial designs are (1) parallel study design, (2) cross-over design, (3) factorial design, and (4) withdrawal group design. The trials can also be classified according to the stage of the trial (Phase I, II, III, and IV) or the nature of the trial (efficacy vs. effectiveness trials, superiority vs. equivalence trials). Randomization is one of the procedures by which we allocate different interventions to the groups. It ensures that all the included participants have a specified probability of being allocated to either of the groups in the intervention study. If participants and the investigator know about the allocation of the intervention, then it is called an "open trial." However, many of the trials are not open - they are blinded. Blinding is useful to minimize bias in clinical trials. The researcher should familiarize themselves with the CONSORT statement and the appropriate Clinical Trials Registry of India.

  3. Transparency and public accessibility of clinical trial information in Croatia: how it affects patient participation in clinical trials.

    Science.gov (United States)

    Šolić, Ivana; Stipčić, Ana; Pavličević, Ivančica; Marušić, Ana

    2017-06-15

    Despite increased visibility of clinical trials through international trial registries, patients often remain uninformed of their existence, especially if they do not have access to adequate information about clinical research, including the language of the information. The aim of this study was to describe the context for transparency of clinical trials in Croatia in relation to countries in Central and Eastern Europe, and to assess how informed Croatian patients are about clinical trials and their accessibility. We assessed the transparency of clinical trials from the data available in the public domain. We also conducted an anonymous survey on a convenience sample of 257 patients visiting two family medicine offices or an oncology department in south Croatia, and members of national patients' associations. Despite legal provisions for transparency of clinical trials in Croatia, they are still not sufficiently visible in the public domain. Among countries from Central and Eastern Europe, Croatia has the fewest number of registered trials in the EU Clinical Trials Registry. 66% of the patients in the survey were aware of the existence of clinical trials but only 15% were informed about possibilities of participating in a trial. Although 58% of the respondents were willing to try new treatments, only 6% actually participated in a clinical trial. Only 2% of the respondents were aware of publicly available trial registries. Our study demonstrates that there is low transparency of clinical trials in Croatia, and that Croatian patients are not fully aware of clinical trials and the possibilities of participating in them, despite reported availability of Internet resources and good communication with their physicians. There is a need for active policy measures to increase the awareness of and access to clinical trials to patients in Croatia, particularly in their own language.

  4. Regorafenib in the Real-Life Clinical Practice: Data from the Czech Registry.

    Science.gov (United States)

    Kopeckova, Katerina; Buchler, Tomas; Bortlicek, Zbynek; Hejduk, Karel; Chloupkova, Renata; Melichar, Bohuslav; Pokorna, Petra; Tomasek, Jiri; Linke, Zdenek; Petruzelka, Lubos; Kiss, Igor; Prausova, Jana

    2017-02-01

    To describe the use of regorafenib for the treatment of metastatic colorectal cancer (mCRC) in clinical practice in the Czech Republic, and to describe the clinical outcomes of patients in terms of safety and survival. The data of patients treated with regorafenib were extracted from the national CORECT registry. The CORECT registry is a non-interventional post-marketing database, gathering information about patients with CRC and treated with targeted agents. Twenty oncology centres in the Czech Republic contributed to this registry. Collected data included patients' characteristics, disease history, cancer treatments, response to treatments and safety. A total of 148 patients treated with regorafenib in clinical practice were analysed. At regorafenib initiation, almost all patients were fully active or slightly restricted in physical activity. Regorafenib was not administered as first-line treatment in any patient. Median progression-free survival was 3.5 months and median overall survival was 9.3 months. One-year survival rate was 44.6 %. Four partial responses were observed and 51 stable diseases. Progression was observed in 66 patients (44.6 %). The main reported adverse events were skin toxicity (5.4 %) and fatigue (2.0 %). Regorafenib is a well-established treatment for pretreated patients with mCRC, however real-life data are scarce. Our results demonstrated slightly better efficacy of regorafenib and better safety profile in patients with mCRC compared to the randomised trials.

  5. Conducting clinical trials in Singapore.

    Science.gov (United States)

    Woo, K T

    1999-04-01

    All clinical trials in Singapore will now have to conform to the Medicines (Clinical Trials) Amended Regulations 1998 and the Singapore Good Clinical Practice (GCP) Guidelines 1998. The Medical Clinical Research Committee (MCRC) has been established to oversee the conduct of clinical drug trials in Singapore and together with the legislations in place, these will ensure that clinical trials conducted in Singapore are properly controlled and the well-being of trial subjects are safe guarded. All clinical drug trials require a Clinical Trial Certificate from the MCRC before the trial can proceed. The hospital ethics committee (EC) vets the application for a trial certificate before it is sent to MCRC. The drug company sponsoring the trial has to indemnify the trial investigators and the hospital for negligence arising from the trial. The MCRC, apart from ensuring the safety of trial subjects, has to provide continuing review of the clinical trial and monitors adverse events in the course of the trial. The EC will conduct continuing review of clinical trials. When a non-drug clinical trial is carried out, the EC will ensure that the proposed protocol addresses ethical concerns and meets regulatory requirements for such trials. There is great potential for pharmaceutical Research & Development (R&D) in Singapore. We must develop our skills and infrastructure in clinical trials to enable Singapore to be a regional hub for R&D of drugs in Asia.

  6. Prospective registration of clinical trials in India: strategies, achievements & challenges.

    Science.gov (United States)

    Tharyan, Prathap

    2009-02-01

    This paper traces the development of the Clinical Trial Registry-India (CTRI) against the backdrop of the inequities in healthcare and the limitations in the design, conduct, regulation, oversight and reporting of clinical trials in India. It describes the scope and goals of the CTRI, the data elements it seeks and the process of registering clinical trials. It reports progress in trial registration in India and discusses the challenges in ensuring that healthcare decisions are informed by all the evidence. A descriptive survey of developments in clinical trial registration in India from publications in the Indian medical literature supplemented by first hand knowledge of these developments and an evaluation of how well clinical trials registered in the CTRI up to 10 January, 2009 comply with the requirements of the CTRI and the World Health Organization's International Clinical Trial Registry (WHO ICTRP). Considerable inequities exist within the Indian health system. Deficiencies in healthcare provision and uneven regulation of, and access to, affordable healthcare co-exists with a large private health system of uneven quality. India is now a preferred destination for outsourced clinical trials but is plagued by poor ethical oversight of the many trial sites and scant information of their existence. The CTRI's vision of conforming to international requirements for transparency and accountability but also using trial registration as a means of improving trial design, conduct and reporting led to the selection of registry-specific dataset items in addition to those endorsed by the WHO ICTRP. Compliance with these requirements is good for the trials currently registered but these trials represent only a fraction of the trials in progress in India. Prospective trial registration is a reality in India. The challenges facing the CTRI include better engagement with key stakeholders to ensure increased prospective registration of clinical trials and utilization of

  7. Prospective registration, bias risk and outcome-reporting bias in randomised clinical trials of traditional Chinese medicine

    DEFF Research Database (Denmark)

    Liu, Jian-Ping; Han, Mei; Li, Xin-Xue

    2013-01-01

    Clinical trials on Traditional Chinese Medicine (TCM) should be registered in a publicly accessible international trial register and report on all outcomes. We systematically assessed and evaluated TCM trials in registries with their subsequent publications.......Clinical trials on Traditional Chinese Medicine (TCM) should be registered in a publicly accessible international trial register and report on all outcomes. We systematically assessed and evaluated TCM trials in registries with their subsequent publications....

  8. Walking the talk: the need for a trial registry for development interventions

    DEFF Research Database (Denmark)

    Rasmussen, Ole Dahl; Malchow-Møller, Nikolaj; Andersen, Thomas Barnebeck

    2011-01-01

    Recent advances in the use of randomised control trials to evaluate the effect of development interventions promise to enhance our knowledge of what works and why. A core argument supporting randomised studies is the claim that they have high internal validity. The authors argue that this claim...... is weak as long as a trial registry of development interventions is not in place. Without a trial registry, the possibilities for data mining, created by analyses of multiple outcomes and subgroups, undermine internal validity. Drawing on experience from evidence-based medicine and recent examples from...

  9. OARSI Clinical Trials Recommendations

    DEFF Research Database (Denmark)

    Kraus, V B; Blanco, F J; Englund, M

    2015-01-01

    The objective of this work was to describe requirements for inclusion of soluble biomarkers in osteoarthritis (OA) clinical trials and progress toward OA-related biomarker qualification. The Guidelines for Biomarkers Working Group, representing experts in the field of OA biomarker research from...

  10. OARSI Clinical Trials Recommendations

    DEFF Research Database (Denmark)

    McAlindon, T. E.; Driban, J. B.; Henrotin, Y.

    2015-01-01

    The goal of this document is to update the original OARSI recommendations specifically for the design, conduct, and reporting of clinical trials that target symptom or structure modification among individuals with knee osteoarthritis (OA). To develop recommendations for the design, conduct...

  11. Ethics of clinical trials.

    Science.gov (United States)

    Palter, S F

    1996-05-01

    The modern clinical trial is a form of human experimentation. There is a long history of disregard for individual rights of the patient in this context, and special attention must be paid to ethical guidelines for these studies. Clinical trials differ in basic ways from clinical practice. Foremost is the introduction of outside interests, beyond those of the patient's health, into the doctor-patient therapeutic alliance. Steps must be taken to protect the interests of the patient when such outside influence exists. Kantian moral theory and the Hippocratic oath dictate that the physician must respect the individual patient's rights and hold such interests paramount. These principles are the basis for informed consent. Randomization of patients is justified when a condition of equipoise exists. The changing nature of health care delivery in the United States introduces new outside interests into the doctor-patient relationship.

  12. SPIRIT 2013 Statement: defining standard protocol items for clinical trials.

    Science.gov (United States)

    Chan, An-Wen; Tetzlaff, Jennifer M; Altman, Douglas G; Laupacis, Andreas; Gøtzsche, Peter C; Krle A-Jerić, Karmela; Hrobjartsson, Asbjørn; Mann, Howard; Dickersin, Kay; Berlin, Jesse A; Dore, Caroline J; Parulekar, Wendy R; Summerskill, William S M; Groves, Trish; Schulz, Kenneth F; Sox, Harold C; Rockhold, Frank W; Rennie, Drummond; Moher, David

    2015-12-01

    The protocol of a clinical trial serves as the foundation for study planning, conduct, reporting, and appraisal. However, trial protocols and existing protocol guidelines vary greatly in content and quality. This article describes the systematic development and scope of SPIRIT (Standard Protocol Items: Recommendations for Interventional Trials) 2013, a guideline for the minimum content of a clinical trial protocol. The 33-item SPIRIT checklist applies to protocols for all clinical trials and focuses on content rather than format. The checklist recommends a full description of what is planned; it does not prescribe how to design or conduct a trial. By providing guidance for key content, the SPIRIT recommendations aim to facilitate the drafting of high-quality protocols. Adherence to SPIRIT would also enhance the transparency and completeness of trial protocols for the benefit of investigators, trial participants, patients, sponsors, funders, research ethics committees or institutional review boards, peer reviewers, journals, trial registries, policymakers, regulators, and other key stakeholders.

  13. Clinical Outcomes of Splenectomy in Children: Report of the Splenectomy in Congenital Hemolytic Anemia (SICHA) Registry

    Science.gov (United States)

    Rice, Henry E; Englum, Brian R; Rothman, Jennifer; Leonard, Sarah; Reiter, Audra; Thornburg, Courtney; Brindle, Mary; Wright, Nicola; Heeney, Matthew M; Smithers, Charles; Brown, Rebeccah L; Kalfa, Theodosia; Langer, Jacob C; Cada, Michaela; Oldham, Keith T; Scott, J Paul; St. Peter, Shawn; Sharma, Mukta; Davidoff, Andrew M.; Nottage, Kerri; Bernabe, Kathryn; Wilson, David B; Dutta, Sanjeev; Glader, Bertil; Crary, Shelley E; Dassinger, Melvin S; Dunbar, Levette; Islam, Saleem; Kumar, Manjusha; Rescorla, Fred; Bruch, Steve; Campbell, Andrew; Austin, Mary; Sidonio, Robert; Blakely, Martin L

    2014-01-01

    The outcomes of children with congenital hemolytic anemia (CHA) undergoing total splenectomy (TS) or partial splenectomy (PS) remain unclear. In this study, we collected data from 100 children with CHA who underwent TS or PS from 2005–2013 at 16 sites in the Splenectomy in Congenital Hemolytic Anemia (SICHA) consortium using a patient registry. We analyzed demographics and baseline clinical status, operative details, and outcomes at 4, 24, and 52 weeks after surgery. Results were summarized as hematologic outcomes, short-term adverse events (AEs) (≤ 30 days after surgery), and long-term AEs (31–365 days after surgery). For children with hereditary spherocytosis, after surgery there was an increase in hemoglobin (baseline 10.1 ± 1.8 gm/dl, 52 week 12.8 ± 1.6 gm/dl; mean ± SD), decrease in reticulocyte and bilirubin as well as control of symptoms. Children with sickle cell disease had control of clinical symptoms after surgery, but had no change in hematologic parameters. There was an 11% rate of short-term AEs and 11% rate of long-term AEs. As we accumulate more subjects and longer follow-up, use of a patient registry should enhance our capacity for clinical trials and engage all stakeholders in the decision-making process. PMID:25382665

  14. Pilot for the Australian Breast Device Registry (ABDR): a national opt-out clinical quality registry for breast device surgery.

    Science.gov (United States)

    Hopper, Ingrid; Best, Renee L; McNeil, John J; Mulvany, Catherine M; Moore, Colin C M; Elder, Elisabeth; Pase, Marie; Cooter, Rodney D; Evans, Sue M

    2017-12-28

    To establish a pilot clinical quality registry (CQR) to monitor the quality of care and device performance for breast device surgery in Australia. All patients having breast device surgery from contributing hospitals in Australia. A literature review was performed which identified quality indicators for breast device surgery. A pilot CQR was established in 2011 to capture prospective data on breast device surgery. An interim Steering Committee and Management Committee were established to provide clinical governance, and guide quality indicator selection. The registry's minimum dataset was formulated in consultation with stakeholder groups; potential quality indicators were assessed in terms of (1) importance and relevance, (2) usability, (3) feasibility to collect and (4) scientific validity. Data collection was by a two-sided paper-based form with manual data entry. Seven sites were recruited, including one public hospital, four private hospitals and two day surgeries. Patients were recruited and opt-out consent used. The pilot breast device registry provides high-quality population-based data. It provides a model for developing a national CQR for breast devices; its minimum dataset and quality indicators reflect the opinions of the broad range of stakeholders. It is easily scalable, and has formed the basis for other international surgical groups establishing similar registries. © Article author(s) (or their employer(s) unless otherwise stated in the text of the article) 2017. All rights reserved. No commercial use is permitted unless otherwise expressly granted.

  15. Tools in a clinical information system supporting clinical trials at a Swiss University Hospital.

    Science.gov (United States)

    Weisskopf, Michael; Bucklar, Guido; Blaser, Jürg

    2014-12-01

    Issues concerning inadequate source data of clinical trials rank second in the most common findings by regulatory authorities. The increasing use of electronic clinical information systems by healthcare providers offers an opportunity to facilitate and improve the conduct of clinical trials and the source documentation. We report on a number of tools implemented into the clinical information system of a university hospital to support clinical research. In 2011/2012, a set of tools was developed in the clinical information system of the University Hospital Zurich to support clinical research, including (1) a trial registry for documenting metadata on the clinical trials conducted at the hospital, (2) a patient-trial-assignment-tool to tag patients in the electronic medical charts as participants of specific trials, (3) medical record templates for the documentation of study visits and trial-related procedures, (4) online queries on trials and trial participants, (5) access to the electronic medical records for clinical monitors, (6) an alerting tool to notify of hospital admissions of trial participants, (7) queries to identify potentially eligible patients in the planning phase as trial feasibility checks and during the trial as recruitment support, and (8) order sets to facilitate the complete and accurate performance of study visit procedures. The number of approximately 100 new registrations per year in the voluntary trial registry in the clinical information system now matches the numbers of the existing mandatory trial registry of the hospital. Likewise, the yearly numbers of patients tagged as trial participants as well as the use of the standardized trial record templates increased to 2408 documented trial enrolments and 190 reports generated/month in the year 2013. Accounts for 32 clinical monitors have been established in the first 2 years monitoring a total of 49 trials in 16 clinical departments. A total of 15 months after adding the optional feature of

  16. Gateways to clinical trials.

    Science.gov (United States)

    Bayés, M; Rabasseda, X; Prous, J R

    2007-12-01

    Gateways to Clinical Trials are a guide to the most recent clinical trials in current literature and congresses. The data in the following tables has been retrieved from the Clinical Trials Knowledge Area of Prous Science Intergrity, the drug discovery and development portal, http://integrity.prous.com. This issue focuses on the following selection of drugs: 249553, 2-Methoxyestradiol; Abatacept, Adalimumab, Adefovir dipivoxil, Agalsidase beta, Albinterferon alfa-2b, Aliskiren fumarate, Alovudine, Amdoxovir, Amlodipine besylate/atorvastatin calcium, Amrubicin hydrochloride, Anakinra, AQ-13, Aripiprazole, AS-1404, Asoprisnil, Atacicept, Atrasentan; Belimumab, Bevacizumab, Bortezomib, Bosentan, Botulinum toxin type B, Brivaracetam; Catumaxomab, Cediranib, Cetuximab, cG250, Ciclesonide, Cinacalcet hydrochloride, Curcumin, Cypher; Darbepoetin alfa, Denosumab, Dihydrexidine; Eicosapentaenoic acid/docosahexaenoic acid, Entecavir, Erlotinib hydrochloride, Escitalopram oxalate, Etoricoxib, Everolimus, Ezetimibe; Febuxostat, Fenspiride hydrochloride, Fondaparinux sodium; Gefitinib, Ghrelin (human), GSK-1562902A; HSV-tk/GCV; Iclaprim, Imatinib mesylate, Imexon, Indacaterol, Insulinotropin, ISIS-112989; L-Alanosine, Lapatinib ditosylate, Laropiprant; Methoxy polyethylene glycol-epoetin-beta, Mipomersen sodium, Motexafin gadolinium; Natalizumab, Nimotuzumab; OSC, Ozarelix; PACAP-38, Paclitaxel nanoparticles, Parathyroid Hormone-Related Protein-(1-36), Pasireotide, Pegfilgrastim, Peginterferon alfa-2a, Peginterferon alfa-2b, Pemetrexed disodium, Pertuzumab, Picoplatin, Pimecrolimus, Pitavastatin calcium, Plitidepsin; Ranelic acid distrontium salt, Ranolazine, Recombinant human relaxin H2, Regadenoson, RFB4(dsFv)-PE38, RO-3300074, Rosuvastatin calcium; SIR-Spheres, Solifenacin succinate, Sorafenib, Sunitinib malate; Tadalafil, Talabostat, Taribavirin hydrochloride, Taxus, Temsirolimus, Teriparatide, Tiotropium bromide, Tipifarnib, Tirapazamine, Tocilizumab; UCN-01, Ularitide

  17. Gateways to clinical trials.

    Science.gov (United States)

    Bayés, M; Rabasseda, X; Prous, J R

    2005-04-01

    Gateways to Clinical Trials is a guide to the most recent clinical trials in current literature and congresses. The data in the following tables has been retrieved from the Clinical Trials Knowledge Area of Prous Science Integrity, the drug discovery and development portal, http://integrity. prous.com. This issue focuses on the following selection of drugs: ABX-IL-8, Acclaim, adalimumab, AGI-1067, alagebrium chloride, alemtuzumab, Alequel, Androgel, anti-IL-12 MAb, AOD-9604, aripiprazole, atomoxetine hydrochloride; Biphasic insulin aspart, bosentan, botulinum toxin type B, bovine lactoferrin, brivudine; Cantuzumab mertansine, CB-1954, CDB-4124, CEA-TRICOM, choriogonadotropin alfa, cilansetron, CpG-10101, CpG-7909, CTL-102, CTL-102/CB-1954; DAC:GRF, darbepoetin alfa, davanat-1, decitabine, del-1 Genemedicine, dexanabinol, dextofisopam, dnaJP1, dronedarone hydrochloride, dutasteride; Ecogramostim, eletriptan, emtricitabine, EPI-hNE-4, eplerenone, eplivanserin fumarate, erlotinib hydrochloride, ertapenem sodium, escitalopram oxalate, esomeprazole magnesium, etoricoxib, ezetimibe; Falecalcitriol, fingolimod hydrochloride; Gepirone hydrochloride; HBV-ISS, HSV-2 theracine, human insulin; Imatinib mesylate, Indiplon, insulin glargine, ISAtx-247; L612 HuMAb, levodopa/carbidopa/entacapone, lidocaine/prilocaine, LL-2113AD, lucinactant, LY-156735; Meclinertant, metelimumab, morphine hydrochloride, morphine-6-glucuronide; Natalizumab, nimotuzumab, NX-1207, NYVAC-HIV C; Omalizumab, onercept, osanetant; PABA, palosuran sulfate, parathyroid hormone (human recombinant), parecoxib sodium, PBI-1402, PCK-3145, peginterferon alfa-2a, peginterferon alfa-2b, peginterferon alfa-2b/ribavirin, pemetrexed disodium, pimecrolimus, PINC, pregabalin; Ramelteon, rasagiline mesilate, rasburicase, rimonabant hydrochloride, RO-0098557, rofecoxib, rosiglitazone maleate/metformin hydrochloride; Safinamide mesilate, SHL-749, sitaxsentan sodium, sparfosic acid, SprayGel, squalamine, St. John's Wort

  18. Re-Engineering Alzheimer Clinical Trials: Global Alzheimer's Platform Network.

    Science.gov (United States)

    Cummings, J; Aisen, P; Barton, R; Bork, J; Doody, R; Dwyer, J; Egan, J C; Feldman, H; Lappin, D; Truyen, L; Salloway, S; Sperling, R; Vradenburg, G

    2016-06-01

    Alzheimer's disease (AD) drug development is costly, time-consuming, and inefficient. Trial site functions, trial design, and patient recruitment for trials all require improvement. The Global Alzheimer Platform (GAP) was initiated in response to these challenges. Four GAP work streams evolved in the US to address different trial challenges: 1) registry-to-cohort web-based recruitment; 2) clinical trial site activation and site network construction (GAP-NET); 3) adaptive proof-of-concept clinical trial design; and 4) finance and fund raising. GAP-NET proposes to establish a standardized network of continuously funded trial sites that are highly qualified to perform trials (with established clinical, biomarker, imaging capability; certified raters; sophisticated management system. GAP-NET will conduct trials for academic and biopharma industry partners using standardized instrument versions and administration. Collaboration with the Innovative Medicines Initiative (IMI) European Prevention of Alzheimer's Disease (EPAD) program, the Canadian Consortium on Neurodegeneration in Aging (CCNA) and other similar international initiatives will allow conduct of global trials. GAP-NET aims to increase trial efficiency and quality, decrease trial redundancy, accelerate cohort development and trial recruitment, and decrease trial costs. The value proposition for sites includes stable funding and uniform training and trial execution; the value to trial sponsors is decreased trial costs, reduced time to execute trials, and enhanced data quality. The value for patients and society is the more rapid availability of new treatments for AD.

  19. Clinical trials in India.

    Science.gov (United States)

    Maiti, Rituparna; M, Raghavendra

    2007-07-01

    The concept of outsourcing for the development and global studies on new drugs has become widely accepted in the pharmaceutical industry due to its cost and uncertainty. India is going to be the most preferred location for contract pharma research and development due to its huge treatment naïve population, human resources, technical skills, adoption/amendment/implementation of rules/laws by regulatory authorities, and changing economic environment. But still 'miles to go' to fulfill the pre-requisites to ensure India's success. In spite of all the pitfalls, the country is ambitious and optimist to attract multinational pharmaceutical companies to conduct their clinical trials in India.

  20. Clinical features of paediatric pulmonary hypertension : a registry study

    NARCIS (Netherlands)

    Berger, Rolf M. F.; Beghetti, Maurice; Humpl, Tilman; Raskob, Gary E.; Ivy, D. Dunbar; Jing, Zhi-Cheng; Bonnet, Damien; Schulze-Neick, Ingram; Barst, Robyn J.

    2012-01-01

    Background Paediatric pulmonary hypertension, is an important cause of morbidity and mortality, and is insufficiently characterised in children. The Tracking Outcomes and Practice in Pediatric Pulmonary Hypertension (TOPP) registry is a global, prospective study designed to provide information about

  1. [Maraviroc: clinical trials results].

    Science.gov (United States)

    Chidiac, C; Katlama, C; Yeni, P

    2008-03-01

    Just over a decade after identification of chemokine receptors CCR5 and CXCR4 as coreceptors for HIV, maraviroc (Celsentri), the first CCR5 antagonist, has recently obtained its Marketing Authorization in the United States and Europe, for treatment of treatment-experienced adult patients infected with only CCR5-tropic HIV-1 detectable. CCR5 antagonists, after fusion inhibitor enfuvirtide available since 2003, also belong to entry inhibitors. These molecules, unlike previous antiretrovirals, do not target the virus but its target cell by blocking viral penetration. Maraviroc has shown its clinical efficacy in patients failing other antiretroviral classes. Its safety profile was similar to placebo in two large phase III trials. However, careful assessment of both hepatic and immunologic safety of this new therapeutic class is needed. Viral tropism testing has to be investigated before using maraviroc in the clinic, because CCR5 antagonists are not active against CXCR4 viruses. For the moment indicated for the treatment-experienced patient population, maraviroc could in the future benefit to other types of patients, depending on ongoing trials results.

  2. Existing data sources for clinical epidemiology: Aarhus University Clinical Trial Candidate Database, Denmark.

    Science.gov (United States)

    Nørrelund, Helene; Mazin, Wiktor; Pedersen, Lars

    2014-01-01

    Denmark is facing a reduction in clinical trial activity as the pharmaceutical industry has moved trials to low-cost emerging economies. Competitiveness in industry-sponsored clinical research depends on speed, quality, and cost. Because Denmark is widely recognized as a region that generates high quality data, an enhanced ability to attract future trials could be achieved if speed can be improved by taking advantage of the comprehensive national and regional registries. A "single point-of-entry" system has been established to support collaboration between hospitals and industry. When assisting industry in early-stage feasibility assessments, potential trial participants are identified by use of registries to shorten the clinical trial startup times. The Aarhus University Clinical Trial Candidate Database consists of encrypted data from the Danish National Registry of Patients allowing an immediate estimation of the number of patients with a specific discharge diagnosis in each hospital department or outpatient specialist clinic in the Central Denmark Region. The free access to health care, thorough monitoring of patients who are in contact with the health service, completeness of registration at the hospital level, and ability to link all databases are competitive advantages in an increasingly complex clinical trial environment.

  3. [Clinical trials in nursing journals].

    Science.gov (United States)

    Di Giulio, Paola; Campagna, Sara; Dimonte, Valerio

    2014-01-01

    Clinical trials are pivotal for the development of nursing knowledge. To describe the clinical trials published in nursing journals in the last two years and propose some general reflections on nursing research. A search with the key-word trial was done on PubMed (2009-2013) on Cancer Nursing, European Journal of Oncology Nursing, International Journal of Nursing Studies, Journal of Advanced Nursing, Journal of Clinical Nursing and Nursing Research. Of 228 trials identified, 104 (45.8%) were published in the last 2 years. Nurses from Asian countries published the larger number of trials. Educational and supportive interventions were the most studied (61/104 trials), followed by clinical interventions (33/104). Samples were limited and most trials are monocentric. A growing number of trials is published, on issues relevant for the nursing profession, however larger samples and multicentric studies would be necessary.

  4. Gateways to clinical trials.

    Science.gov (United States)

    Bayés, M; Rabasseda, X; Prous, J R

    2006-10-01

    Gateways to Clinical Trials are a guide to the most recent clinical trials in current literature and congresses. The data the following tables have been retrieved from the Clinical Trials Knowledge Area of Prous Science Integrity, the drug discovery and development portal, http://integrity.prous.com. This issues focuses on the following selection of drugs: (-)-Epigallocatechin gallate, (-)-gossypol, 2-deoxyglucose, 3,4-DAP, 7-monohydroxyethylrutoside; Ad5CMV-p53, adalimumab, adefovir dipivoxil, ADH-1, alemtuzumab, aliskiren fumarate, alvocidib hydrochloride, aminolevulinic acid hydrochloride, aminolevulinic acid methyl ester, amrubicin hydrochloride, AN-152, anakinra, anecortave acetate, antiasthma herbal medicine intervention, AP-12009, AP-23573, apaziquone, aprinocarsen sodium, AR-C126532, AR-H065522, aripiprazole, armodafinil, arzoxifene hydrochloride, atazanavir sulfate, atilmotin, atomoxetine hydrochloride, atorvastatin, avanafil, azimilide hydrochloride; Bevacizumab, biphasic insulin aspart, BMS-214662, BN-83495, bortezomib, bosentan, botulinum toxin type B; Caspofungin acetate, cetuximab, chrysin, ciclesonide, clevudine, clofarabine, clopidogrel, CNF-1010, CNTO-328, CP-751871, CX-717, Cypher; Dapoxetine hydrochloride, darifenacin hydrobromide, dasatinib, deferasirox, dextofisopam, dextromethorphan/quinidine sulfate, diclofenac, dronedarone hydrochloride, drotrecogin alfa (activated), duloxetine hydrochloride, dutasteride; Edaravone, efaproxiral sodium, emtricitabine, entecavir, eplerenone, epratuzumab, erlotinib hydrochloride, escitalopram oxalate, etoricoxib, ezetimibe, ezetimibe/simvastatin; Finrozole, fipamezole hydrochloride, fondaparinux sodium, fulvestrant; Gabapentin enacarbil, gaboxadol, gefitinib, gestodene, ghrelin (human); Human insulin, human papillomavirus vaccine; Imatinib mesylate, immunoglobulin intravenous (human), indiplon, insulin detemir, insulin glargine, insulin glulisine, intranasal insulin, istradefylline, i.v. gamma

  5. Review of patient registries in dermatology.

    Science.gov (United States)

    DiMarco, Gabriella; Hill, Dane; Feldman, Steven R

    2016-10-01

    Patient registries are datasets containing information on patients with a particular disease or patients who are undergoing a specific treatment. Our objective was to search for and catalog the types of registries being used in dermatology and investigate their characteristics and uses. We searched Google, the Registry of Patient Registries, Orphanet, and ClinicalTrials.gov to compile a list of dermatology disease registries. We also conducted a literature review on the uses of dermatology registries using PubMed. We identified 48 dermatology patient registries, with 23 distinct diseases represented. We also identified 11 registries used for postmarketing surveillance of skin disease. Our search was limited to registries in English. Registries are commonly used for the study of rare dermatologic diseases and for postsurveillance monitoring of systemic therapies in more common dermatologic diseases, such as psoriasis. Copyright © 2016 American Academy of Dermatology, Inc. Published by Elsevier Inc. All rights reserved.

  6. Review of U.S. registries for psoriasis.

    Science.gov (United States)

    Amin, Mina; No, Daniel J; Wu, Jashin J

    2017-12-01

    Patient registries are databases comprised of standardized clinical data for a specific population of patients with a particular disease or medical condition. Information from patient registries allows clinicians to assess long-lasting outcomes in patients with a specific disease, such as psoriasis. Our primary objective was to identify available psoriasis registries in the United States (U.S.) and evaluate the application of patient registries compared to clinical trials. We searched Google, the Registry of Patient Registries, Orphanet and ClinicalTrials.gov to create a list of U.S. psoriasis registries. We also performed a literature review on the application of psoriasis registries using PubMed. We identified 6 psoriasis patient registries in the United States. Patient registries are frequently used for psoriasis in the U.S. and provide important information about the safety, efficacy and long-term effects of systemic therapies.

  7. Myositis registries and biorepositories: powerful tools to advance clinical, epidemiologic and pathogenic research.

    Science.gov (United States)

    Rider, Lisa G; Dankó, Katalin; Miller, Frederick W

    2014-11-01

    Clinical registries and biorepositories have proven extremely useful in many studies of diseases, especially rare diseases. Given their rarity and diversity, the idiopathic inflammatory myopathies, or myositis syndromes, have benefited from individual researchers' collections of cohorts of patients. Major efforts are being made to establish large registries and biorepositories that will allow many additional studies to be performed that were not possible before. Here, we describe the registries developed by investigators and patient support groups that are currently available for collaborative research purposes. We have identified 46 myositis research registries, including many with biorepositories, which have been developed for a wide variety of purposes and have resulted in great advances in understanding the range of phenotypes, clinical presentations, risk factors, pathogenic mechanisms, outcome assessment, therapeutic responses, and prognoses. These are now available for collaborative use to undertake additional studies. Two myositis patient registries have been developed for research, and myositis patient support groups maintain demographic registries with large numbers of patients available to be contacted for potential research participation. Investigator-initiated myositis research registries and biorepositories have proven extremely useful in understanding many aspects of these rare and diverse autoimmune diseases. These registries and biorepositories, in addition to those developed by myositis patient support groups, deserve continued support to maintain the momentum in this field as they offer major opportunities to improve understanding of the pathogenesis and treatment of these diseases in cost-effective ways.

  8. Design, development and deployment of a Diabetes Research Registry to facilitate recruitment in clinical research.

    Science.gov (United States)

    Tan, Meng H; Bernstein, Steven J; Gendler, Stephen; Hanauer, David; Herman, William H

    2016-03-01

    A major challenge in conducting clinical trials/studies is the timely recruitment of eligible subjects. Our aim is to develop a Diabetes Research Registry (DRR) to facilitate recruitment by matching potential subjects interested in research with approved clinical studies using study entry criteria abstracted from their electronic health records (EHR). A committee with expertise in diabetes, quality improvement, information technology, and informatics designed and developed the DRR. Using a hybrid approach, we identified and consented patients interested in research, abstracted their EHRs to assess common eligibility criteria, and contacted them about their interest in participating in specific studies. Investigators submit their requests with study entry criteria to the DRR which then provides a list of potential subjects who may be directly contacted for their study. The DRR meets all local, regional and federal regulatory requirements. After 5 years, the DRR has over 5000 registrants. About 30% have type 1 diabetes and 70% have type 2 diabetes. There are almost equal proportions of men and women. During this period, 31 unique clinical studies from 19 unique investigators requested lists of potential subjects for their studies. Eleven grant applications from 10 unique investigators used aggregated counts of potentially eligible subjects in their applications. The DRR matches potential subjects interested in research with approved clinical studies using study entry criteria abstracted from their EHR. By providing large lists of potentially eligible study subjects quickly, the DRR facilitated recruitment in 31 clinical studies. Copyright © 2016 Elsevier Inc. All rights reserved.

  9. The impact of a model-based clinical regional registry for attention-deficit hyperactivity disorder.

    Science.gov (United States)

    Zanetti, Michele; Cartabia, Massimo; Didoni, Anna; Fortinguerra, Filomena; Reale, Laura; Mondini, Matteo; Bonati, Maurizio

    2017-09-01

    This article describes the development and clinical impact of the Italian Regional ADHD Registry, aimed at collecting and monitoring diagnostic and therapeutic pathways of care for attention-deficit hyperactivity disorder children and adolescents, launched by the Italian Lombardy Region in June 2011. In particular, the model-based software used to run the registry and manage clinical care data acquisition and monitoring, is described. This software was developed using the PROSAFE programme, which is already used for data collection in many Italian intensive care units, as a stand-alone interface case report form. The use of the attention-deficit hyperactivity disorder regional registry led to an increase in the appropriateness of the clinical management of all patients included in the registry, proving to be an important instrument in ensuring an appropriate healthcare strategy for children and adolescents with attention-deficit/hyperactivity disorder.

  10. Practical and conceptual issues of clinical trial registration for Brazilian researchers

    Directory of Open Access Journals (Sweden)

    Carolina Gomes Freitas

    Full Text Available CONTEXT AND OBJECTIVE: Clinical trial registration is a prerequisite for publication in respected scientific journals. Recent Brazilian regulations also require registration of some clinical trials in the Brazilian Clinical Trials Registry (ReBEC but there is little information available about practical issues involved in the registration process. This article discusses the importance of clinical trial registration and the practical issues involved in this process. DESIGN AND SETTING: Descriptive study conducted by researchers within a postgraduate program at a public university in São Paulo, Brazil. METHODS: Information was obtained from clinical trial registry platforms, article reference lists and websites (last search: September 2014 on the following topics: definition of a clinical trial, history, purpose and importance of registry platforms, the information that should be registered and the registration process. RESULTS: Clinical trial registration aims to avoid publication bias and is required by Brazilian journals indexed in LILACS and SciELO and by journals affiliated to the International Committee of Medical Journal Editors (ICMJE. Recent Brazilian regulations require that all clinical trials (phases I to IV involving new drugs to be marketed in this country must be registered in ReBEC. The pros and cons of using different clinical trial registration platforms are discussed. CONCLUSIONS: Clinical trial registration is important and various mechanisms to enforce its implementation now exist. Researchers should take into account national regulations and publication requirements when choosing the platform on which they will register their trial.

  11. Cross-Over Clinical Trials?

    Directory of Open Access Journals (Sweden)

    Latif Gachkar

    2017-01-01

    Full Text Available Abstract Cross-Over Clinical Trials in comparison with Parallel groups clinical trials have some advantages such as control of confounding variables, small sample size, and short time to implement the research project. But this type of research has few essential limitations that discusses in this monogram.

  12. Streamlining cardiovascular clinical trials to improve efficiency and generalisability.

    Science.gov (United States)

    Zannad, Faiez; Pfeffer, Marc A; Bhatt, Deepak L; Bonds, Denise E; Borer, Jeffrey S; Calvo-Rojas, Gonzalo; Fiore, Louis; Lund, Lars H; Madigan, David; Maggioni, Aldo Pietro; Meyers, Catherine M; Rosenberg, Yves; Simon, Tabassome; Stough, Wendy Gattis; Zalewski, Andrew; Zariffa, Nevine; Temple, Robert

    2017-08-01

    Controlled trials provide the most valid determination of the efficacy and safety of an intervention, but large cardiovascular clinical trials have become extremely costly and complex, making it difficult to study many important clinical questions. A critical question, and the main objective of this review, is how trials might be simplified while maintaining randomisation to preserve scientific integrity and unbiased efficacy assessments. Experience with alternative approaches is accumulating, specifically with registry-based randomised controlled trials that make use of data already collected. This approach addresses bias concerns while still capitalising on the benefits and efficiencies of a registry. Several completed or ongoing trials illustrate the feasibility of using registry-based controlled trials to answer important questions relevant to daily clinical practice. Randomised trials within healthcare organisation databases may also represent streamlined solutions for some types of investigations, although data quality (endpoint assessment) is likely to be a greater concern in those settings. These approaches are not without challenges, and issues pertaining to informed consent, blinding, data quality and regulatory standards remain to be fully explored. Collaboration among stakeholders is necessary to achieve standards for data management and analysis, to validate large data sources for use in randomised trials, and to re-evaluate ethical standards to encourage research while also ensuring that patients are protected. The rapidly evolving efforts to streamline cardiovascular clinical trials have the potential to lead to major advances in promoting better care and outcomes for patients with cardiovascular disease. © Article author(s) (or their employer(s) unless otherwise stated in the text of the article) 2017. All rights reserved. No commercial use is permitted unless otherwise expressly granted.

  13. Social media in clinical trials.

    Science.gov (United States)

    Thompson, Michael A

    2014-01-01

    Social media has potential in clinical trials for pointing out trial issues, addressing barriers, educating, and engaging multiple groups involved in cancer clinical research. Social media is being used in clinical trials to highlight issues such as poor accrual and barriers; educate potential participants and physicians about clinical trial options; and is a potential indirect or direct method to improve accrual. We are moving from a passive "push" of information to patients to a "pull" of patients requesting information. Patients and advocates are often driving an otherwise reluctant health care system into communication. Online patient communities are creating new information repositories. Potential clinical trial participants are using the Twittersphere and other sources to learn about potential clinical trial options. We are seeing more organized patient-centric and patient-engaged forums with the potential to crowd source to improve clinical trial accrual and design. This is an evolving process that will meet many individual, institutional, and regulatory obstacles as we move forward in a changed research landscape.

  14. Protocol for a scoping review of post-trial extensions of randomised controlled trials using individually linked administrative and registry data.

    Science.gov (United States)

    Fitzpatrick, Tiffany; Perrier, Laure; Tricco, Andrea C; Straus, Sharon E; Jüni, Peter; Zwarenstein, Merrick; Lix, Lisa M; Smith, Mark; Rosella, Laura C; Henry, David A

    2017-02-17

    Well-conducted randomised controlled trials (RCTs) provide the least biased estimates of intervention effects. However, RCTs are costly and time-consuming to perform and long-term follow-up of participants may be hampered by lost contacts and financial constraints. Advances in computing and population-based registries have created new possibilities for increasing the value of RCTs by post-trial extension using linkage to routinely collected administrative/registry data in order to determine long-term interventional effects. There have been recent important examples, including 20+ years follow-up studies of trials of pravastatin and mammography. Despite the potential value of post-trial extension, there has been no systematic study of this literature. This scoping review aims to characterise published post-trial extension studies, assess their value, and identify any potential challenges associated with this approach. This review will use the recommended methods for scoping reviews. We will search MEDLINE, EMBASE and the Cochrane Central Register of Controlled Trials. A draft search strategy is included in this protocol. Review of titles and abstracts, full texts of potentially eligible studies and data/information extraction will be conducted independently by pairs of investigators. Eligible studies will be RCTs that investigated healthcare interventions that were extended by individual linkage to administrative/registry/electronic medical records data after the completion of the planned follow-up period. Information concerning the original trial, characteristics of the extension study, any clinical, policy or ethical implications and methodological or practical challenges will be collected using standardised forms. As this study uses secondary data, and does not include person-level data, ethics approval is not required. We aim to disseminate these findings through journals and conferences targeting triallists and researchers involved in health data linkage. We

  15. Demographic, clinical and antibody characteristics of patients with digital ulcers in systemic sclerosis: data from the DUO Registry

    NARCIS (Netherlands)

    Denton, Christopher P.; Krieg, Thomas; Guillevin, Loic; Schwierin, Barbara; Rosenberg, Daniel; Silkey, Mariabeth; Zultak, Maurice; Matucci-Cerinic, Marco; Stetter, M.; Lackner, K.; Tomi, N.; Hafner, F.; Brodmann, M.; Kuen-Spiegel, M.; Kolle, H.; Raffier, B.; Hamberger, N.; Metz, S.; Siebel, C.; Trummer, M.; Thonhofer, R.; Illmer, X.; Trautinger, F.; Schmidt, P.; Rintelen, B.; Sautner, J.; Willfort-Ehringer, A.; Margeta, C.; Monshi, B.; Pirkhammer, D.; Richter, L.; Holzer, G.; Minmair, G.; Broll, H.; Takacs, M.; Hirschl, M.; Mesaric, P.; Feldmann, R.; Semmelweis, K.; Hundstorfer, M.; Reinhart, V.; Maurer, B.; Verner, D.; Distler, O.; Schmidt-Bosshard, R.; Bohmova, J.; Prochazkova, L.; Nemec, P.; Fojtik, Z.; Soukup, T.; Smrzova, A.; Suchy, D.; Zemanova, I.; Becvar, R.; Gawlik, A.; Koch, M.; Rauen, T.; Voss, B.; Kurthen, R.; Unholzer, A.; Starz, H.; Welzel, J.; Plaumann, K.; Merk, B.; Bloching, H. H.; Moosig, F.; Frey, P.; Kahl, S.; Schleenbecker, H.; Storck-Mueller, K.; Schwarting, A.; Hazenbiller, A.; Nichelmann, V.; Flaig, W.; Rumbaur, C.; Boesenberg, I.; Schmeiser, T.; Marx, J.; Mayer, L.; Stein, T.; Ochs, W.; Rasche, C.; Worm, M.; Riemekasten, G.; Deuschle, K.; Becker, M.; Kleiner, H. J.; Schulze, K.; Tiggers, C.; Peters, J.; Kirschke, J.; Schaefer, C.; Monshausen, M.; Mengden, T.; Sadeghlar, F.; Seidel, M.; Hillebrecht, C.; Andresen, J.; Reemtsen, R.; Stoeckl, F.; Sperling, S.; Podda, M.; Wagner, N.; Guenzel, J.; Wuerzburg, I.; Luethke, K.; Enderlein, M.; Kayser, M.; Gerber, A.; Haust, M.; Hoff, N. P.; Mota, R.; Akanay-Diesel, S.; Jahnke, K.; Mettler, S.; Toeller, S.; Zwenger, S.; Klein, E.; Hahn, K.; Beyer, C.; Distler, J.; Katzemich, A.; Erfurt-berge, C.; Sticherling, M.; Schuch, F.; Rapp, P.; Mitchell, A.; Freundlieb, C.; Rushentsova, U.; Himsel, A.; Henkemeier, U.; Eilbacher, P.; Ullrich-Guenther, C.; Neul, S.; Oelsner, M.; Hermanns, G.; Fiene, M.; Gause, A.; Mensing, C.; Klings, D.; Mensing, H.; Messall, J.; Zuper, R.; May, D.; Bruckner, L.; Sheikh, N.; Aries, P.; Kirchberg, S.; Funkert, A.; Blank, N.; Lupaschko, S.; Schwuerzer-Voit, M.; Meier, L.; Herr, U.; Meier, U.; Neek, G.; Wernitzsch, H.; Pfoehler, C.; Assmann, G.; Vosswinkel, J.; Krog, B.; Wollersdorfer, E.; Oltmann-Schroeder, J.; Zeuner, R.; Uhlig, S.; Barth, S.; Huegel, R.; Glaeser, R.; Rabe, B.; Schuster, J.; Scholz, J.; Kremer, K.; Robakidze-Torbahn, M.; Moinzadeh, P.; Mittag, M.; Dohse, A.; Muhlack, A.; Schultz, L.; Schult, S.; Frambach, Y.; Kettenbach, A.; Fell, I.; Schweda, K.; Steinbrink, K.; Podobinska, M.; Harmuth, W.; Nielen, C.; Kaczmarczyk, A.; Kellner, C.; von Oelhafen, J.; von Bildering, P. B.; Kunze, S.; Niedermeier, A.; Messer, G.; Sardy, M.; Bekou, V.; Belloni, B.; Huettig, B.; Ziai, M.; Hein, R.; Hallecker, A.; Gaubitz, M.; Hallermann, C.; Schmidt, K.; Herrgott, I.; Hildebrandt, B.; Eiden, E.; Guertler, I.; Gernot Scheibl, E.; Brand, H.; Kaeding, U.; Weiss, E.; Reischel, N.; Kern, S.; Baumann, C.; Hellmich, B.; Loeffler, C.; Pflugfelder, J.; Karaenke, P.; Ruchenburg, J.; Blume, J.; Zabel, M.; Deppermann, N.; Chromik, S.; Metzler, C.; Krupp, E.; Rumpel, H.; Krause Rostock, J.-O.; Kneitz, C.; Federow, I.; Schneider, K.; Semmler, M.; Hapke, S.; Barnd, A.; Linke, M.; Kampe-Juzak, E.; Knoebel, K.; Niefanger, K.; Wilhelm, H. U.; Lauterwein, B.; Fierlbeck, G.; Schanz, S.; Pfeiffer, C.; Hassel, R.; Wahn, H.; Schildt, K.; von Elling, A.; Boro, D.; Ebel, J.; Ahmadi, K.; Moritz, D.; Dietl, S.; Dyballa, J.; Alsheimer, B.; Schuetz, N.; Schuart, T.; Mueglich, C.; Tony, H. P.; Marina, P.; Deininger, F.; Hartmann, F.; Olsen, A. B.; Sondergaard, K. H.; Naderi, Y.; Iversen, L. V.; Karlsmark, T.; Knudsen, J. B.; Gil, J. G.; Lopez, J. C. F.; Tasende, J. A. P.; Gonzales, M. F.; Sandoval, A. A.; del Carmen Torres Martin, M.; Corteguera, M.; Barca, B. A.; Montes, I. C.; de la Torre, R. G.; Victoria Egurbide, M.; Pros, A.; Munoz, J.; Simeon, C. P.; Espinosa, G.; Espinposa, G.; Rodriguez, M. A. P.; Castellvi, I.; Mascaro, J. M.; Bellido, D.; Manzanedo, V. S.; Huertas, M. P.; Sanchez, M. D. M.; Trenado, M. S. S.; Garcia, P. V.; Gines Martinez, F.; Angeles Aquirre, M.; del Rio, A. H.; Vazquez, J. L. G.; Coleman, J. V.; Lopez, M. R.; Sanchez, P. S.; Aizpuru, E. M. F.; Mateo, F. J. N.; Callejas, J. L.; Ortego, N.; Santo, M. P.; Rubio, M.; Martin, I.; Cruz, A.; Crespo, M.; Ramos, P. C.; Fernandez, A. S.-A.; Filloy, J. A. M.; Rodriguez, T. R. V.; Marhuenda, A. R.; Blanco, J. J. R.; Hernan, M. G. B.; Mendoza, A. Z.; de la Puente, C.; Rabaneda, E. V.; de Vicuna, R. G.; del Mar Ripoll Macias, M.; del la Pena Lefebvre, P. G.; de Ramon, E.; Camps, M. T.; Fernandez, C.; Miguelez, R.; Uson, J.; Delgado, E. G.; Villaverde, V.; Maceiras, F.; Cruz, J.; Mosquera, J. A.; Mera, A.; Pampin, E. P.; Blanco, J. S.; Maneiro, J. R.; Diaz, J. J.; Losada, L.; Caamano, M.; Fernandez, S.; Insua, S. A.; Laurin, C. U.; Sanchez, J.; Fernandez, N. C.; Becerra, N. D.; Garcia, A.; Nicolas, G. M.; del Carmen Ortega de la O, M.; Rueda, A.; Calvo, J.; Roman Ivorra, J.; Sancho Alegre, J. J.; Barbado, J.; Montes, J.; Saez, L.; Kaarto, A.; Makinen, H.; Madaule, S.; Dadban, A.; Lok, C.; Ferrandiz, D.; Moiton, M. P.; Magy-Bertrand, N.; Taieb, A.; Droitcourt, C.; Belin, E.; Balquiere, S.; Prey, S.; Boulon, C.; Constans, J.; Richez, C.; Sassolas, B.; Misery, L.; Greco, M.; collet, E.; Berthier, S.; Leguy-Seguin, V.; Imbert, B.; Carpentier, P.; Blaise, S.; Maillard, H.; Beneton, N.; Launay, D.; Hachulla, E.; Woijtasik, G.; Charlanne, H.; Lambert, M.; Jourdain, N.; Hatron, P. Y.; Morell, S.; Spars, A.; Couraud, A.; Doeffel-hantz, V.; Fauchais, A. L.; Vidal, E.; Goudran, G.; Bezanahary, H.; Boussely, N.; Manea, P.; Dumonteil, S.; Loustaud-ratti, V.; Hot, A.; Coppere, B.; Desmurs-Clavel, H.; Ninet, J.; Girard-Madoux, M. H.; Granel, B.; Keynote, A.; Khau van Kien, A.; Rullier, P.; Le Quellec, A.; Riviere, S.; Bessis, D.; Cohen, J. D.; Farcas, C.; Granel-brocard, F.; Agard, C.; Durant, C.; Fuzibet, J. G.; Queyrel, V.; Berezne, A.; Guillevin, L.; Mouthon, L.; Frances, C.; Toledano, C.; Cabane, J.; Tiev, K.; Farge, D.; Keshtmand, H.; Lazareth, I.; Priollet, P.; Michon-Pasturel, U.; Wipff, J.; Assous, N.; Cartry, O.; Kostrzwewa, E.; Doutre, M. S.; Blum, L.; Reguiai, Z.; Letremy, A.; Perlat, A.; Cazalets-lacoste, C.; Decaux, O.; Jego, P.; Duval-modeste, A. B.; Deboves, O.; Sordet, C.; Chatelus, E.; Chiffot, H.; Sibillia, J.; Couret, B.; Moulis, G.; Sailler, L.; Adoue, D.; Gaches, F.; Diot, E.; Skowron, F.; Zenone, T.; Quemeneur, T.; Kyndt, X.; Wahl, D.; Zuily, S.; Moline, T.; Bravetti, V.; Galanopoulos, N.; Vasilopoulos, D.; Vlachoyannopoulos, P.; Kritikos, I.; Tsifetaki, N.; Koutroumbas, A.; Garyfallos, A.; Athanassiou, P.; Aslanidis, S.; Kamali, S.; Dimitroulas, T.; Galanopoulo, V.; Elezoglou, A.; Grier, A.; Murray, M.; O'Rourke, M.; Gabrielli, A.; Lapadula, G.; Serafino, L.; Terlizzi, N.; Bellissimo, S.; Stisi, S.; Malavolta, N.; Airo, P.; Vacca, A.; Battaglia, E.; Foti, R.; Mazzuca, S.; Bortoluzzi, A.; Trotta, F.; Galluccio, F.; Marucci, A.; Cantatore, F.; Bucci, R.; Puppo, F.; de Angeli, R.; Grassi, W.; Cipriani, P.; Mazzone, A.; Faggioli, P.; Severino, A.; Scorza, R.; Belloli, L.; Ughi, N.; Antivalle, M.; del Papa, N.; Maglione, W.; Zeni, S.; Ferri, C.; Colaci, M.; Varcasia, G.; Cuomo, G.; Cozzi, F.; Triolo, G.; Gatti, S.; Montecucco, C. M.; Doveri, M.; Nigro, A.; Olivieri, I.; Bajoochi, G.; Rosato, E.; Salsano, F.; Faustini, F.; Ferraccioli, G.; Colonna, L.; Pallotta, S.; Riccieri, V.; Mussi, A.; Bellisai, F.; Galeazzi, M.; Fusaro, E.; Saracco, M.; Pellerito, R.; Masolini, P.; de Vita, S.; Lombardi, S.; Lunardi, C.; Moolenburgh, J. D.; Heurkens, A. H. M.; Voskuyl, A.; Hak, A. E.; Stroes, E. S. K.; Remans, J.; Gerdes, V.; van Woerkom, J. M.; de Long, A. J. L.; Kaasjager, H. A. H.; Visser, H.; Janssen, M.; van Guldener, C.; van Neer, F.; Vos, P.; Peters, A. J.; Hulsmans, H.; Ronday, K.; Goekoop, R.; Ewals, J.; Valentijn, R.; de Bois, M.; Westedt, M. L.; Siewertsz van Reesema, D.; Knifjj-Dutmer, E.; Stolk, J. N.; Willems, H.; Kuiper-geertsma, D. G.; Baudaoin, P.; Fretter, P.; Westra, R.; Sonnaville, P. B. J.; Smit, A.; Bootsma, H.; Brouwer, L.; Bijl, M.; Molders, N.; Lebrun, C.; van der Veen, M. J.; Noordzij, M.; Houben, H.; Landewe, R. M. B.; Vercoutere, W.; Jahangier de Veen, Z. N.; Zijlstra, T. R.; Ubels, F.; Bruyn, G.; Jansen, P.; Schuerwegh, A.; Huizinga, T. W. J.; Paassen, P.; Hurkens, T.; Geurts, M.; van den Hoogen, F.; Vonk, M.; Jacobs, P. J. C.; Groenendael, J. H. L. M.; Seys, P.; van Zeben, D.; van Paassen, H.; Groenendael, J.; Han, K. H.; Wlarvens, M.; van Hagen, M.; van Daele, P.; Dolhain, R.; Gerards, A. H.; van der Lubbe, P.; Kanter, M. D. E.; Muller, W. H.; Ton, E.; van Krugten, M.; van Gameren, I.; Lanting, P.; den Hengst, C.; Gjessdal, C. G.; Hjertaker, S. L.; Madland, T. M.; Bendvold, A.; Bitter, H.; Hoffmann-Vold, A. M.; Midtvedt, O.; Bakland, G.; Aslkaksen, H. K.; Seip, M.; Kalstad, S.; Koldingsnes, W.; Grandauent, B.; Nordvag, B. Y.; Stran, E. K.; Skomsvoll, J.; Andersen, M.; Thomsen, R. S.; Pedersen, T.; Bakkeheim, V.; Cordeiro, A.; Alves, J.; Oliveira, S.; Coelho, P.; Resende, C.; Ponte, C.; Almeida, I.; Silva, I.; Santos, C.; Camara, I.; Costa, J.; Hellstrom, H.; Mohammad, A.; Lind, I.; Lind, K.; Bracin, T.; Liljequist, E.; Vingren, T.; Ostenson, A.; Hermansson, E.; Thorsson, C.; Soderlin, M.; Nordin, A.; Waldheim, E.; Vengemyr, K.; Albertsson, K.; Karlsson, M. L.; Rydvald, Y.; Rizk, M.; Dolnicar, A. S.; Lukac, J.; James, J.; McHugh, N.; Cole, S.; Brown, S.; Hamilton, A.; Faizal, A.; Hall, F.; Murphy, K.; Skingle, S.; Harris, H.; Madhok, F.; Hampson, R.; Baguley, E.; Ogunbambi G, O.; Lamb, J.; Anderson, M.; Moots, R.; White-Alao, B.; Morrison, C.; Dobson, J.; Gordon, P.; Salerno, R.; Denton, C.; Parker, L.; Ochiel, R.; Vincent, R.; Zimba, S.; Ngcozana, T.; Xu, Y.; D'Cruz, D.; Choong, L. M.; Herrick, A.; Wragg, E.; Manning, J.; Moore, T.; Kelsey, C.; Chakravarty, K.; Skyes, H.; Athiveer, P.

    2012-01-01

    The Digital Ulcers Outcome (DUO) Registry was designed to describe the clinical and antibody characteristics, disease course and outcomes of patients with digital ulcers associated with systemic sclerosis (SSc). The DUO Registry is a European, prospective, multicentre, observational, registry of SSc

  16. OARSI Clinical Trials Recommendations

    DEFF Research Database (Denmark)

    Katz, J N; Losina, E; Lohmander, L S

    2015-01-01

    To highlight methodological challenges in the design and conduct of randomized trials of surgical interventions and to propose strategies for addressing these challenges. This paper focuses on three broad areas: enrollment; intervention; and assessment including implications for analysis. For eac...

  17. Quality Assurance for Clinical Trials

    Science.gov (United States)

    Ibbott, Geoffrey S.; Haworth, Annette; Followill, David S.

    2013-01-01

    Cooperative groups, of which the Radiation Therapy Oncology Group is one example, conduct national clinical trials that often involve the use of radiation therapy. In preparation for such a trial, the cooperative group prepares a protocol to define the goals of the trial, the rationale for its design, and the details of the treatment procedure to be followed. The Radiological Physics Center (RPC) is one of several quality assurance (QA) offices that is charged with assuring that participating institutions deliver doses that are clinically consistent and comparable. The RPC does this by conducting a variety of independent audits and credentialing processes. The RPC has compiled data showing that credentialing can help institutions comply with the requirements of a cooperative group clinical protocol. Phantom irradiations have been demonstrated to exercise an institution’s procedures for planning and delivering advanced external beam techniques (1–3). Similarly, RPC data indicate that a rapid review of patient treatment records or planning procedures can improve compliance with clinical trials (4). The experiences of the RPC are presented as examples of the contributions that a national clinical trials QA center can make to cooperative group trials. These experiences illustrate the critical need for comprehensive QA to assure that clinical trials are successful and cost-effective. The RPC is supported by grants CA 10953 and CA 81647 from the National Cancer Institute, NIH, DHHS. PMID:24392352

  18. Clinical trial network for the promotion of clinical research for rare diseases in Japan: muscular dystrophy clinical trial network.

    Science.gov (United States)

    Shimizu, Reiko; Ogata, Katsuhisa; Tamaura, Akemi; Kimura, En; Ohata, Maki; Takeshita, Eri; Nakamura, Harumasa; Takeda, Shin'ichi; Komaki, Hirofumi

    2016-07-11

    Duchenne muscular dystrophy (DMD) is the most commonly inherited neuromuscular disease. Therapeutic agents for the treatment of rare disease, namely "orphan drugs", have recently drawn the attention of researchers and pharmaceutical companies. To ensure the successful conduction of clinical trials to evaluate novel treatments for patients with rare diseases, an appropriate infrastructure is needed. One of the effective solutions for the lack of infrastructure is to establish a network of rare diseases. To accomplish the conduction of clinical trials in Japan, the Muscular dystrophy clinical trial network (MDCTN) was established by the clinical research group for muscular dystrophy, including the National Center of Neurology and Psychiatry, as well as national and university hospitals, all which have a long-standing history of research cooperation. Thirty-one medical institutions (17 national hospital organizations, 10 university hospitals, 1 national center, 2 public hospitals, and 1 private hospital) belong to this network and collaborate to facilitate clinical trials. The Care and Treatment Site Registry (CTSR) calculates and reports the proportion of patients with neuromuscular diseases in the cooperating sites. In total, there are 5,589 patients with neuromuscular diseases in Japan and the proportion of patients with each disease is as follows: DMD, 29 %; myotonic dystrophy type 1, 23 %; limb girdle muscular dystrophy, 11 %; Becker muscular dystrophy, 10 %. We work jointly to share updated health care information and standardized evaluations of clinical outcomes as well. The collaboration with the patient registry (CTSR), allows the MDCTN to recruit DMD participants with specific mutations and conditions, in a remarkably short period of time. Counting with a network that operates at a national level is important to address the corresponding national issues. Thus, our network will be able to contribute with international research activity, which can lead to

  19. Randomised clinical trial

    DEFF Research Database (Denmark)

    Reimer, C; Lødrup, A; Smith, G

    2016-01-01

    of an alginate (Gaviscon Advance, Reckitt Benckiser, Slough, UK) on reflux symptoms in patients with persistent symptoms despite once daily PPI. MethodsThis was a multicentre, randomised, placebo-controlled, 7-day double-blind trial preceded by a 7-day run-in period. Reflux symptoms were assessed using...

  20. Are data from national quality registries used in quality improvement at Swedish hospital clinics?

    Science.gov (United States)

    Fredriksson, Mio; Halford, Christina; Eldh, Ann Catrine; Dahlström, Tobias; Vengberg, Sofie; Wallin, Lars; Winblad, Ulrika

    2017-11-01

    To investigate the use of data from national quality registries (NQRs) in local quality improvement as well as purported key factors for effective clinical use in Sweden. Comparative descriptive: a web survey of all Swedish hospitals participating in three NQRs with different levels of development (certification level). Heads of the clinics and physician(s) at clinics participating in the Swedish Stroke Register (Riksstroke), the Swedish National Registry of Gallstone Surgery and Endoscopic Retrograde Cholangiopancreatography (GallRiks) and the Swedish Lung Cancer Registry (NLCR). Individual and unit level use of NQRs in local quality improvement, and perceptions on data quality, organizational conditions and user motivation. Riksstroke data were reported as most extensively used at individual and unit levels (x̅ 17.97 of 24 and x̅ 27.06 of 35). Data quality and usefulness was considered high for the two most developed NQRs (x̅ 19.86 for Riksstroke and x̅ 19.89 for GallRiks of 25). Organizational conditions were estimated at the same level for Riksstroke and GallRiks (x̅ 12.90 and x̅ 13.28 of 20) while the least developed registry, the NLCR, had lower estimates (x̅ 10.32). In Riksstroke, the managers requested registry data more often (x̅ 15.17 of 20). While there were significant differences between registries in key factors such as management interest, use of NQR data in local quality improvement seems rather prevalent, at least for Riksstroke. The link between the registry's level of development and factors important for routinization of innovations such as NQRs needs investigation. © The Author 2017. Published by Oxford University Press in association with the International Society for Quality in Health Care. All rights reserved. For permissions, please e-mail: journals.permissions@oup.com

  1. Trial publication after registration in ClinicalTrials.Gov: a cross-sectional analysis.

    Directory of Open Access Journals (Sweden)

    Joseph S Ross

    2009-09-01

    Full Text Available ClinicalTrials.gov is a publicly accessible, Internet-based registry of clinical trials managed by the US National Library of Medicine that has the potential to address selective trial publication. Our objectives were to examine completeness of registration within ClinicalTrials.gov and to determine the extent and correlates of selective publication.We examined reporting of registration information among a cross-section of trials that had been registered at ClinicalTrials.gov after December 31, 1999 and updated as having been completed by June 8, 2007, excluding phase I trials. We then determined publication status among a random 10% subsample by searching MEDLINE using a systematic protocol, after excluding trials completed after December 31, 2005 to allow at least 2 y for publication following completion. Among the full sample of completed trials (n = 7,515, nearly 100% reported all data elements mandated by ClinicalTrials.gov, such as intervention and sponsorship. Optional data element reporting varied, with 53% reporting trial end date, 66% reporting primary outcome, and 87% reporting trial start date. Among the 10% subsample, less than half (311 of 677, 46% of trials were published, among which 96 (31% provided a citation within ClinicalTrials.gov of a publication describing trial results. Trials primarily sponsored by industry (40%, 144 of 357 were less likely to be published when compared with nonindustry/nongovernment sponsored trials (56%, 110 of 198; p<0.001, but there was no significant difference when compared with government sponsored trials (47%, 57 of 122; p = 0.22. Among trials that reported an end date, 75 of 123 (61% completed prior to 2004, 50 of 96 (52% completed during 2004, and 62 of 149 (42% completed during 2005 were published (p = 0.006.Reporting of optional data elements varied and publication rates among completed trials registered within ClinicalTrials.gov were low. Without greater attention to reporting of all data

  2. Clinical trials. A pending subject.

    Science.gov (United States)

    Gil-Extremera, B; Jiménez-López, P; Mediavilla-García, J D

    2018-04-01

    Clinical trials are essential tools for the progress of clinical medicine in its diagnostic and therapeutic aspects. Since the first trial in 1948, which related tobacco use with lung cancer, there have been more than 150,000 clinical trials to date in various areas (paediatrics, cardiology, oncology, endocrinology, etc.). This article highlights the importance for all physicians to participate, over the course of their professional career, in a clinical trial, due to the inherent benefits for patients, the progress of medicine and for curricular prestige. The authors have created a synthesis of their experience with clinical trials on hypertension, diabetes, dyslipidaemia and ischaemic heart disease over the course of almost 3 decades. Furthermore, a brief reference has been made to the characteristics of a phase I unit, as well as to a number of research studies currently underway. Copyright © 2017 Elsevier España, S.L.U. and Sociedad Española de Medicina Interna (SEMI). All rights reserved.

  3. Modifying the Clinical Research Infrastructure at a Dedicated Clinical Trials Unit: Assessment of Trial Development, Activation, and Participant Accrual.

    Science.gov (United States)

    Tang, Chad; Hess, Kenneth R; Sanders, Dwana; Davis, Suzanne E; Buzdar, Aman U; Kurzrock, Razelle; Lee, J Jack; Meric-Bernstam, Funda; Hong, David S

    2017-03-15

    Purpose: Information on processes for trials assessing investigational therapeutics is sparse. We assessed the trial development processes within the Department of Investigational Cancer Therapeutics (ICT) at MD Anderson Cancer Center (Houston, TX) and analyzed their effects on the trial activation timeline and enrolment. Experimental Design: Data were from a prospectively maintained registry that tracks all clinical studies at MD Anderson. From this database, we identified 2,261 activated phase I-III trials; 221 were done at the ICT. ICT trials were matched to trials from other MD Anderson departments by phase, sponsorship, and submission year. Trial performance metrics were compared with paired Wilcoxon signed rank tests. Results: We identified three facets of the ICT research infrastructure: parallel processing of trial approval steps; a physician-led research team; and regular weekly meetings to foster research accountability. Separate analyses were conducted stratified by sponsorship [industry (133 ICT and 133 non-ICT trials) or institutional (68 ICT and 68 non-ICT trials)]. ICT trial development was faster from IRB approval to activation (median difference of 1.1 months for industry-sponsored trials vs. 2.3 months for institutional) and from activation to first enrolment (median difference of 0.3 months for industry vs. 1.2 months for institutional; all matched P infrastructure within a large academic cancer center was associated with efficient trial development and participant accrual. Clin Cancer Res; 23(6); 1407-13. ©2016 AACR . ©2016 American Association for Cancer Research.

  4. Transparency in ovarian cancer clinical trial results: ClinicalTrials.gov versus PubMed, Embase and Google scholar.

    Science.gov (United States)

    Roberto, Anna; Radrezza, Silvia; Mosconi, Paola

    2018-04-10

    In recent years the question of the lack of transparency in clinical research has been debated by clinicians, researchers, citizens and their representatives, authors and publishers. This is particularly important for infrequent cancers such as ovarian cancer, where treatment still gives disappointing results in the majority of cases. Our aim was to assess the availability to the public of results in ClinicalTrials.gov, and the frequency of non-publication of results in ClinicalTrials.gov and in PubMed, Embase and Google Scholar. We collected all trials on ovarian cancer identified as "completed status" in the ClinicalTrials.gov registry on 17 January 2017. We checked the availability of the results in ClinicalTrials.gov and systematically identified published manuscripts on results. Out of 2725 trials on ovarian cancer identified, 752 were classified as "completed status". In those closed between 2008 and 2015, excluding phase I, the frequency of results in ClinicalTrials.gov was 35%. Of the 752 completed studies the frequency of published results in PubMed, Embase or Google Scholar ranged from 57.9% to 69.7% in the last years. These findings show a lack of transparency and credibility of research. Citizens or patients' representatives, with the medical community, should continuously support initiatives to improve the publication and dissemination of clinical study results.

  5. OARSI Clinical Trials Recommendations

    DEFF Research Database (Denmark)

    Emery, C. A.; Roos, Ewa M.; Verhagen, E.

    2015-01-01

    The risk of post-traumatic osteoarthritis (PTOA) substantially increases following joint injury. Research efforts should focus on investigating the efficacy of preventative strategies in high quality randomized controlled trials (RCT). The objective of these OARSI RCT recommendations is to inform...... the design, conduct and analytical approaches to RCTs evaluating the preventative effect of joint injury prevention strategies. Recommendations regarding the design, conduct, and reporting of RCTs evaluating injury prevention interventions were established based on the consensus of nine researchers...... internationally with expertise in epidemiology, injury prevention and/or osteoarthritis (OA). Input and resultant consensus was established through teleconference, face to face and email correspondence over a 1 year period. Recommendations for injury prevention RCTs include context specific considerations...

  6. The relational clinical database: a possible solution to the star wars in registry systems.

    Science.gov (United States)

    Michels, D K; Zamieroski, M

    1990-12-01

    In summary, having data from other service areas available in a relational clinical database could resolve many of the problems existing in today's registry systems. Uniting sophisticated information systems into a centralized database system could definitely be a corporate asset in managing the bottom line.

  7. Factors Contributing to Exacerbating Vulnerabilities in Global Clinical Trials

    Science.gov (United States)

    da Silva, Ricardo E.; Amato, Angélica A.; Guilhem, Dirce B.; de Carvalho, Marta R.; Lima, Elisangela da C.; Novaes, Maria Rita C. G.

    2018-01-01

    Background: Although policies and guidelines make use of the concept of vulnerability, few define it. The European Union's directive for clinical trials does not include explanations for or the reasoning behind the designation of certain groups as vulnerable. Emerging economies from lower middle-income countries have, in recent years, had the largest average annual growth rate, as well as increase, in number of clinical trials registered in the US government's database. Nevertheless, careful supervision of research activities has to be ensured. Objective: To describe and analyze the features of the clinical trials involving vulnerable populations in various countries classified by development status and geographic region. Methods: Retrospective study that involved analysis of data obtained from the International Clinical Trials Registry Platform (ICTRP) database between 01/2014 and 12/2014 from countries with (i) highest trial densities during 2005 to 2012, (ii) highest average growth rate in clinical trials, and (iii) greatest trial capabilities. Results: Statistical analysis of this study showed that patients incapable of giving consent personally are 11.4 times more likely to be vulnerable patients than patients who are capable, and that patients in upper-middle-income countries are 1.7 times more likely to be vulnerable patients than patients from high-income countries when participating in global clinical trials. Malaysia (21%), Egypt (20%), Turkey (19%), Israel (18%), and Brazil (17%) had the highest percentages of vulnerable populations involving children. Conclusions: Although the inability to provide consent personally was a factor associated with vulnerability, arbitrary criteria may have been considered when classifying the populations of clinical trials as vulnerable. The EU Clinical Trials Register should provide guidance regarding exactly what aspects or factors should be taken into account to frame given populations as vulnerable, because

  8. Adaptive designs in clinical trials

    Directory of Open Access Journals (Sweden)

    Suresh Bowalekar

    2011-01-01

    Full Text Available In addition to the expensive and lengthy process of developing a new medicine, the attrition rate in clinical research was on the rise, resulting in stagnation in the development of new compounds. As a consequence to this, the US Food and Drug Administration released a critical path initiative document in 2004, highlighting the need for developing innovative trial designs. One of the innovations suggested the use of adaptive designs for clinical trials. Thus, post critical path initiative, there is a growing interest in using adaptive designs for the development of pharmaceutical products. Adaptive designs are expected to have great potential to reduce the number of patients and duration of trial and to have relatively less exposure to new drug. Adaptive designs are not new in the sense that the task of interim analysis (IA/review of the accumulated data used in adaptive designs existed in the past too. However, such reviews/analyses of accumulated data were not necessarily planned at the stage of planning clinical trial and the methods used were not necessarily compliant with clinical trial process. The Bayesian approach commonly used in adaptive designs was developed by Thomas Bayes in the 18th century, about hundred years prior to the development of modern statistical methods by the father of modern statistics, Sir Ronald A. Fisher, but the complexity involved in Bayesian approach prevented its use in real life practice. The advances in the field of computer and information technology over the last three to four decades has changed the scenario and the Bayesian techniques are being used in adaptive designs in addition to other sequential methods used in IA. This paper attempts to describe the various adaptive designs in clinical trial and views of stakeholders about feasibility of using them, without going into mathematical complexities.

  9. Adaptive designs in clinical trials.

    Science.gov (United States)

    Bowalekar, Suresh

    2011-01-01

    In addition to the expensive and lengthy process of developing a new medicine, the attrition rate in clinical research was on the rise, resulting in stagnation in the development of new compounds. As a consequence to this, the US Food and Drug Administration released a critical path initiative document in 2004, highlighting the need for developing innovative trial designs. One of the innovations suggested the use of adaptive designs for clinical trials. Thus, post critical path initiative, there is a growing interest in using adaptive designs for the development of pharmaceutical products. Adaptive designs are expected to have great potential to reduce the number of patients and duration of trial and to have relatively less exposure to new drug. Adaptive designs are not new in the sense that the task of interim analysis (IA)/review of the accumulated data used in adaptive designs existed in the past too. However, such reviews/analyses of accumulated data were not necessarily planned at the stage of planning clinical trial and the methods used were not necessarily compliant with clinical trial process. The Bayesian approach commonly used in adaptive designs was developed by Thomas Bayes in the 18th century, about hundred years prior to the development of modern statistical methods by the father of modern statistics, Sir Ronald A. Fisher, but the complexity involved in Bayesian approach prevented its use in real life practice. The advances in the field of computer and information technology over the last three to four decades has changed the scenario and the Bayesian techniques are being used in adaptive designs in addition to other sequential methods used in IA. This paper attempts to describe the various adaptive designs in clinical trial and views of stakeholders about feasibility of using them, without going into mathematical complexities.

  10. Using an International Clinical Registry of Regional Anesthesia to Identify Targets for Quality Improvement

    Science.gov (United States)

    Sites, Brian D.; Barrington, Michael J.; Davis, Matthew

    2014-01-01

    Background Despite the widespread use of regional anesthesia, limited information on clinical performance exists. Institutions, therefore, have little knowledge of how they are performing in regards to both safety and effectiveness. In this study, we demonstrate how a medical institution (or physician/physician group) may use data from a multi-center clinical registry of regional anesthesia to inform quality improvement strategies. Methods We analyzed data from the International Registry of Regional Anesthesia that includes prospective data on peripheral regional anesthesia procedures from 19 centers located around the world. Using data from the clinical registry, we present summary statistics of the overall safety and effectiveness of regional anesthesia. Furthermore, we demonstrate, using a variety of performance measures, how these data can be used by hospitals to identify areas for quality improvement. To do so, we compare the performance of one member institution (a United States medical center in New Hampshire) to that of the other 18 member institutions of the clinical registry. Results The clinical registry contained information on 23,271 blocks that were performed between June 1, 2011, and May 1, 2014, on 16,725 patients. The overall success rate was 96.7%, immediate complication rate was 2.2%, and the all-cause 60-day rate of neurological sequelae was 8.3 (95% CI, 7.2–9.7) per 10,000. Registry wide major hospital events included 7 wrong site blocks, 3 seizures, 1 complete heart block, 1 retroperitoneal hematoma, and 3 pneumothoraces. For our reference medical center, we identified areas meriting quality improvement. Specifically, after accounting for differences in the age, sex, and health status of patient populations, the reference medical center appeared to rely more heavily on opioids for post procedure management, had higher patient pain scores, and experienced delayed discharge when compared with other member institutions. Conclusions To our

  11. Validation of the "United Registries for Clinical Assessment and Research" (UR-CARE), a European online registry for clinical care and research in Inflammatory Bowel Disease

    DEFF Research Database (Denmark)

    Burisch, Johan; Gisbert, Javier P; Siegmund, Britta

    2018-01-01

    Background: The "United Registries for Clinical Assessment and Research" (UR-CARE) database is an initiative of the European Crohn's and Colitis Organisation (ECCO) to facilitate daily patient care and research studies in inflammatory bowel disease (IBD). Herein, we sought to validate the database......-99%); Case 5: 91% (87-93%)]. These numbers did not differ significantly from those found 6 weeks later (NcNemar's test p>0.05). Conclusion: The UR-CARE database appears to be feasible, valid and reliable as a tool and easy to use regardless of prior user experience and level of clinical IBD experience. UR......-CARE has the potential to enhance future European collaborations regarding clinical research in IBD....

  12. Pediatric Obstructive Uropathy: Clinical Trials

    International Nuclear Information System (INIS)

    Chan, C. M. C.; Scheinman, J. I.; Roth, K. S.

    2005-01-01

    As the powerful tools of molecular biology continue to delineate new concepts of pathogenesis of diseases, new molecular-level therapeutic modalities are certain to emerge. In order to design and execute clinical trials to evaluate outcomes of these new treatment modalities, we will soon need a new supply of investigators with training and experience in clinical research. The slowly-progressive nature of chronic pediatric kidney disease often results in diagnosis being made at a time remote from initial result, and the inherently slow rate of progression makes changes difficult to measure. Thus, development of molecular markers for both diagnosis and rate of progression will be critical to studies of new therapeutic modalities. We will review general aspects of clinical trials and will use current and past studies as examples to illustrate specific points, especially as these apply to chronic kidney disease associated with obstructive uropathy in children. (author)

  13. Clinical Trials in Noninfectious Uveitis

    Science.gov (United States)

    Kim, Jane S.; Knickelbein, Jared E.; Nussenblatt, Robert B.; Sen, H. Nida

    2015-01-01

    The treatment of noninfectious uveitis continues to remain a challenge for many ophthalmologists. Historically, clinical trials in uveitis have been sparse, and thus, most treatment decisions have largely been based on clinical experience and consensus guidelines. The current treatment paradigm favors initiation then tapering of corticosteroids with addition of steroid-sparing immunosuppressive agents for persistence or recurrence of disease. Unfortunately, in spite of a multitude of highly unfavorable systemic effects, corticosteroids are still regarded as the mainstay of treatment for many patients with chronic and refractory noninfectious uveitis. However, with the success of other conventional and biologic immunomodulatory agents in treating systemic inflammatory and autoimmune conditions, interest in targeted treatment strategies for uveitis has been renewed. Multiple clinical trials on steroid-sparing immunosuppressive agents, biologic agents, intraocular corticosteroid implants, and topical ophthalmic solutions have already been completed, and many more are ongoing. This review discusses the results and implications of these clinical trials investigating both alternative and novel treatment options for noninfectious uveitis. PMID:26035763

  14. Azacitidine for Front-Line Therapy of Patients with AML: Reproducible Efficacy Established by Direct Comparison of International Phase 3 Trial Data with Registry Data from the Austrian Azacitidine Registry of the AGMT Study Group

    Directory of Open Access Journals (Sweden)

    Lisa Pleyer

    2017-02-01

    Full Text Available We recently published a clinically-meaningful improvement in median overall survival (OS for patients with acute myeloid leukaemia (AML, >30% bone marrow (BM blasts and white blood cell (WBC count ≤15 G/L, treated with front-line azacitidine versus conventional care regimens within a phase 3 clinical trial (AZA-AML-001; NCT01074047; registered: February 2010. As results obtained in clinical trials are facing increased pressure to be confirmed by real-world data, we aimed to test whether data obtained in the AZA-AML-001 trial accurately represent observations made in routine clinical practice by analysing additional AML patients treated with azacitidine front-line within the Austrian Azacitidine Registry (AAR; NCT01595295; registered: May 2012 and directly comparing patient-level data of both cohorts. We assessed the efficacy of front-line azacitidine in a total of 407 patients with newly-diagnosed AML. Firstly, we compared data from AML patients with WBC ≤ 15 G/L and >30% BM blasts included within the AZA-AML-001 trial treated with azacitidine (“AML-001” cohort; n = 214 with AAR patients meeting the same inclusion criteria (“AAR (001-like” cohort; n = 95. The current analysis thus represents a new sub-analysis of the AML-001 trial, which is directly compared with a new sub-analysis of the AAR. Baseline characteristics, azacitidine application, response rates and OS were comparable between all patient cohorts within the trial or registry setting. Median OS was 9.9 versus 10.8 months (p = 0.616 for “AML-001” versus “AAR (001-like” cohorts, respectively. Secondly, we pooled data from both cohorts (n = 309 and assessed the outcome. Median OS of the pooled cohorts was 10.3 (95% confidence interval: 8.7, 12.6 months, and the one-year survival rate was 45.8%. Thirdly, we compared data from AAR patients meeting AZA-AML-001 trial inclusion criteria (n = 95 versus all AAR patients with World Health Organization (WHO-defined AML (

  15. A federated semantic metadata registry framework for enabling interoperability across clinical research and care domains.

    Science.gov (United States)

    Sinaci, A Anil; Laleci Erturkmen, Gokce B

    2013-10-01

    In order to enable secondary use of Electronic Health Records (EHRs) by bridging the interoperability gap between clinical care and research domains, in this paper, a unified methodology and the supporting framework is introduced which brings together the power of metadata registries (MDR) and semantic web technologies. We introduce a federated semantic metadata registry framework by extending the ISO/IEC 11179 standard, and enable integration of data element registries through Linked Open Data (LOD) principles where each Common Data Element (CDE) can be uniquely referenced, queried and processed to enable the syntactic and semantic interoperability. Each CDE and their components are maintained as LOD resources enabling semantic links with other CDEs, terminology systems and with implementation dependent content models; hence facilitating semantic search, much effective reuse and semantic interoperability across different application domains. There are several important efforts addressing the semantic interoperability in healthcare domain such as IHE DEX profile proposal, CDISC SHARE and CDISC2RDF. Our architecture complements these by providing a framework to interlink existing data element registries and repositories for multiplying their potential for semantic interoperability to a greater extent. Open source implementation of the federated semantic MDR framework presented in this paper is the core of the semantic interoperability layer of the SALUS project which enables the execution of the post marketing safety analysis studies on top of existing EHR systems. Copyright © 2013 Elsevier Inc. All rights reserved.

  16. Benefits and harms in clinical trials of duloxetine for treatment of major depressive disorder

    DEFF Research Database (Denmark)

    Maund, Emma; Tendal, Britta; Hróbjartsson, Asbjørn

    2014-01-01

    , with respect to benefits and major harms. DESIGN: Data on primary efficacy analysis and major harms extracted from each data source and compared. SETTING: Nine randomised placebo controlled trials of duloxetine (total 2878 patients) submitted to the European Medicines Agency (EMA) for marketing approval.......gov and the manufacturer's online clinical trial registry were searched for trial results. RESULTS: Clinical study reports fully described the primary efficacy analysis and major harms (deaths (including suicides), suicide attempts, serious adverse events, and discontinuations because of adverse events). There were minor......, respectively. We also found publication bias in relation to beneficial effects. CONCLUSION: Clinical study reports contained extensive data on major harms that were unavailable in journal articles and in trial registry reports. There were inconsistencies between protocols and clinical study reports and within...

  17. Benefits and harms in clinical trials of duloxetine for treatment of major depressive disorder

    DEFF Research Database (Denmark)

    Maund, Emma; Tendal, Britta; Hróbjartsson, Asbjørn

    2014-01-01

    , respectively. We also found publication bias in relation to beneficial effects. CONCLUSION: Clinical study reports contained extensive data on major harms that were unavailable in journal articles and in trial registry reports. There were inconsistencies between protocols and clinical study reports and within.......gov and the manufacturer's online clinical trial registry were searched for trial results. RESULTS: Clinical study reports fully described the primary efficacy analysis and major harms (deaths (including suicides), suicide attempts, serious adverse events, and discontinuations because of adverse events). There were minor...... clinical study reports. Clinical study reports should be used as the data source for systematic reviews of drugs, but they should first be checked against protocols and within themselves for accuracy and consistency....

  18. Gatekeepers for pragmatic clinical trials.

    Science.gov (United States)

    Whicher, Danielle M; Miller, Jennifer E; Dunham, Kelly M; Joffe, Steven

    2015-10-01

    To successfully implement a pragmatic clinical trial, investigators need access to numerous resources, including financial support, institutional infrastructure (e.g. clinics, facilities, staff), eligible patients, and patient data. Gatekeepers are people or entities who have the ability to allow or deny access to the resources required to support the conduct of clinical research. Based on this definition, gatekeepers relevant to the US clinical research enterprise include research sponsors, regulatory agencies, payers, health system and other organizational leadership, research team leadership, human research protections programs, advocacy and community groups, and clinicians. This article provides a framework to help guide gatekeepers' decision-making related to the use of resources for pragmatic clinical trials. Relevant ethical considerations for gatekeepers include (1) concern for the interests of individuals, groups, and communities affected by the gatekeepers' decisions, including protection from harm and maximization of benefits; (2) advancement of organizational mission and values; and (3) stewardship of financial, human, and other organizational resources. Separate from these ethical considerations, gatekeepers' actions will be guided by relevant federal, state, and local regulations. This framework also suggests that to further enhance the legitimacy of their decision-making, gatekeepers should adopt transparent processes that engage relevant stakeholders when feasible and appropriate. We apply this framework to the set of gatekeepers responsible for making decisions about resources necessary for pragmatic clinical trials in the United States, describing the relevance of the criteria in different situations and pointing out where conflicts among the criteria and relevant regulations may affect decision-making. Recognition of the complex set of considerations that should inform decision-making will guide gatekeepers in making justifiable choices regarding

  19. HIV/AIDS Clinical Trials Fact Sheet

    Science.gov (United States)

    ... AIDS Drugs Clinical Trials Apps skip to content HIV Overview Home Understanding HIV/AIDS Fact Sheets HIV/ ... 4 p.m. ET) Send us an email HIV/AIDS Clinical Trials Last Reviewed: August 25, 2017 ...

  20. NCI National Clinical Trials Network Structure

    Science.gov (United States)

    Learn about how the National Clinical Trials Network (NCTN) is structured. The NCTN is a program of the National Cancer Institute that gives funds and other support to cancer research organizations to conduct cancer clinical trials.

  1. Clinical trials in neurology: design, conduct, analysis

    National Research Council Canada - National Science Library

    Ravina, Bernard

    2012-01-01

    .... Clinical Trials in Neurology aims to improve the efficiency of clinical trials and the development of interventions in order to enhance the development of new treatments for neurologic diseases...

  2. Clinical trial registration in oral health journals.

    Science.gov (United States)

    Smaïl-Faugeron, V; Fron-Chabouis, H; Durieux, P

    2015-03-01

    Prospective registration of randomized controlled trials (RCTs) represents the best solution to reporting bias. The extent to which oral health journals have endorsed and complied with RCT registration is unknown. We identified journals publishing RCTs in dentistry, oral surgery, and medicine in the Journal Citation Reports. We classified journals into 3 groups: journals requiring or recommending trial registration, journals referring indirectly to registration, and journals providing no reference to registration. For the 5 journals with the highest 2012 impact factors in each group, we assessed whether RCTs with results published in 2013 had been registered. Of 78 journals examined, 32 (41%) required or recommended trial registration, 19 (24%) referred indirectly to registration, and 27 (35%) provided no reference to registration. We identified 317 RCTs with results published in the 15 selected journals in 2013. Overall, 73 (23%) were registered in a trial registry. Among those, 91% were registered retrospectively and 32% did not report trial registration in the published article. The proportion of trials registered was not significantly associated with editorial policies: 29% with results in journals that required or recommended registration, 15% in those that referred indirectly to registration, and 21% in those providing no reference to registration (P = 0.05). Less than one-quarter of RCTs with results published in a sample of oral health journals were registered with a public registry. Improvements are needed with respect to how journals inform and require their authors to register their trials. © International & American Associations for Dental Research.

  3. [Principles of controlled clinical trials].

    Science.gov (United States)

    Martini, P

    1962-01-01

    The recovery of the patient should be facilitated as the result of therapeutic research. The basic rule for every therapeutic-clinical trial mist involve a comparison of therapeutic approaches. In acute conditions, such as acute infectious diseases, infarcts, etc., comparisons should be made between two or more groups: the collective therapeutic comparison = the between patients trial. The formation of groups, to be compared one with the other can be justified only if one is reasonably sure that a pathogenic condition indeed exists. In chronic diseases, which extend essentially unchanged over a lengthy period but are nevertheless reversible, therapeutic comparisons may be made between two or more time intervals within the course of the disease in the same individual. This type of therapeutic trial rests primarily upon a (refined!) type of specious reasoning and secondarily, upon modified statistics: the individual therapeutic comparison = the within patient trial. The collective therapeutic comparison, on the one hand, and the individual therapeutic comparison on the other, overlap somewhat in scope. The immediate therapeutic effect is not always an indication of its true value, which may become evident only upon long-term treatment. The short-term trials of therapeutic regimens in an individual must, therefore, be frequently supplemented by long-term trials which can only be carried out by comparing two groups. For many clinical investigations, therefore, the joint efforts of numerous hospitals are absolutely necessary. The second basic rule of therapeutic research is the elimination of secondary causes. The difficulties introduced by these secondary considerations are far greater in therapeutic trials carried out on ambulatory patients than has been hitherto realized. In order to remove subjective secondary causes, the author demanded, in 1931, the use of hidden or illusory media (placebos, dummies) that is, unconscious causative agents. The double blind

  4. Comparison between publicly accessible publications, registries, and protocols of phase III trials indicated persistence of selective outcome reporting.

    Science.gov (United States)

    Zhang, Sheng; Liang, Fei; Li, Wenfeng

    2017-11-01

    The decision to make protocols of phase III randomized controlled trials (RCTs) publicly accessible by leading journals was a landmark event in clinical trial reporting. Here, we compared primary outcomes defined in protocols with those in publications describing the trials and in trial registration. We identified phase III RCTs published between January 1, 2012, and June 30, 2015, in The New England Journal of Medicine, The Lancet, The Journal of the American Medical Association, and The BMJ with available protocols. Consistency in primary outcomes between protocols and registries (articles) was evaluated. We identified 299 phase III RCTs with available protocols in this analysis. Out of them, 25 trials (8.4%) had some discrepancy for primary outcomes between publications and protocols. Types of discrepancies included protocol-defined primary outcome reported as nonprimary outcome in publication (11 trials, 3.7%), protocol-defined primary outcome omitted in publication (10 trials, 3.3%), new primary outcome introduced in publication (8 trials, 2.7%), protocol-defined nonprimary outcome reported as primary outcome in publication (4 trials, 1.3%), and different timing of assessment of primary outcome (4 trials, 1.3%). Out of trials with discrepancies in primary outcome, 15 trials (60.0%) had discrepancies that favored statistically significant results. Registration could be seen as a valid surrogate of protocol in 237 of 299 trials (79.3%) with regard to primary outcome. Despite unrestricted public access to protocols, selective outcome reporting persists in a small fraction of phase III RCTs. Only studies from four leading journals were included, which may cause selection bias and limit the generalizability of this finding. Copyright © 2017 Elsevier Inc. All rights reserved.

  5. Clinical verification of genetic results returned to research participants: findings from a Colon Cancer Family Registry.

    Science.gov (United States)

    Laurino, Mercy Y; Truitt, Anjali R; Tenney, Lederle; Fisher, Douglass; Lindor, Noralane M; Veenstra, David; Jarvik, Gail P; Newcomb, Polly A; Fullerton, Stephanie M

    2017-11-01

    The extent to which participants act to clinically verify research results is largely unknown. This study examined whether participants who received Lynch syndrome (LS)-related findings pursued researchers' recommendation to clinically verify results with testing performed by a CLIA-certified laboratory. The Fred Hutchinson Cancer Research Center site of the multinational Colon Cancer Family Registry offered non-CLIA individual genetic research results to select registry participants (cases and their enrolled relatives) from 2011 to 2013. Participants who elected to receive results were counseled on the importance of verifying results at a CLIA-certified laboratory. Twenty-six (76.5%) of the 34 participants who received genetic results completed 2- and 12-month postdisclosure surveys; 42.3% of these (11/26) participated in a semistructured follow-up interview. Within 12 months of result disclosure, only 4 (15.4%) of 26 participants reported having verified their results in a CLIA-certified laboratory; of these four cases, all research and clinical results were concordant. Reasons for pursuing clinical verification included acting on the recommendation of the research team and informing future clinical care. Those who did not verify results cited lack of insurance coverage and limited perceived personal benefit of clinical verification as reasons for inaction. These findings suggest researchers will need to address barriers to seeking clinical verification in order to ensure that the intended benefits of returning genetic research results are realized. © 2017 The Authors. Molecular Genetics & Genomic Medicine published by Wiley Periodicals, Inc.

  6. Assessing Clinical Trial-Associated Workload in Community-Based Research Programs Using the ASCO Clinical Trial Workload Assessment Tool.

    Science.gov (United States)

    Good, Marjorie J; Hurley, Patricia; Woo, Kaitlin M; Szczepanek, Connie; Stewart, Teresa; Robert, Nicholas; Lyss, Alan; Gönen, Mithat; Lilenbaum, Rogerio

    2016-05-01

    Clinical research program managers are regularly faced with the quandary of determining how much of a workload research staff members can manage while they balance clinical practice and still achieve clinical trial accrual goals, maintain data quality and protocol compliance, and stay within budget. A tool was developed to measure clinical trial-associated workload, to apply objective metrics toward documentation of work, and to provide clearer insight to better meet clinical research program challenges and aid in balancing staff workloads. A project was conducted to assess the feasibility and utility of using this tool in diverse research settings. Community-based research programs were recruited to collect and enter clinical trial-associated monthly workload data into a web-based tool for 6 consecutive months. Descriptive statistics were computed for self-reported program characteristics and workload data, including staff acuity scores and number of patient encounters. Fifty-one research programs that represented 30 states participated. Median staff acuity scores were highest for staff with patients enrolled in studies and receiving treatment, relative to staff with patients in follow-up status. Treatment trials typically resulted in higher median staff acuity, relative to cancer control, observational/registry, and prevention trials. Industry trials exhibited higher median staff acuity scores than trials sponsored by the National Institutes of Health/National Cancer Institute, academic institutions, or others. The results from this project demonstrate that trial-specific acuity measurement is a better measure of workload than simply counting the number of patients. The tool was shown to be feasible and useable in diverse community-based research settings. Copyright © 2016 by American Society of Clinical Oncology.

  7. Current challenges for clinical trials of cardiovascular medical devices.

    Science.gov (United States)

    Zannad, Faiez; Stough, Wendy Gattis; Piña, Ileana L; Mehran, Roxana; Abraham, William T; Anker, Stefan D; De Ferrari, Gaetano M; Farb, Andrew; Geller, Nancy L; Kieval, Robert S; Linde, Cecilia; Redberg, Rita F; Stein, Kenneth; Vincent, Alphons; Woehrle, Holger; Pocock, Stuart J

    2014-07-15

    Several features of cardiovascular devices raise considerations for clinical trial conduct. Prospective, randomized, controlled trials remain the highest quality evidence for safety and effectiveness assessments, but, for instance, blinding may be challenging. In order to avoid bias and not confound data interpretation, the use of objective endpoints and blinding patients, study staff, core labs, and clinical endpoint committees to treatment assignment are helpful approaches. Anticipation of potential bias should be considered and planned for prospectively in a cardiovascular device trial. Prospective, single-arm studies (often referred to as registry studies) can provide additional data in some cases. They are subject to selection bias even when carefully designed; thus, they are generally not acceptable as the sole basis for pre-market approval of high risk cardiovascular devices. However, they complement the evidence base and fill the gaps unanswered by randomized trials. Registry studies present device safety and effectiveness in day-to-day clinical practice settings and detect rare adverse events in the post-market period. No single research design will be appropriate for every cardiovascular device or target patient population. The type of trial, appropriate control group, and optimal length of follow-up will depend on the specific device, its potential clinical benefits, the target patient population and the existence (or lack) of effective therapies, and its anticipated risks. Continued efforts on the part of investigators, the device industry, and government regulators are needed to reach the optimal approach for evaluating the safety and performance of innovative devices for the treatment of cardiovascular disease. Copyright © 2014 Elsevier Ireland Ltd. All rights reserved.

  8. Uses of cancer registries for public health and clinical research in Europe: Results of the European Network of Cancer Registries survey among 161 population-based cancer registries during 2010–2012

    NARCIS (Netherlands)

    Siesling, Sabine; Louwman, W.J.; Kwast, A.; van den Hurk, C.J.G.; O'Callaghan, M.; Rosso, S.; Zanetti, R.; Storm, H.; Comber, H.; Steliarova-Foucher, E.; Coebergh, J.W.W.

    2015-01-01

    Aim To provide insight into cancer registration coverage, data access and use in Europe. This contributes to data and infrastructure harmonisation and will foster a more prominent role of cancer registries (CRs) within public health, clinical policy and cancer research, whether within or outside the

  9. Accrual to Cancer Clinical Trials

    LENUS (Irish Health Repository)

    Kelly, C

    2016-07-01

    Accrual to cancer clinical trials (CCT) is imperative to safeguard continued improvement in cancer outcomes. A retrospective chart review was performed of patients (n=140) starting a new anti-cancer agent in a north Dublin cancer centre. This review was performed over a four-month period, beginning in November 2015. Only 29% (n=41) had a CCT option. The overall accrual rate to CCT was 5% (n=7), which is comparable to internationally reported figures. The main reasons for failure to recruit to CCT included the lack of a CCT option for cancer type (n=30, 23%), stage (n=25, 19%), and line of treatment (n=23, 17%). Over the last decade, the rate of accrual to CCTs has in fact doubled and the number of trials open to recruitment has tripled. Ongoing governmental and philanthropic support is necessary to continue this trend to further expand CCT patient options with a target accrual rate of 10%.

  10. Where are clinical trials going? Society and clinical trials.

    Science.gov (United States)

    Sleight, P

    2004-02-01

    Clinical trials now increasingly impinge on society at large. First there is growing emphasis from health organizations on the need for unbiased evidence about the effectiveness of promoted remedies. Second, as most novel treatments accrue increased costs to society, these need to be evaluated in terms of value for money. Third, there has been confusion and concern about the resolution of conflicting evidence, especially the role of advertising and commercial pressures from a powerful pharmaceutical industry motivated by profit. Fourth, there is concern about research fraud and the ethics of clinical trials. Fifth, there is increasing suspicion of political advice, which sometimes has sought to reassure an anxious public on the basis of complex and possibly inadequate scientific information. Some of these issues are addressed by truly independent and properly constituted data and safety monitoring committees, which are of particular importance when academic investigators or universities have a large financial conflict of interest. This is now more problematic with the current encouragement of investigator-led spin-off companies. These issues are best resolved by independent financial support (from government or other institutions) rather than relying on the commercial sponsor.

  11. Credentialing for participation in clinical trials

    International Nuclear Information System (INIS)

    Followill, David S.; Urie, Marcia; Galvin, James M.; Ulin, Kenneth; Xiao, Ying; FitzGerald, Thomas J.

    2012-01-01

    The National Cancer Institute (NCI) clinical cooperative groups have been instrumental over the past 50 years in developing clinical trials and evidence-based clinical trial processes for improvements in patient care. The cooperative groups are undergoing a transformation process to launch, conduct, and publish clinical trials more rapidly. Institutional participation in clinical trials can be made more efficient and include the expansion of relationships with international partners. This paper reviews the current processes that are in use in radiation therapy trials and the importance of maintaining effective credentialing strategies to assure the quality of the outcomes of clinical trials. The paper offers strategies to streamline and harmonize credentialing tools and processes moving forward as the NCI undergoes transformative change in the conduct of clinical trials.

  12. Credentialing for participation in clinical trials

    Energy Technology Data Exchange (ETDEWEB)

    Followill, David S. [Radiological Physics Center, Department of Radiation Physics, University of Texas MD Anderson Cancer Center, Houston, TX (United States); Urie, Marcia [Quality Assurance Review Center, Department of Radiation Oncology, University of Massachusetts Medical School, Lincoln, RI (United States); Galvin, James M. [Department of Radiation Oncology, Jefferson Medical College, Thomas Jefferson University, Philadelphia, PA (United States); Radiation Therapy Oncology Group, Philadelphia, PA (United States); Ulin, Kenneth [Quality Assurance Review Center, Department of Radiation Oncology, University of Massachusetts Medical School, Lincoln, RI (United States); Department of Radiation Oncology, University of Massachusetts Medical School, Worcester, MA (United States); Xiao, Ying [Department of Radiation Oncology, Jefferson Medical College, Thomas Jefferson University, Philadelphia, PA (United States); Radiation Therapy Oncology Group, Philadelphia, PA (United States); FitzGerald, Thomas J., E-mail: dfollowi@mdanderson.org [Quality Assurance Review Center, Department of Radiation Oncology, University of Massachusetts Medical School, Lincoln, RI (United States); Department of Radiation Oncology, University of Massachusetts Medical School, Worcester, MA (United States)

    2012-12-26

    The National Cancer Institute (NCI) clinical cooperative groups have been instrumental over the past 50 years in developing clinical trials and evidence-based clinical trial processes for improvements in patient care. The cooperative groups are undergoing a transformation process to launch, conduct, and publish clinical trials more rapidly. Institutional participation in clinical trials can be made more efficient and include the expansion of relationships with international partners. This paper reviews the current processes that are in use in radiation therapy trials and the importance of maintaining effective credentialing strategies to assure the quality of the outcomes of clinical trials. The paper offers strategies to streamline and harmonize credentialing tools and processes moving forward as the NCI undergoes transformative change in the conduct of clinical trials.

  13. Effectiveness of an annular closure device in a "real-world" population: stratification of registry data using screening criteria from a randomized controlled trial.

    Science.gov (United States)

    Kuršumović, Adisa; Rath, Stefan A

    2018-01-01

    Increased focus has been put on the use of "'real-world" data to support randomized clinical trial (RCT) evidence for clinical decision-making. The objective of this study was to assess the performance of an annular closure device (ACD) after stratifying a consecutive series of "real-world" patients by the screening criteria of an ongoing RCT. This was a single-center registry analysis of 164 subjects who underwent limited discectomy combined with ACD for symptomatic lumbar disc herniation. Patients were stratified into two groups using the selection criteria of a pivotal RCT on the same device: Trial (met inclusion; n=44) or non-Trial (did not meet inclusion; n=120). Patient-reported outcomes, including Oswestry Disability Index (ODI) and visual analog scale (VAS) for leg and back pain, and adverse events were collected from baseline to last follow-up (mean: Trial - 15.6 months; non-Trial - 14.6 months). Statistical analyses were performed with significance set at p population. Stratification of this "real-world" series on the basis of RCT screening criteria did not result in significant between-group differences. These findings suggest that the efficacy of the ACD extends beyond the strictly defined patient population being studied in the RCT of this device. Furthermore, reducing the reherniation rate following lumbar discectomy has positive clinical and economic implications.

  14. Clinical trials and gender medicine.

    Science.gov (United States)

    Cassese, Mariarita; Zuber, Veronica

    2011-01-01

    Women use more medicines than men because they fall ill more often and suffer more from chronic diseases, but also because women pay more attention to their health and have more consciousness and care about themselves. Although medicines can have different effects on women and men, women still represent a small percentage in the first phases of trials (22%) which are essential to verify drugs dosage, side effects, and safety. Even though women are more present in trials, studies results are not presented with a gender approach. This situation is due to educational, social, ethical and economical factors. The scientific research must increase feminine presence in clinical trials in order to be equal and correct, and all the key stakeholder should be involved in this process. We still have a long way to cover and it doesn't concern only women but also children and old people. The aim is to have a medicine not only illness-focused but patient-focused: a medicine able to take into consideration all the patient characteristics and so to produce a really personalized therapy. What above described is part of the reasons why in 2005 was founded the National Observatory for Women's Health (Osservatorio Nazionale sulla Salute della Donna, ONDa) which promotes a gender health awareness and culture in Italy, at all the levels of the civil and scientific society.

  15. Clinical trials and gender medicine

    Directory of Open Access Journals (Sweden)

    Mariarita Cassese

    2011-01-01

    Full Text Available Women use more medicines than men because they fall ill more often and suffer more from chronic diseases, but also because women pay more attention to their health and have more consciousness and care about themselves. Although medicines can have different effects on women and men, women still represent a small percentage in the first phases of trials (22% which are essential to verify drugs dosage, side effects, and safety. Even though women are more present in trials, studies results are not presented with a gender approach. This situation is due to educational, social, ethical and economical factors. The scientific research must increase feminine presence in clinical trials in order to be equal and correct, and all the key stakeholder should be involved in this process. We still have a long way to cover and it doesn't concern only women but also children and old people. The aim is to have a medicine not only illness-focused but patient-focused: a medicine able to take into consideration all the patient characteristics and so to produce a really personalized therapy. What above described is part of the reasons why in 2005 was founded the National Observatory for Women's Health (Osservatorio Nazionale sulla Salute della Donna, ONDa which promotes a gender health awareness and culture in Italy, at all the levels of the civil and scientific society.

  16. Maximizing scientific knowledge from randomized clinical trials

    DEFF Research Database (Denmark)

    Gustafsson, Finn; Atar, Dan; Pitt, Bertram

    2010-01-01

    Trialists have an ethical and financial responsibility to plan and conduct clinical trials in a manner that will maximize the scientific knowledge gained from the trial. However, the amount of scientific information generated by randomized clinical trials in cardiovascular medicine is highly vari...

  17. Decision aids for people considering taking part in clinical trials.

    Science.gov (United States)

    Gillies, Katie; Cotton, Seonaidh C; Brehaut, Jamie C; Politi, Mary C; Skea, Zoe

    2015-11-27

    Several interventions have been developed to promote informed consent for participants in clinical trials. However, many of these interventions focus on the content and structure of information (e.g. enhanced information or changes to the presentation format) rather than the process of decision making. Patient decision aids support a decision making process about medical options. Decision aids support the decision process by providing information about available options and their associated outcomes, alongside information that enables patients to consider what value they place on particular outcomes, and provide structured guidance on steps of decision making. They have been shown to be effective for treatment and screening decisions but evidence on their effectiveness in the context of informed consent for clinical trials has not been synthesised. To assess the effectiveness of decision aids for clinical trial informed consent compared to no intervention, standard information (i.e. usual practice) or an alternative intervention on the decision making process. We searched the following databases and to March 2015: Cochrane Central Register of Controlled Trials (CENTRAL), The Cochrane Library; MEDLINE (OvidSP) (from 1950); EMBASE (OvidSP) (from 1980); PsycINFO (OvidSP) (from 1806); ASSIA (ProQuest) (from 1987); WHO International Clinical Trials Registry Platform (ICTRP) (http://apps.who.int/trialsearch/); ClinicalTrials.gov; ISRCTN Register (http://www.controlled-trials.com/isrctn/). We also searched reference lists of included studies and relevant reviews. We contacted study authors and other experts. There were no language restrictions. We included randomised and quasi-randomised controlled trials comparing decision aids in the informed consent process for clinical trials alone, or in conjunction with standard information (such as written or verbal) or alongside alternative interventions (e.g. paper-based versus web-based decision aids). Included trials involved

  18. NIH Clinical Research Trials and You

    Science.gov (United States)

    ... Info Lines Health Services Locator HealthCare.gov NIH Clinical Research Trials and You Talking to Your Doctor Science ... Labs & Clinics Training Opportunities Library Resources Research Resources Clinical Research Resources Safety, Regulation and Guidance More » Quick Links ...

  19. Readmissions after stroke: linked data from the Australian Stroke Clinical Registry and hospital databases.

    Science.gov (United States)

    Kilkenny, Monique F; Dewey, Helen M; Sundararajan, Vijaya; Andrew, Nadine E; Lannin, Natasha; Anderson, Craig S; Donnan, Geoffrey A; Cadilhac, Dominique A

    2015-07-20

    To assess the feasibility of linking a national clinical stroke registry with hospital admissions and emergency department data; and to determine factors associated with hospital readmission after stroke or transient ischaemic attack (TIA) in Australia. Data from the Australian Stroke Clinical Registry (AuSCR) at a single Victorian hospital were linked to coded, routinely collected hospital datasets for admissions (Victorian Admitted Episodes Dataset) and emergency presentations (Victorian Emergency Minimum Dataset) in Victoria from 15 June 2009 to 31 December 2010, using stepwise deterministic data linkage techniques. Association of patient characteristics, social circumstances, processes of care and discharge outcomes with all-cause readmissions within 1 year from time of hospital discharge after an index admission for stroke or TIA. Of 788 patients registered in the AuSCR, 46% (359/781) were female, 83% (658/788) had a stroke, and the median age was 76 years. Data were successfully linked for 782 of these patients (99%). Within 1 year of their index stroke or TIA event, 42% of patients (291/685) were readmitted, with 12% (35/286) readmitted due to a stroke or TIA. Factors significantly associated with 1-year hospital readmission were two or more presentations to an emergency department before the index event (adjusted odds ratio [aOR], 1.57; 95% CI, 1.02-2.43), higher Charlson comorbidity index score (aOR, 1.19; 95% CI, 1.07-1.32) and diagnosis of TIA on the index admission (aOR, 2.15; 95% CI, 1.30-3.56). Linking clinical registry data with routinely collected hospital data for stroke and TIA is feasible in Victoria. Using these linked data, we found that readmission to hospital is common in this patient group and is related to their comorbid conditions.

  20. FORWARD: A Registry and Longitudinal Clinical Database to Study Fragile X Syndrome.

    Science.gov (United States)

    Sherman, Stephanie L; Kidd, Sharon A; Riley, Catharine; Berry-Kravis, Elizabeth; Andrews, Howard F; Miller, Robert M; Lincoln, Sharyn; Swanson, Mark; Kaufmann, Walter E; Brown, W Ted

    2017-06-01

    Advances in the care of patients with fragile X syndrome (FXS) have been hampered by lack of data. This deficiency has produced fragmentary knowledge regarding the natural history of this condition, healthcare needs, and the effects of the disease on caregivers. To remedy this deficiency, the Fragile X Clinic and Research Consortium was established to facilitate research. Through a collective effort, the Fragile X Clinic and Research Consortium developed the Fragile X Online Registry With Accessible Research Database (FORWARD) to facilitate multisite data collection. This report describes FORWARD and the way it can be used to improve health and quality of life of FXS patients and their relatives and caregivers. FORWARD collects demographic information on individuals with FXS and their family members (affected and unaffected) through a 1-time registry form. The longitudinal database collects clinician- and parent-reported data on individuals diagnosed with FXS, focused on those who are 0 to 24 years of age, although individuals of any age can participate. The registry includes >2300 registrants (data collected September 7, 2009 to August 31, 2014). The longitudinal database includes data on 713 individuals diagnosed with FXS (data collected September 7, 2012 to August 31, 2014). Longitudinal data continue to be collected on enrolled patients along with baseline data on new patients. FORWARD represents the largest resource of clinical and demographic data for the FXS population in the United States. These data can be used to advance our understanding of FXS: the impact of cooccurring conditions, the impact on the day-to-day lives of individuals living with FXS and their families, and short-term and long-term outcomes. Copyright © 2017 by the American Academy of Pediatrics.

  1. A clinical registry of dementia based on the principle of epidemiological surveillance

    Directory of Open Access Journals (Sweden)

    Turbau Josefina

    2009-01-01

    Full Text Available Abstract Background Traditional epidemiological studies do not allow elucidating the reality of referral and diagnosis patterns of dementia in routine clinical practice within a defined territory. This information is useful and necessary in order to plan and allocate healthcare resources. This paper presents the results from a dementia case registry based on epidemiological surveillance fundamentals. Methods Standardised registry of dementia diagnoses made in 2007 by specialised care centres in the Health Region of Girona (RSG (Spain, which encompasses an area of 5,517 sq. km and a reference population of 690,207 inhabitants. Results 577 cases of dementia were registered, of which 60.7% corresponded to cases of Alzheimer's disease. Presenile dementia accounted for 9.3% of the cases. Mean time between the onset of symptoms and clinical diagnosis was 2.4 years and the severity of the dementia was mild in 60.7% of the cases. High blood pressure, a family history of dementia, dislipidemia, and a past history of depression were the most common conditions prior to the onset of the disease (>20%. Conclusion The ReDeGi is a viable epidemiological surveillance device that provides information about the clinical and demographic characteristics of patients diagnosed with dementia in a defined geographical area.

  2. A clinical registry of dementia based on the principle of epidemiological surveillance

    Science.gov (United States)

    Garre-Olmo, Josep; Flaqué, Margarita; Gich, Jordi; Pulido, Teresa Osuna; Turbau, Josefina; Vallmajo, Natalia; Viñas, Marta; López-Pousa, Secundí

    2009-01-01

    Background Traditional epidemiological studies do not allow elucidating the reality of referral and diagnosis patterns of dementia in routine clinical practice within a defined territory. This information is useful and necessary in order to plan and allocate healthcare resources. This paper presents the results from a dementia case registry based on epidemiological surveillance fundamentals. Methods Standardised registry of dementia diagnoses made in 2007 by specialised care centres in the Health Region of Girona (RSG) (Spain), which encompasses an area of 5,517 sq. km and a reference population of 690,207 inhabitants. Results 577 cases of dementia were registered, of which 60.7% corresponded to cases of Alzheimer's disease. Presenile dementia accounted for 9.3% of the cases. Mean time between the onset of symptoms and clinical diagnosis was 2.4 years and the severity of the dementia was mild in 60.7% of the cases. High blood pressure, a family history of dementia, dislipidemia, and a past history of depression were the most common conditions prior to the onset of the disease (>20%). Conclusion The ReDeGi is a viable epidemiological surveillance device that provides information about the clinical and demographic characteristics of patients diagnosed with dementia in a defined geographical area. PMID:19175921

  3. Risk of discontinuation of Advanced Therapy Medicinal Products clinical trials.

    Science.gov (United States)

    Hanna, Eve; Rémuzat, Cecile; Auquier, Pascal; Toumi, Mondher

    2016-01-01

    Advanced therapy medicinal products (ATMPs) constitute a class of innovative products that encompasses gene therapy, somatic cell therapy, and tissue-engineered products (TEP). There is an increased investment of commercial and non-commercial sponsors in this field and a growing number of ATMPs randomized clinical trials (RCT) and patients enrolled in such trials. RCT generate data to prove the efficacy of a new therapy, but the discontinuation of RCTs wastes scarce resources. Our objective is to identify the number and characteristics of discontinued ATMPs trials in order to evaluate the rate of discontinuation. We searched for ATMPs trials conducted between 1999 to June 2015 using three databases, which are Clinicaltrials.gov, the International Clinical Trials Registry Platform (ICTRP), and the EU Drug Regulating Authorities Clinical Trials (EudraCT). We selected the ATMPs trials after elimination of the duplicates. We identified the disease areas and the sponsors as commercial or non-commercial organizations. We classified ATMPs by type and trial status, that is, ongoing, completed, terminated, discontinued, and prematurely ended. Then, we calculated the rate of discontinuation. Between 1999 and June 2015, 143 withdrawn, terminated, or prematurely ended ATMPs clinical trials were identified. Between 1999 and June 2013, 474 ongoing and completed clinical trials were identified. Therefore, the rate of discontinuation of ATMPs trials is 23.18%, similar to that for non-ATMPs drugs in development. The probability of discontinuation is, respectively, 27.35, 16.28, and 16.34% for cell therapies, gene therapies, and TEP. The highest discontinuation rate is for oncology (43%), followed by cardiology (19.2%). It is almost the same for commercial and non-commercial sponsors; therefore, the discontinuation reason may not be financially driven. No failure risk rate per development phase is available for ATMPs. The discontinuation rate may prove helpful when assessing the

  4. Validation of the 'United Registries for Clinical Assessment and Research' [UR-CARE], a European Online Registry for Clinical Care and Research in Inflammatory Bowel Disease.

    Science.gov (United States)

    Burisch, Johan; Gisbert, Javier P; Siegmund, Britta; Bettenworth, Dominik; Thomsen, Sandra Bohn; Cleynen, Isabelle; Cremer, Anneline; Ding, Nik John Sheng; Furfaro, Federica; Galanopoulos, Michail; Grunert, Philip Christian; Hanzel, Jurij; Ivanovski, Tamara Knezevic; Krustins, Eduards; Noor, Nurulamin; O'Morain, Neil; Rodríguez-Lago, Iago; Scharl, Michael; Tua, Julia; Uzzan, Mathieu; Ali Yassin, Nuha; Baert, Filip; Langholz, Ebbe

    2018-04-27

    The 'United Registries for Clinical Assessment and Research' [UR-CARE] database is an initiative of the European Crohn's and Colitis Organisation [ECCO] to facilitate daily patient care and research studies in inflammatory bowel disease [IBD]. Herein, we sought to validate the database by using fictional case histories of patients with IBD that were to be entered by observers of varying experience in IBD. Nineteen observers entered five patient case histories into the database. After 6 weeks, all observers entered the same case histories again. For each case history, 20 key variables were selected to calculate the accuracy for each observer. We assumed that the database was such that ≥ 90% of the entered data would be correct. The overall proportion of correctly entered data was calculated using a beta-binomial regression model to account for inter-observer variation and compared to the expected level of validity. Re-test reliability was assessed using McNemar's test. For all case histories, the overall proportion of correctly entered items and their confidence intervals included the target of 90% (Case 1: 92% [88-94%]; Case 2: 87% [83-91%]; Case 3: 93% [90-95%]; Case 4: 97% [94-99%]; Case 5: 91% [87-93%]). These numbers did not differ significantly from those found 6 weeks later [NcNemar's test p > 0.05]. The UR-CARE database appears to be feasible, valid and reliable as a tool and easy to use regardless of prior user experience and level of clinical IBD experience. UR-CARE has the potential to enhance future European collaborations regarding clinical research in IBD.

  5. Ambulatory blood pressure monitoring in daily clinical practice - the Spanish ABPM Registry experience.

    Science.gov (United States)

    Gorostidi, Manuel; Banegas, José R; de la Sierra, Alejandro; Vinyoles, Ernest; Segura, Julián; Ruilope, Luis M

    2016-01-01

    Many patients are hypertensive at the medical settings but show normal blood pressure out of the doctor's office, and are classified as white-coat hypertensives. On the other hand, many patients with controlled hypertension at the clinic show ambulatory blood pressure levels above the thresholds considered for an adequate blood pressure control, known as having masked hypertension. Using data from the Spanish Ambulatory Blood Pressure Monitoring Registry (Spanish ABPM Registry), a national program developed to promote the use of the ambulatory technique for hypertension management in daily practice, we have reviewed the main strengths of this approach, that is the ability to detect discrepancies of blood pressure status with respect to office blood pressure measurement, and to better assess accurate rates of hypertension control. White-coat hypertension within patients with elevated office blood pressure, and masked hypertension within office-controlled patients affected one of three patients in each office status. On the other hand, rates of ambulatory blood pressure control (50%) doubled those of office blood pressure control (25%), still remaining half the patients uncontrolled. We think that a systematic use of ambulatory blood pressure monitoring, and strategies to improve blood pressure control constitute key priorities in hypertension management. © 2015 Stichting European Society for Clinical Investigation Journal Foundation.

  6. The Swedish MS registryclinical support tool and scientific resource.

    Science.gov (United States)

    Hillert, J; Stawiarz, L

    2015-01-01

    The Swedish MS registry (SMSreg) is designed to assure quality health care for patients with multiple sclerosis (MS). It has been active since 2001 and web-based since 2004. It runs on government funding only and is used in all Swedish neurology departments. The SMSreg currently includes data on 14,500 of Sweden's estimated 17,500 prevalent patients with MS. One important function of SMSreg, to which participation is voluntary, is to serve as a tool for decision support and to provide an easy overview of the patient information needed at clinical visits. This is its core feature and explains why the majority of Swedish MS specialists contribute data. Another success factor for SMSreg is that entered data can be readily accessed, either through a query function into Excel format or through a set of predesigned tables and diagrams in which parameters can be selected. Recent development includes a portal allowing patients to view a summary of their registered data and to report a set of patient-reported outcomes. SMSreg data have been used in close to 100 published scientific reports. Current projects include an incidence cohort (EIMS), post-marketing cohorts of patients on novel disease-modifying drugs (IMSE), and a prevalence cohort (GEMS). As these studies combine physical sampling and questionnaire data with clinical documentation and possible linkage to other public registries, together they provide an excellent platform for integrated MS research. © 2015 John Wiley & Sons A/S. Published by John Wiley & Sons Ltd.

  7. Opioid detoxification : from controlled clinical trial to clinical practice

    NARCIS (Netherlands)

    Dijkstra, Boukje A G; De Jong, Cor A J; Wensing, Michel; Krabbe, Paul F M; van der Staak, Cees P F

    2010-01-01

    Controlled clinical trials have high internal validity but suffer from difficulties in external validity. This study evaluates the generalizability of the results of a controlled clinical trial on rapid detoxification in the everyday clinical practice of two addiction treatment centers. The results

  8. Spine device clinical trials: design and sponsorship.

    Science.gov (United States)

    Cher, Daniel J; Capobianco, Robyn A

    2015-05-01

    Multicenter prospective randomized clinical trials represent the best evidence to support the safety and effectiveness of medical devices. Industry sponsorship of multicenter clinical trials is purported to lead to bias. To determine what proportion of spine device-related trials are industry-sponsored and the effect of industry sponsorship on trial design. Analysis of data from a publicly available clinical trials database. Clinical trials of spine devices registered on ClinicalTrials.gov, a publicly accessible trial database, were evaluated in terms of design, number and location of study centers, and sample size. The relationship between trial design characteristics and study sponsorship was evaluated using logistic regression and general linear models. One thousand six hundred thrity-eight studies were retrieved from ClinicalTrials.gov using the search term "spine." Of the 367 trials that focused on spine surgery, 200 (54.5%) specifically studied devices for spine surgery and 167 (45.5%) focused on other issues related to spine surgery. Compared with nondevice trials, device trials were far more likely to be sponsored by the industry (74% vs. 22.2%, odds ratio (OR) 9.9 [95% confidence interval 6.1-16.3]). Industry-sponsored device trials were more likely multicenter (80% vs. 29%, OR 9.8 [4.8-21.1]) and had approximately four times as many participating study centers (pdevices not sponsored by the industry. Most device-related spine research is industry-sponsored. Multicenter trials are more likely to be industry-sponsored. These findings suggest that previously published studies showing larger effect sizes in industry-sponsored vs. nonindustry-sponsored studies may be biased as a result of failure to take into account the marked differences in design and purpose. Copyright © 2015 Elsevier Inc. All rights reserved.

  9. X-linked hypohidrotic ectodermal dysplasia (XLHED): clinical and diagnostic insights from an international patient registry.

    Science.gov (United States)

    Fete, Mary; Hermann, Julie; Behrens, Jeffrey; Huttner, Kenneth M

    2014-10-01

    The web-based Ectodermal Dysplasia International Registry (EDIR) is a comprehensive patient-reported survey contributing to an understanding of ectodermal dysplasia (ED). XLHED is the most common of the genetic ED syndromes and was the primary diagnosis reported by 223/835 respondents (141 males and 82 females). Overall, 96% of XLHED registrants reported as least one other affected family member and 21% reported a family history of infant or childhood deaths, consistent with the published mortality data in this disorder. In general, XLHED is diagnosed by the triad of decreased sweating, reduced hair, and hypodontia (present in 89%, 74%, and 74% of XLHED respondents). Additionally, the registry dataset confirmed a spectrum of life-long XLHED clinical complications including recurrent sinus infections (49% males, 52% females), nasal congestion often foul smelling and interfering with feeding (73% males, 27% females), eczema (66% males, 40% females), wheezing (66% males, 45% females), and a hoarse, raspy voice (67% males, 23% females). The Registry results also highlighted features consistently differentiating XLHED from the non-hypohidrotic ED syndromes including the frequency of infant/childhood deaths, the presence of limb/digit abnormalities, feeding issues related to nasal discharge, dentures, and a diagnosis of asthma. These results represent the largest collection of data on a broad-spectrum of health-related issues affecting ED patients. This project provides information for expanding knowledge of the natural history of XLHED, and as such may facilitate the diagnosis and treatment of its varied and lifelong medical challenges. © 2014 Wiley Periodicals, Inc.

  10. Ethics of clinical trials in Nigeria.

    Science.gov (United States)

    Okonta, Patrick I

    2014-05-01

    The conduct of clinical trials for the development and licensing of drugs is a very important aspect of healthcare. Drug research, development and promotion have grown to a multi-billion dollar global business. Like all areas of human endeavour involving generation and control of huge financial resources, it could be subject to deviant behaviour, sharp business practices and unethical practices. The main objective of this review is to highlight potential ethical challenges in the conduct of clinical trials in Nigeria and outline ways in which these can be avoided. Current international and national regulatory and ethical guidelines are reviewed to illustrate the requirements for ethical conduct of clinical trials. Past experiences of unethical conduct of clinical trials especially in developing countries along with the increasing globalisation of research makes it imperative that all players should be aware of the ethical challenges in clinical trials and the benchmarks for ethical conduct of clinical research in Nigeria.

  11. Inherited Retinal Degenerative Clinical Trial Network

    Science.gov (United States)

    2009-10-01

    clinical efforts that will impact the NEER network going forward and laid the ground work for the CTECs to participate in ongoing clinical trials for...Clinical Implications: • How will the proposed clinical trial have a significant impact on disease outcome? 34 • How will the clinical trial offer...was 0 041U>< for pat<t!nts NPtS and <H08, 0 4 1ux !01 Ct 110, 1nd 10.0 lux f01 < H13 OJ)Ilo •her on~tion are indiuttd AhtrNtor19 stimuli Wl’f1! pres

  12. The database for aggregate analysis of ClinicalTrials.gov (AACT and subsequent regrouping by clinical specialty.

    Directory of Open Access Journals (Sweden)

    Asba Tasneem

    Full Text Available BACKGROUND: The ClinicalTrials.gov registry provides information regarding characteristics of past, current, and planned clinical studies to patients, clinicians, and researchers; in addition, registry data are available for bulk download. However, issues related to data structure, nomenclature, and changes in data collection over time present challenges to the aggregate analysis and interpretation of these data in general and to the analysis of trials according to clinical specialty in particular. Improving usability of these data could enhance the utility of ClinicalTrials.gov as a research resource. METHODS/PRINCIPAL RESULTS: The purpose of our project was twofold. First, we sought to extend the usability of ClinicalTrials.gov for research purposes by developing a database for aggregate analysis of ClinicalTrials.gov (AACT that contains data from the 96,346 clinical trials registered as of September 27, 2010. Second, we developed and validated a methodology for annotating studies by clinical specialty, using a custom taxonomy employing Medical Subject Heading (MeSH terms applied by an NLM algorithm, as well as MeSH terms and other disease condition terms provided by study sponsors. Clinical specialists reviewed and annotated MeSH and non-MeSH disease condition terms, and an algorithm was created to classify studies into clinical specialties based on both MeSH and non-MeSH annotations. False positives and false negatives were evaluated by comparing algorithmic classification with manual classification for three specialties. CONCLUSIONS/SIGNIFICANCE: The resulting AACT database features study design attributes parsed into discrete fields, integrated metadata, and an integrated MeSH thesaurus, and is available for download as Oracle extracts (.dmp file and text format. This publicly-accessible dataset will facilitate analysis of studies and permit detailed characterization and analysis of the U.S. clinical trials enterprise as a whole. In

  13. Evaluation of the predictive value of a clinical worsening definition using 2-year outcomes in patients with pulmonary arterial hypertension: a REVEAL Registry analysis.

    Science.gov (United States)

    Frost, Adaani E; Badesch, David B; Miller, Dave P; Benza, Raymond L; Meltzer, Leslie A; McGoon, Michael D

    2013-11-01

    Time to clinical worsening has been proposed as a primary end point in clinical trials of pulmonary arterial hypertension (PAH); however, neither standardized nor validated definitions of clinical worsening across PAH trials exist. This study aims to evaluate a proposed definition of clinical worsening within a large prospective, observational registry of patients with PAH with respect to its value as a predictor of proximate (within 1 year) risk for subsequent major events (ie, death, transplantation, or atrial septostomy). We assessed overall 2-year survival and survival free from major events to determine the relationship between clinical worsening and major events among adults with hemodynamically defined PAH (N = 3,001). Freedom from clinical worsening was defined as freedom from worsening functional class (FC), a ≥ 15% reduction in 6-min walk distance (6MWD), all-cause hospitalization, or the introduction of parenteral prostacyclin analog therapy. In the 2 years of follow-up, 583 patients died. Four hundred twenty-six died after a documented clinical worsening event, including FC worsening (n = 128), a ≥ 15% reduction in 6MWD (n = 118), all-cause hospitalization (n = 370), or introduction of a prostacyclin analog (n = 91). Patients who experienced clinical worsening had significantly poorer subsequent 1-year survival postworsening than patients who did not worsen (P < .001). Clinical worsening was highly predictive of subsequent proximate mortality in this analysis from an observational study. These results validate the use of clinical worsening as a meaningful prognostic tool in clinical practice and as a primary end point in clinical trial design. ClinicalTrials.gov; No.: NCT00370214; URL: www.clinicaltrials.gov.

  14. Understanding the patient perspective on research access to national health records databases for conduct of randomized registry trials.

    Science.gov (United States)

    Avram, Robert; Marquis-Gravel, Guillaume; Simard, François; Pacheco, Christine; Couture, Étienne; Tremblay-Gravel, Maxime; Desplantie, Olivier; Malhamé, Isabelle; Bibas, Lior; Mansour, Samer; Parent, Marie-Claude; Farand, Paul; Harvey, Luc; Lessard, Marie-Gabrielle; Ly, Hung; Liu, Geoffrey; Hay, Annette E; Marc Jolicoeur, E

    2018-07-01

    Use of health administrative databases is proposed for screening and monitoring of participants in randomized registry trials. However, access to these databases raises privacy concerns. We assessed patient's preferences regarding use of personal information to link their research records with national health databases, as part of a hypothetical randomized registry trial. Cardiology patients were invited to complete an anonymous self-reported survey that ascertained preferences related to the concept of accessing government health databases for research, the type of personal identifiers to be shared and the type of follow-up preferred as participants in a hypothetical trial. A total of 590 responders completed the survey (90% response rate), the majority of which were Caucasians (90.4%), male (70.0%) with a median age of 65years (interquartile range, 8). The majority responders (80.3%) would grant researchers access to health administrative databases for screening and follow-up. To this end, responders endorsed the recording of their personal identifiers by researchers for future record linkage, including their name (90%), and health insurance number (83.9%), but fewer responders agreed with the recording of their social security number (61.4%, pgranting researchers access to the administrative databases (OR: 1.69, 95% confidence interval: 1.03-2.90; p=0.04). The majority of Cardiology patients surveyed were supportive of use of their personal identifiers to access administrative health databases and conduct long-term monitoring in the context of a randomized registry trial. Copyright © 2018 Elsevier Ireland Ltd. All rights reserved.

  15. Clinical Trials Management | Division of Cancer Prevention

    Science.gov (United States)

    Information for researchers about developing, reporting, and managing NCI-funded cancer prevention clinical trials. Protocol Information Office The central clearinghouse for clinical trials management within the Division of Cancer Prevention.Read more about the Protocol Information Office. | Information for researchers about developing, reporting, and managing NCI-funded

  16. Inherited Retinal Degenerative Clinical Trial Network. Addendum

    Science.gov (United States)

    2013-10-01

    inherited orphan retinal degenerative diseases and dry age-related macular degeneration (AMD) through the conduct of clinical trials and other...design and conduct of effective and efficient clinical trials for inherited orphan retinal degenerative diseases and dry AMD; • Limited number and...linica l trial in the NEER network for autosomal dominant retinitis pigmentosa, and the ProgSTAR studies for Stargardt disease ) . As new interventions b

  17. Evolution of the clinical trial landscape in Asia Pacific

    Directory of Open Access Journals (Sweden)

    Yathindranath S

    2014-07-01

    Full Text Available Shourav Yathindranath,1 Amar Kureishi,2 Simranjit Singh,3 Spencer Yeow,3 Grace Geng,4 Karen Wai,1 Audrey Ho,1 Elvira Zenaida Lansang,1 Ken J Lee5 1Feasibility and Site Identification Asia, 2Drug Development Asia, 3Strategic Planning Asia, Quintiles East Asia Private Limited, Singapore; 4People’s Republic of China Site Services, Quintiles, Beijing, People’s Republic of China; 5Asia Site Services, Quintiles East Asia Private Limited, Singapore Introduction: Asia Pacific has and continues to be one of the fastest-growing pharmaceutical markets in the world. This growth has a carry-over effect of driving pharmaceutical research and development investment in the region. Coupled with this, there have been multiple initiatives conducted by governments and other research focused organizations and societies in the region to help support this growth in research. In this report, we discuss the latest developments in pharmaceutical research and development in Asia Pacific and how these various initiatives have made an impact. Methods: An extensive search of the major clinical trial registries, along with the literature and Internet review of the recent developments in clinical trials, was performed comparing two time periods – 2009–2010 and 2011–2012. Results: In overall numbers, the clinical trial industry in Asia Pacific has remained stable when comparing the two time periods, with stable volumes of clinical trial numbers and site numbers. However, on closer inspection, a dynamic change in geography, nature, and therapeutic areas of the trials being conducted is observed. Japan, South Korea, People’s Republic of China, and Taiwan continue to be major clinical trial destinations. Developing countries, such as Indonesia, Vietnam, and Philippines, have seen rising standards of living and medical care; this is starting to impact their contribution to trials. Also, there are an increasing number of local trials in Asia Pacific with a bigger role

  18. [Concordance in the registry of dementia among the main sources of clinical information].

    Science.gov (United States)

    Marta-Moreno, Javier; Obón-Azuara, Blanca; Gimeno-Felíu, Luis; Achkar-Tuglaman, Nesib Nicolás; Poblador-Plou, Beatriz; Calderón-Larrañaga, Amaia; Prados-Torres, Alexandra

    2016-01-01

    The objective of this work was to analyse the concordance in the registry of dementia among the main sources of clinical information, with the aim of determining their usefulness for epidemiological and clinical research. Descriptive study of patients assigned to the Aragon Health Service in 2010 (n=1,344,891). (i)the pharmacy billing database (n=9,392); (ii)Primary Care electronic health records (EHR) (n=9,471), and (iii)the hospital minimum basic data set (n=3,289). When studying the concordance of the databases, the group of patients with a specific treatment for dementia (i.e., acetylcholinesterase inhibitors and/or memantine) was taken as the reference. The diagnosis in Primary Care was missing for 47.3% of patients taking anti-dementia drugs. The same occurred with 38.3% of dementia patients admitted to hospital during the study year. Among patients with a diagnosis of dementia in the EHR, only half (52.3%) was under treatment for this condition. This percentage decreased to 34.4% in patients with the diagnosis registered in the hospital database. The weak concordance in the registry of the dementia diagnosis between the main health information systems makes their use and analysis more complex, and supports the need to include all available health data sources in order to gain a global picture of the epidemiological and clinical reality of this health condition. Copyright © 2015 SEGG. Publicado por Elsevier España, S.L.U. All rights reserved.

  19. Measuring clinical trial transparency: an empirical analysis of newly approved drugs and large pharmaceutical companies.

    Science.gov (United States)

    Miller, Jennifer E; Wilenzick, Marc; Ritcey, Nolan; Ross, Joseph S; Mello, Michelle M

    2017-12-05

    To define a series of clinical trial transparency measures and apply them to large pharmaceutical and biotechnology companies and their 2014 FDA-approved drugs. Cross-sectional descriptive analysis of all clinical trials supporting 2014 Food and Drugs Administration (FDA)-approved new drug applications (NDAs) for novel drugs sponsored by large companies. Data from over 45 sources, including Drugs@FDA.gov, ClinicalTrials.gov, corporate and international registries; PubMed, Google Scholar, EMBASE, corporate press releases, Securities and Exchange Commission (SEC) filings and personal communications with drug manufacturers. Trial registration, results reporting, clinical study report (CSR) synopsis sharing, biomedical journal publication, and FDA Amendments Acts (FDAAA) compliance, analysed on the drug level. The FDA approved 19 novel new drugs, sponsored by 11 large companies, involving 553 trials, in 2014. We analysed 505 relevant trials. Per drug, a median of 100% (IQR 86%-100%) of trials in patients were registered, 71% (IQR 57%-100%) reported results or shared a CSR synopsis, 80% (70%-100%) were published and 96% (80%-100%) were publicly available in some form by 13 months after FDA approval. Disclosure rates were lower at FDA approval (65%) and improved significantly by 6 months post FDA approval. Per drug, a median of 100% (IQR 75%-100%) of FDAAA-applicable trials were compliant. Half of reviewed drugs had publicly disclosed results for all trials in patients in our sample. One trial was uniquely registered in a corporate registry, and not ClinicalTrials.gov; 0 trials were uniquely registered in international registries. Among large pharmaceutical companies and new drugs, clinical trial transparency is high based on several standards, although opportunities for improvement remain. Transparency is markedly higher for trials in patients than among all trials supporting drug approval, including trials in healthy volunteers. Ongoing efforts to publicly track

  20. Construction of ethics in clinical research: clinical trials registration

    OpenAIRE

    C. A. Caramori

    2007-01-01

    Scientific development that has been achieved through decades finds in clinical research a great possibility of translating findings to human health application. Evidence given by clinical trials allows everyone to have access to the best health services. However, the millionaire world of pharmaceutical industries has stained clinical research with doubt and improbability. Study results (fruits of controlled clinical trials) and scientific publications (selective, manipulated and with wrong c...

  1. Long-term results from the Contura multilumen balloon breast brachytherapy catheter phase 4 registry trial.

    Science.gov (United States)

    Cuttino, Laurie W; Arthur, Douglas W; Vicini, Frank; Todor, Dorin; Julian, Thomas; Mukhopadhyay, Nitai

    2014-12-01

    To describe the long-term outcomes from a completed, multi-institutional phase 4 registry trial using the Contura multilumen balloon (CMLB) breast brachytherapy catheter to deliver accelerated partial breast irradiation (APBI) in patients with early-stage breast cancer. Three hundred forty-two evaluable patients were enrolled by 23 institutions between January 2008 and February 2011. All patients received 34 Gy in 10 fractions, delivered twice daily. Rigorous target coverage and normal tissue dose constraints were observed. The median follow-up time was 36 months (range, 1-54 months). For the entire patient cohort of 342 patients, 10 patients experienced an ipsilateral breast tumor recurrence (IBTR). Eight of these IBTR were classified as true recurrences/marginal miss (TRMM), and 2 were elsewhere failures (EF). Local recurrence-free survival was 97.8% at 3 years. For the entire cohort, 88% of patients had good to excellent overall cosmesis. The overall incidence of infection was 8.5%. Symptomatic seroma was reported in only 4.4% of patients. A separate analysis was performed to determine whether improved outcomes would be observed for patients treated at high-volume centers with extensive brachytherapy experience. Three IBTR were observed in this cohort, only 1 of which was classified as a TRMM. Local recurrence-free survival at high-volume centers was 98.1% at 3 years. Overall cosmetic outcome and toxicity were superior in patients treated at high-volume centers. In these patients, 95% had good to excellent overall cosmesis. Infection was observed in only 2.9% of patients, and symptomatic seroma was reported in only 1.9%. Use of the CMLB for APBI delivery is associated with acceptable long-term local control and toxicity. Local recurrence-free survival was 97.8% at 3 years. Significant (grade 3) toxicity was uncommon, and no grade 4 toxicity was observed. Treatment at high-volume centers was associated with decreased late toxicity. Copyright © 2014 Elsevier Inc

  2. Long-Term Results From the Contura Multilumen Balloon Breast Brachytherapy Catheter Phase 4 Registry Trial

    Energy Technology Data Exchange (ETDEWEB)

    Cuttino, Laurie W., E-mail: lcuttino@mcvh-vcu.edu [Department of Radiation Oncology, Virginia Commonwealth University, Richmond, Virginia (United States); Arthur, Douglas W. [Department of Radiation Oncology, Virginia Commonwealth University, Richmond, Virginia (United States); Vicini, Frank [Michigan Healthcare Professionals/21st Century Onoclogy, Farmington Hills, Michigan (United States); Todor, Dorin [Department of Radiation Oncology, Virginia Commonwealth University, Richmond, Virginia (United States); Julian, Thomas [Allegheny Hospital, Temple School of Medicine, Pittsburgh, Pennsylvania (United States); Mukhopadhyay, Nitai [Department of Radiation Oncology, Virginia Commonwealth University, Richmond, Virginia (United States)

    2014-12-01

    Purpose: To describe the long-term outcomes from a completed, multi-institutional phase 4 registry trial using the Contura multilumen balloon (CMLB) breast brachytherapy catheter to deliver accelerated partial breast irradiation (APBI) in patients with early-stage breast cancer. Methods and Materials: Three hundred forty-two evaluable patients were enrolled by 23 institutions between January 2008 and February 2011. All patients received 34 Gy in 10 fractions, delivered twice daily. Rigorous target coverage and normal tissue dose constraints were observed. Results: The median follow-up time was 36 months (range, 1-54 months). For the entire patient cohort of 342 patients, 10 patients experienced an ipsilateral breast tumor recurrence (IBTR). Eight of these IBTR were classified as true recurrences/marginal miss (TRMM), and 2 were elsewhere failures (EF). Local recurrence-free survival was 97.8% at 3 years. For the entire cohort, 88% of patients had good to excellent overall cosmesis. The overall incidence of infection was 8.5%. Symptomatic seroma was reported in only 4.4% of patients. A separate analysis was performed to determine whether improved outcomes would be observed for patients treated at high-volume centers with extensive brachytherapy experience. Three IBTR were observed in this cohort, only 1 of which was classified as a TRMM. Local recurrence-free survival at high-volume centers was 98.1% at 3 years. Overall cosmetic outcome and toxicity were superior in patients treated at high-volume centers. In these patients, 95% had good to excellent overall cosmesis. Infection was observed in only 2.9% of patients, and symptomatic seroma was reported in only 1.9%. Conclusion: Use of the CMLB for APBI delivery is associated with acceptable long-term local control and toxicity. Local recurrence-free survival was 97.8% at 3 years. Significant (grade 3) toxicity was uncommon, and no grade 4 toxicity was observed. Treatment at high-volume centers was associated

  3. Characteristics of the first 1000 headaches in an outpatient headache clinic registry.

    Science.gov (United States)

    Guerrero, Ángel L; Rojo, Esther; Herrero, Sonia; Neri, María J; Bautista, Lourdes; Peñas, María L; Cortijo, Elisa; Mulero, Patricia; Fernández, Rosa

    2011-02-01

    To analyze the incidence and characteristics of the first 1000 headaches in an outpatient clinic. Headache is a common cause of medical consultation, both in primary care and in specialist neurology outpatient clinics. The International Classification of Headache Disorders, 2nd Edition (ICHD-II), enables headaches to be classified in a precise and reproducible manner. In January 2008, an outpatient headache clinic was set up in Hospital Clínico Universitario, a tertiary hospital in Valladolid, Spain. Headaches were classified prospectively in accordance with ICHD-II criteria. In each case we recorded age and sex, duration of headache, ancillary tests required, and previous symptomatic or prophylactic therapies. In January 2010, the registry included 1000 headaches in 682 patients. The women/men ratio was 2.46/1 and the mean age of the patients was 43.19 ± 17.1 years (range: 14-94 years). Patients were referred from primary care (53.4%), general neurology clinics (36.6%), and other specialist clinics (9%). The headaches were grouped (ICHD-II classification) as follows: group 1 (Migraine), 51.4%; group 2 (Tension-type headache), 16%; group 3 (Trigeminal autonomic cephalalgias), 2.6%; group 4 (Other primary headaches) and group 13 (Cranial neuralgias), 3.4%. The diagnostic criteria of chronic migraine were satisfied in 8.5% of migraines. Regarding secondary headaches, 1.1% of all cases were included in group 5 (Headaches attributed to trauma) and 8.3% in group 8 (Headaches attributed to a substance or its withdrawal). Only 3.4% of headaches were classified in group 14 (Unspecified or not elsewhere classified), and 5.2% were included in the groups listed in the ICHD-II research appendix. This registry outlines the characteristics of patients seen in an outpatient headache clinic in a tertiary hospital; our results are similar to those previously reported for this type of outpatient clinic. Migraine was the most common diagnosis. Most headaches can be classified using

  4. Current situation and challenge of registry in China.

    Science.gov (United States)

    Zhang, Yang; Feng, Yuji; Qu, Zhi; Qi, Yali; Zhan, Siyan

    2014-09-01

    Increasing emphasis has been placed on registries for an organized system used in developing clinical research to improve health care. China has sufficient data that can be applied broadly, but the heterogeneity and irregularity of registries limit their applicability. This article aims to describe the status of registries in China and the related challenges. Patient registries for observational studies were retrieved from the International Clinical Trials Registry to quantitatively evaluate the number of comparatively high-quality registries in China. A literature search was also performed to provide support and updates. A total of 64 patient registries were retrieved from ClinicalTrials.gov using disease, product, and health service as criteria. The sample sizes ranged from 15 to 30,400, with only 12 registries marked as completed. This article describes and compares the detailed information in many aspects. The efficient use of registries has already made considerable progress in China; however, registries still require standardization, high-quality transition, and coordinated development.

  5. Best clinical trials reported in 2010.

    Science.gov (United States)

    Garner, John B; Grayburn, Paul A; Yancy, Clyde W

    2011-07-01

    Each year, a number of clinical trials emerge with data sufficient to change clinical practice. Determining which findings will result in practice change and which will provide only incremental benefit can be a dilemma for clinicians. The authors review selected clinical trials reported in 2010 in journals, at society meetings, and at conferences, focusing on those studies that have the potential to change clinical practice. This review offers 3 separate means of analysis: an abbreviated text summary, organized by subject area; a comprehensive table of relevant clinical trials that provides a schematic review of the hypotheses, interventions, methods, primary end points, results, and implications; and a complete bibliography for further reading as warranted. It is hoped that this compilation of relevant clinical trials and their important findings released in 2010 will be of benefit in the everyday practice of cardiovascular medicine. Copyright © 2011 Elsevier Inc. All rights reserved.

  6. Coupled variable selection for regression modeling of complex treatment patterns in a clinical cancer registry.

    Science.gov (United States)

    Schmidtmann, I; Elsäßer, A; Weinmann, A; Binder, H

    2014-12-30

    For determining a manageable set of covariates potentially influential with respect to a time-to-event endpoint, Cox proportional hazards models can be combined with variable selection techniques, such as stepwise forward selection or backward elimination based on p-values, or regularized regression techniques such as component-wise boosting. Cox regression models have also been adapted for dealing with more complex event patterns, for example, for competing risks settings with separate, cause-specific hazard models for each event type, or for determining the prognostic effect pattern of a variable over different landmark times, with one conditional survival model for each landmark. Motivated by a clinical cancer registry application, where complex event patterns have to be dealt with and variable selection is needed at the same time, we propose a general approach for linking variable selection between several Cox models. Specifically, we combine score statistics for each covariate across models by Fisher's method as a basis for variable selection. This principle is implemented for a stepwise forward selection approach as well as for a regularized regression technique. In an application to data from hepatocellular carcinoma patients, the coupled stepwise approach is seen to facilitate joint interpretation of the different cause-specific Cox models. In conditional survival models at landmark times, which address updates of prediction as time progresses and both treatment and other potential explanatory variables may change, the coupled regularized regression approach identifies potentially important, stably selected covariates together with their effect time pattern, despite having only a small number of events. These results highlight the promise of the proposed approach for coupling variable selection between Cox models, which is particularly relevant for modeling for clinical cancer registries with their complex event patterns. Copyright © 2014 John Wiley & Sons

  7. Microbicide clinical trial adherence: insights for introduction.

    Science.gov (United States)

    Woodsong, Cynthia; MacQueen, Kathleen; Amico, K Rivet; Friedland, Barbara; Gafos, Mitzy; Mansoor, Leila; Tolley, Elizabether; McCormack, Sheena

    2013-04-08

    After two decades of microbicide clinical trials it remains uncertain if vaginally- delivered products will be clearly shown to reduce the risk of HIV infection in women and girls. Furthermore, a microbicide product with demonstrated clinical efficacy must be used correctly and consistently if it is to prevent infection. Information on adherence that can be gleaned from microbicide trials is relevant for future microbicide safety and efficacy trials, pre-licensure implementation trials, Phase IV post-marketing research, and microbicide introduction and delivery. Drawing primarily from data and experience that has emerged from the large-scale microbicide efficacy trials completed to-date, the paper identifies six broad areas of adherence lessons learned: (1) Adherence measurement in clinical trials, (2) Comprehension of use instructions/Instructions for use, (3) Unknown efficacy and its effect on adherence/Messages regarding effectiveness, (4) Partner influence on use, (5) Retention and continuation and (6) Generalizability of trial participants' adherence behavior. Each is discussed, with examples provided from microbicide trials. For each of these adherence topics, recommendations are provided for using trial findings to prepare for future microbicide safety and efficacy trials, Phase IV post-marketing research, and microbicide introduction and delivery programs.

  8. Clinical trials: bringing research to the bedside.

    Science.gov (United States)

    Arvay, C A

    1991-02-01

    Over the years, clinical trials with their structured treatment plans and multicenter involvement have been instrumental in developing new treatments and establishing standard of care therapy. While clinical trials strive to advance medical knowledge, they provide scientifically sound, state of the art care and their use should be increased. The Brain Tumor Cooperative Group, one such NCI-sponsored cooperative group, has been the primary group for the treatment of malignant gliomas. As the field of neuro-oncology expands, the neuroscience nurse needs to develop an understanding of clinical trials and their operation. The nurse is in an optimal position to support medical research and the research participant.

  9. clinical trials of Sutherlandia frutescens

    African Journals Online (AJOL)

    economic and political imperatives surrounding randomised controlled trials and the ambiguous, or even ..... the medicinal properties of the plant, as reported both in the book, and also in the .... London, UK: Harvard University Press. Latour, B.

  10. The Kidney Awareness Registry and Education (KARE) study: protocol of a randomized controlled trial to enhance provider and patient engagement with chronic kidney disease.

    Science.gov (United States)

    Tuot, Delphine S; Velasquez, Alexandra; McCulloch, Charles E; Banerjee, Tanushree; Zhu, Yunnuo; Hsu, Chi-yuan; Handley, Margaret; Schillinger, Dean; Powe, Neil R

    2015-10-22

    Chronic kidney disease (CKD) is common and is associated with excess mortality and morbidity. Better management could slow progression of disease, prevent metabolic complications, and reduce cardiovascular outcomes. Low patient awareness of CKD and ineffective patient-provider communication can impede such efforts. We developed provider and patient-directed interventions that harness health information technology to enhance provider recognition of CKD and delivery of guideline concordant care and augment patient understanding and engagement in CKD care. We report the design and protocol of the Kidney Awareness Registry and Education (KARE) Study, a 2x2 factorial randomized controlled trial that examines the impact of a multi-level intervention on health outcomes among low-income English, Spanish and Cantonese-speaking patients with CKD in a safety net system. The intervention includes: (1) implementation of a primary care electronic CKD registry that notifies practice teams of patients' CKD status and employs a patient profile and quarterly feedback to encourage provision of guideline-concordant care at point-of-care and via outreach; and (2) a language-concordant, culturally-sensitive self-management support program that consists of automated telephone modules, provision of low-literacy written patient-educational materials and telephone health coaching. The primary outcomes of the trial are changes in systolic blood pressure (BP) and the proportion of patients with BP control (≤ 140/90 mmHg) after one year. Secondary outcomes include patient understanding of CKD, participation in healthy behaviors, and practice team delivery of guideline-concordant CKD care. Results from the KARE study will provide data on the feasibility, effectiveness, and acceptability of technology-based interventions that support primary care efforts at improving health outcomes among vulnerable patients with CKD. ClinicalTrials.gov, number: NCT01530958.

  11. Registry-based randomized controlled trials merged the strength of randomized controlled trails and observational studies and give rise to more pragmatic trials.

    Science.gov (United States)

    Mathes, Tim; Buehn, Stefanie; Prengel, Peggy; Pieper, Dawid

    2018-01-01

    The objective of this study was to analyze the features of registry-based randomized trials (rRCTs). We systematically searched PubMed for rRCTs. Study selection was performed independently by two reviewers. We extracted all data in standardized tables and prepared descriptive summary statistics. The search resulted in 1,202 hits. We included 71 rRCTs. Most rRCTs were from Denmark and Sweden. Chronic conditions were considered in 82.2%. A preventive intervention was examined in 45.1%. The median of included patients was 2,000 (range: 69-246,079). Definition of the study population was mostly broad. Study procedures were regularly little standardized. The number of included and analyzed patients was the same in 82.1%. In half of the rRCTs, more than one registry was utilized. Various linkage techniques were used. In median, two outcomes were collected from the registry/ies. The median follow-up of the rRCTs was 5.3 years (range: 6 weeks to 27 years). Information on quality of registry data was reported in 11.3%. rRCTs can provide valid (randomization, low lost-to-follow-up rates, generalizable) patient important long-term comparative-effectiveness data for relative little effort. Researchers planning an RCT should always check whether existing registries can be used for data collection. Reporting on data quality must be improved for use in evidence synthesis. Copyright © 2017 Elsevier Inc. All rights reserved.

  12. Efficient design of clinical trials and epidemiological research: is it possible?

    Science.gov (United States)

    Lauer, Michael S; Gordon, David; Wei, Gina; Pearson, Gail

    2017-08-01

    Randomized clinical trials and large-scale, cohort studies continue to have a critical role in generating evidence in cardiovascular medicine; however, the increasing concern is that ballooning costs threaten the clinical trial enterprise. In this Perspectives article, we discuss the changing landscape of clinical research, and clinical trials in particular, focusing on reasons for the increasing costs and inefficiencies. These reasons include excessively complex design, overly restrictive inclusion and exclusion criteria, burdensome regulations, excessive source-data verification, and concerns about the effect of clinical research conduct on workflow. Thought leaders have called on the clinical research community to consider alternative, transformative business models, including those models that focus on simplicity and leveraging of digital resources. We present some examples of innovative approaches by which some investigators have successfully conducted large-scale, clinical trials at relatively low cost. These examples include randomized registry trials, cluster-randomized trials, adaptive trials, and trials that are fully embedded within digital clinical care or administrative platforms.

  13. Marketing and clinical trials: a case study

    Directory of Open Access Journals (Sweden)

    Entwistle Vikki A

    2007-11-01

    Full Text Available Abstract Background Publicly funded clinical trials require a substantial commitment of time and money. To ensure that sufficient numbers of patients are recruited it is essential that they address important questions in a rigorous manner and are managed well, adopting effective marketing strategies. Methods Using methods of analysis drawn from management studies, this paper presents a structured assessment framework or reference model, derived from a case analysis of the MRC's CRASH trial, of 12 factors that may affect the success of the marketing and sales activities associated with clinical trials. Results The case study demonstrates that trials need various categories of people to buy in – hence, to be successful, trialists must embrace marketing strategies to some extent. Conclusion The performance of future clinical trials could be enhanced if trialists routinely considered these factors.

  14. Marketing and clinical trials: a case study.

    Science.gov (United States)

    Francis, David; Roberts, Ian; Elbourne, Diana R; Shakur, Haleema; Knight, Rosemary C; Garcia, Jo; Snowdon, Claire; Entwistle, Vikki A; McDonald, Alison M; Grant, Adrian M; Campbell, Marion K

    2007-11-20

    Publicly funded clinical trials require a substantial commitment of time and money. To ensure that sufficient numbers of patients are recruited it is essential that they address important questions in a rigorous manner and are managed well, adopting effective marketing strategies. Using methods of analysis drawn from management studies, this paper presents a structured assessment framework or reference model, derived from a case analysis of the MRC's CRASH trial, of 12 factors that may affect the success of the marketing and sales activities associated with clinical trials. The case study demonstrates that trials need various categories of people to buy in - hence, to be successful, trialists must embrace marketing strategies to some extent. The performance of future clinical trials could be enhanced if trialists routinely considered these factors.

  15. OARSI Clinical Trials Recommendations: Design and conduct of clinical trials of rehabilitation interventions for osteoarthritis.

    Science.gov (United States)

    Fitzgerald, G K; Hinman, R S; Zeni, J; Risberg, M A; Snyder-Mackler, L; Bennell, K L

    2015-05-01

    A Task Force of the Osteoarthritis Research Society International (OARSI) has previously published a set of guidelines for the conduct of clinical trials in osteoarthritis (OA) of the hip and knee. Limited material available on clinical trials of rehabilitation in people with OA has prompted OARSI to establish a separate Task Force to elaborate guidelines encompassing special issues relating to rehabilitation of OA. The Task Force identified three main categories of rehabilitation clinical trials. The categories included non-operative rehabilitation trials, post-operative rehabilitation trials, and trials examining the effectiveness of devices (e.g., assistive devices, bracing, physical agents, electrical stimulation, etc.) that are used in rehabilitation of people with OA. In addition, the Task Force identified two main categories of outcomes in rehabilitation clinical trials, which include outcomes related to symptoms and function, and outcomes related to disease modification. The guidelines for rehabilitation clinical trials provided in this report encompass these main categories. The report provides guidelines for conducting and reporting on randomized clinical trials. The topics include considerations for entering patients into trials, issues related to conducting trials, considerations for selecting outcome measures, and recommendations for statistical analyses and reporting of results. The focus of the report is on rehabilitation trials for hip, knee and hand OA, however, we believe the content is broad enough that it could be applied to rehabilitation trials for other regions as well. Copyright © 2015 Osteoarthritis Research Society International. Published by Elsevier Ltd. All rights reserved.

  16. Clinical Differences between Subtypes of Atrial Fibrillation and Flutter: Cross-Sectional Registry of 407 Patients

    Directory of Open Access Journals (Sweden)

    Eduardo Dytz Almeida

    2015-01-01

    Full Text Available Introduction: Atrial fibrillation and atrial flutter account for one third of hospitalizations due to arrhythmias, determining great social and economic impacts. In Brazil, data on hospital care of these patients is scarce. Objective: To investigate the arrhythmia subtype of atrial fibrillation and flutter patients in the emergency setting and compare the clinical profile, thromboembolic risk and anticoagulants use. Methods: Cross-sectional retrospective study, with data collection from medical records of every patient treated for atrial fibrillation and flutter in the emergency department of Instituto de Cardiologia do Rio Grande do Sul during the first trimester of 2012. Results: We included 407 patients (356 had atrial fibrillation and 51 had flutter. Patients with paroxysmal atrial fibrillation were in average 5 years younger than those with persistent atrial fibrillation. Compared to paroxysmal atrial fibrillation patients, those with persistent atrial fibrillation and flutter had larger atrial diameter (48.6 ± 7.2 vs. 47.2 ± 6.2 vs. 42.3 ± 6.4; p < 0.01 and lower left ventricular ejection fraction (66.8 ± 11 vs. 53.9 ± 17 vs. 57.4 ± 16; p < 0.01. The prevalence of stroke and heart failure was higher in persistent atrial fibrillation and flutter patients. Those with paroxysmal atrial fibrillation and flutter had higher prevalence of CHADS2 score of zero when compared to those with persistent atrial fibrillation (27.8% vs. 18% vs. 4.9%; p < 0.01. The prevalence of anticoagulation in patients with CHA2DS2-Vasc ≤ 2 was 40%. Conclusions: The population in our registry was similar in its comorbidities and demographic profile to those of North American and European registries. Despite the high thromboembolic risk, the use of anticoagulants was low, revealing difficulties for incorporating guideline recommendations. Public health strategies should be adopted in order to improve these rates.

  17. Problematic trial detection in ClinicalTrials.gov

    NARCIS (Netherlands)

    Hartgerink, C.H.J.; George, Stephen

    2015-01-01

    Clinical trials are crucial in determining the effectiveness of treatments and directly affect clinical and policy decisions. These decisions are undermined if the data are problematic due to data fabrication or other errors. Researchers have worked on developing statistical methods to detect

  18. A machine learning approach to identify clinical trials involving nanodrugs and nanodevices from ClinicalTrials.gov.

    Science.gov (United States)

    de la Iglesia, Diana; García-Remesal, Miguel; Anguita, Alberto; Muñoz-Mármol, Miguel; Kulikowski, Casimir; Maojo, Víctor

    2014-01-01

    Clinical Trials (CTs) are essential for bridging the gap between experimental research on new drugs and their clinical application. Just like CTs for traditional drugs and biologics have helped accelerate the translation of biomedical findings into medical practice, CTs for nanodrugs and nanodevices could advance novel nanomaterials as agents for diagnosis and therapy. Although there is publicly available information about nanomedicine-related CTs, the online archiving of this information is carried out without adhering to criteria that discriminate between studies involving nanomaterials or nanotechnology-based processes (nano), and CTs that do not involve nanotechnology (non-nano). Finding out whether nanodrugs and nanodevices were involved in a study from CT summaries alone is a challenging task. At the time of writing, CTs archived in the well-known online registry ClinicalTrials.gov are not easily told apart as to whether they are nano or non-nano CTs-even when performed by domain experts, due to the lack of both a common definition for nanotechnology and of standards for reporting nanomedical experiments and results. We propose a supervised learning approach for classifying CT summaries from ClinicalTrials.gov according to whether they fall into the nano or the non-nano categories. Our method involves several stages: i) extraction and manual annotation of CTs as nano vs. non-nano, ii) pre-processing and automatic classification, and iii) performance evaluation using several state-of-the-art classifiers under different transformations of the original dataset. The performance of the best automated classifier closely matches that of experts (AUC over 0.95), suggesting that it is feasible to automatically detect the presence of nanotechnology products in CT summaries with a high degree of accuracy. This can significantly speed up the process of finding whether reports on ClinicalTrials.gov might be relevant to a particular nanoparticle or nanodevice, which is

  19. Maximizing scientific knowledge from randomized clinical trials

    DEFF Research Database (Denmark)

    Gustafsson, Finn; Atar, Dan; Pitt, Bertram

    2010-01-01

    , in particular with respect to collaboration with the trial sponsor and to analytic pitfalls. The advantages of creating screening databases in conjunction with a given clinical trial are described; and finally, the potential for posttrial database studies to become a platform for training young scientists...

  20. Critical concepts in adaptive clinical trials

    Directory of Open Access Journals (Sweden)

    Park JJH

    2018-03-01

    Full Text Available Jay JH Park,1 Kristian Thorlund,2,3 Edward J Mills2,3 1Department of Medicine, University of British Columbia, Vancouver, BC, Canada; 2Department of Health Research Methods, Evidence, and Impact (HEI, McMaster University, Hamilton, ON, Canada; 3The Bill and Melinda Gates Foundation, Seattle, WA, USA Abstract: Adaptive clinical trials are an innovative trial design aimed at reducing resources, decreasing time to completion and number of patients exposed to inferior interventions, and improving the likelihood of detecting treatment effects. The last decade has seen an increasing use of adaptive designs, particularly in drug development. They frequently differ importantly from conventional clinical trials as they allow modifications to key trial design components during the trial, as data is being collected, using preplanned decision rules. Adaptive designs have increased likelihood of complexity and also potential bias, so it is important to understand the common types of adaptive designs. Many clinicians and investigators may be unfamiliar with the design considerations for adaptive designs. Given their complexities, adaptive trials require an understanding of design features and sources of bias. Herein, we introduce some common adaptive design elements and biases and specifically address response adaptive randomization, sample size reassessment, Bayesian methods for adaptive trials, seamless trials, and adaptive enrichment using real examples. Keywords: adaptive designs, response adaptive randomization, sample size reassessment, Bayesian adaptive trials, seamless trials, adaptive enrichment

  1. A web-based, patient driven registry for Angelman syndrome: the global Angelman syndrome registry.

    Science.gov (United States)

    Napier, Kathryn R; Tones, Megan; Simons, Chloe; Heussler, Helen; Hunter, Adam A; Cross, Meagan; Bellgard, Matthew I

    2017-08-01

    Angelman syndrome (AS) is a rare neurodevelopmental disorder that is characterised by severe global developmental delays, ataxia, loss of speech, epilepsy, sleep disorders, and a happy disposition. There is currently no cure for AS, though several pharmaceutical companies are anticipating drug trials for new therapies to treat AS. The Foundation for Angelman Therapeutics (FAST) Australia therefore identified a need for a global AS patient registry to identify patients for recruitment for clinical trials.The Global AS Registry was deployed in September 2016 utilising the Rare Disease Registry Framework, an open-source tool that enables the efficient creation and management of patient registries. The Global AS Registry is web-based and allows parents and guardians worldwide to register, provide informed consent, and enter data on individuals with AS. 286 patients have registered in the first 8 months since deployment.We demonstrate the successful deployment of the first patient-driven global registry for AS. The data generated from the Global AS Registry will be crucial in identifying patients suitable for clinical trials and in informing research that will identify treatments for AS, and ultimately improve the lives of individuals and their families living with AS.

  2. Variation in performance measure criteria significantly affects cardiology practice rankings: Insights from the National Cardiovascular Data Registry's Practice Innovation and Clinical Excellence Registry.

    Science.gov (United States)

    Eapen, Zubin J; Tang, Fengming; Jones, Phil G; Maddox, Thomas M; Oetgen, William J; Spertus, John A; Rumsfeld, John S; Heidenreich, Paul A; Peterson, Eric D; Drozda, Joseph P

    2015-06-01

    Million Hearts is a national initiative to prevent 1 million heart attacks and strokes over 5 years by improving cardiovascular prevention. An important tool in the success of programs like Million Hearts is public ranking on the quality of practices, yet different measures may provide different rankings, so the true quality of practices is difficult to discern. We evaluated the quality of ambulatory cardiology care using performance measure metrics. We compared rankings of practices participating in the National Cardiovascular Data Registry's Practice Innovation and Clinical Excellence Registry using measures from (1) the physician quality reporting system and (2) the American College of Cardiology/American Heart Association/Physician Consortium for Performance Improvement. We compared achievement rates for measures between the 2 frameworks and determined correlations in rankings using Spearman correlation coefficients. From January 1, 2008 to December 31, 2012, there were 1,711,326 patients enrolled from 111 US practices. Among eligible patients, the physician quality reporting system and American College of Cardiology/American Heart Association/Physician Consortium for Performance Improvement measures were achieved in 76.1% versus 77.4% for antiplatelet prescription (P performance and failing to achieve public health goals. Copyright © 2015 Elsevier Inc. All rights reserved.

  3. Clinical trial data analysis using R

    National Research Council Canada - National Science Library

    Chen, Ding-Geng; Peace, Karl E

    2011-01-01

    .... Case studies demonstrate how to select the appropriate clinical trial data. The authors introduce the corresponding biostatistical analysis methods, followed by the step-by-step data analysis using R...

  4. Overcoming Age Limits in Cancer Clinical Trials

    Science.gov (United States)

    Adolescents, young adults, and the elderly lag far behind other age groups when it comes to enrolling in clinical trials. Their participation is critical to advancing effective therapies for these age groups.

  5. Blinding in randomized clinical trials: imposed impartiality

    DEFF Research Database (Denmark)

    Hróbjartsson, A; Boutron, I

    2011-01-01

    Blinding, or "masking," is a crucial method for reducing bias in randomized clinical trials. In this paper, we review important methodological aspects of blinding, emphasizing terminology, reporting, bias mechanisms, empirical evidence, and the risk of unblinding. Theoretical considerations...

  6. ORIGINAL ARTICLES Pharmacologically active: clinical trials and ...

    African Journals Online (AJOL)

    2008-01-22

    Jan 22, 2008 ... The US database, on the other hand, clearly identifies 172 ... operating within extended clinical trials R&D value chains. Companies often ... Source: CeSTII Survey Management and Results System internal database. Table III.

  7. Clinical outcomes in clinical trials of anti-HIV treatment

    DEFF Research Database (Denmark)

    Reekie, J; Mocroft, A; J, Neaton

    2007-01-01

    Since the introduction of combination antiretroviral therapy, there has been a decrease in both AIDS-defining illnesses and deaths. This decrease meant that performing clinical trials with clinical outcomes in HIV infection became more time consuming and hence costly. Improved understanding...... the infection, so when treatment is started it is currently a lifelong commitment. Is it reasonable then that guidelines are based almost completely on short-term randomized trials and observational studies of surrogate markers, or is there still a need for trials with clinical outcomes?...

  8. Beyond PubMed: Searching the "Grey Literature" for Clinical Trial Results.

    Science.gov (United States)

    Citrome, Leslie

    2014-07-01

    Clinical trial results have been traditionally communicated through the publication of scholarly reports and reviews in biomedical journals. However, this dissemination of information can be delayed or incomplete, making it difficult to appraise new treatments, or in the case of missing data, evaluate older interventions. Going beyond the routine search of PubMed, it is possible to discover additional information in the "grey literature." Examples of the grey literature include clinical trial registries, patent databases, company and industrywide repositories, regulatory agency digital archives, abstracts of paper and poster presentations on meeting/congress websites, industry investor reports and press releases, and institutional and personal websites.

  9. Clinical Benefit of Ablating Localized Sources for Human Atrial Fibrillation: The Indiana University FIRM Registry.

    Science.gov (United States)

    Miller, John M; Kalra, Vikas; Das, Mithilesh K; Jain, Rahul; Garlie, Jason B; Brewster, Jordan A; Dandamudi, Gopi

    2017-03-14

    Mounting evidence shows that localized sources maintain atrial fibrillation (AF). However, it is unclear in unselected "real-world" patients if sources drive persistent atrial fibrillation (PeAF), long-standing persistent atrial fibrillation (LPeAF), or paroxysmal atrial fibrillation (PAF); if right atrial sites are important; and what the long-term success of source ablation is. The aim of this study was to analyze the role of rotors and focal sources in a large academic registry of consecutive patients undergoing source mapping for AF. One hundred seventy consecutive patients (mean age 59 ± 12 years, 79% men) with PAF (37%), PeAF (31%), or LPeAF (32%). Of these, 73 (43%) had undergone at least 1 prior ablation attempt (mean 1.9 ± 0.8; range: 1 to 4). Focal impulse and rotor modulation (FIRM) with an endocardial basket catheter was used in all cases. FIRM analysis revealed sources in the right atrium in 85% of patients (1.8 ± 1.3) and in the left atrium in 90% of patients (2.0 ± 1.3). FIRM ablation terminated AF to sinus rhythm or atrial flutter or tachycardia in 59% (PAF), 37% (PeAF), and 19% (LPeAF) of patients, with 15 of 67 terminations due to right atrial ablation. On follow-up, freedom from AF after a single FIRM procedure for the entire series was 95% (PAF), 83% (PeAF), and 82% (LPeAF) at 1 year and freedom from all atrial arrhythmias was 77% (PAF), 75% (PeAF), and 57% (LPeAF). In the Indiana University FIRM registry, FIRM-guided ablation produced high single-procedure success, mostly in patients with nonparoxysmal AF. Data from mapping, acute terminations, and outcomes strongly support the mechanistic role of biatrial rotors and focal sources in maintaining AF in diverse populations. Randomized trials of FIRM-guided ablation and mechanistic studies to determine how rotors form, progress, and regress are needed. Copyright © 2017 American College of Cardiology Foundation. Published by Elsevier Inc. All rights reserved.

  10. Clinical outcomes in clinical trials of anti-HIV treatment

    DEFF Research Database (Denmark)

    Reekie, J; Mocroft, A; J, Neaton

    2007-01-01

    Since the introduction of combination antiretroviral therapy, there has been a decrease in both AIDS-defining illnesses and deaths. This decrease meant that performing clinical trials with clinical outcomes in HIV infection became more time consuming and hence costly. Improved understanding...... and knowledge of HIV led to short-term trials using surrogate outcomes such as viral load and CD4 count. This established a faster drug approval process that complimented the rapid need to evaluate and provide access to drugs based on short-term trials. However, no treatment has yet been found that eradicates...... the infection, so when treatment is started it is currently a lifelong commitment. Is it reasonable then that guidelines are based almost completely on short-term randomized trials and observational studies of surrogate markers, or is there still a need for trials with clinical outcomes?...

  11. Developments in statistical evaluation of clinical trials

    CERN Document Server

    Oud, Johan; Ghidey, Wendimagegn

    2014-01-01

    This book describes various ways of approaching and interpreting the data produced by clinical trial studies, with a special emphasis on the essential role that biostatistics plays in clinical trials. Over the past few decades the role of statistics in the evaluation and interpretation of clinical data has become of paramount importance. As a result the standards of clinical study design, conduct and interpretation have undergone substantial improvement. The book includes 18 carefully reviewed chapters on recent developments in clinical trials and their statistical evaluation, with each chapter providing one or more examples involving typical data sets, enabling readers to apply the proposed procedures. The chapters employ a uniform style to enhance comparability between the approaches.

  12. Registry Assessment of Peripheral Interventional Devices (RAPID): Registry assessment of peripheral interventional devices core data elements.

    Science.gov (United States)

    Jones, W Schuyler; Krucoff, Mitchell W; Morales, Pablo; Wilgus, Rebecca W; Heath, Anne H; Williams, Mary F; Tcheng, James E; Marinac-Dabic, J Danica; Malone, Misti L; Reed, Terrie L; Fukaya, Rie; Lookstein, Robert A; Handa, Nobuhiro; Aronow, Herbert D; Bertges, Daniel J; Jaff, Michael R; Tsai, Thomas T; Smale, Joshua A; Zaugg, Margo J; Thatcher, Robert J; Cronenwett, Jack L

    2018-02-01

    The current state of evaluating patients with peripheral artery disease and more specifically of evaluating medical devices used for peripheral vascular intervention (PVI) remains challenging because of the heterogeneity of the disease process, the multiple physician specialties that perform PVI, the multitude of devices available to treat peripheral artery disease, and the lack of consensus about the best treatment approaches. Because PVI core data elements are not standardized across clinical care, clinical trials, and registries, aggregation of data across different data sources and physician specialties is currently not feasible. Under the auspices of the U.S. Food and Drug Administration's Medical Device Epidemiology Network initiative-and its PASSION (Predictable and Sustainable Implementation of the National Registries) program, in conjunction with other efforts to align clinical data standards-the Registry Assessment of Peripheral Interventional Devices (RAPID) workgroup was convened. RAPID is a collaborative, multidisciplinary effort to develop a consensus lexicon and to promote interoperability across clinical care, clinical trials, and national and international registries of PVI. The current manuscript presents the initial work from RAPID to standardize clinical data elements and definitions, to establish a framework within electronic health records and health information technology procedural reporting systems, and to implement an informatics-based approach to promote the conduct of pragmatic clinical trials and registry efforts in PVI. Ultimately, we hope this work will facilitate and improve device evaluation and surveillance for patients, clinicians, health outcomes researchers, industry, policymakers, and regulators. Copyright © 2017 Society for Vascular Surgery. All rights reserved.

  13. Inclusion of Minority Patients in Breast Cancer Clinical Trials: The Role of the Clinical Trial Environment

    National Research Council Canada - National Science Library

    Kaplan, Celia P

    2007-01-01

    .... While inroads to increasing minority inclusion in breast cancer clinical trials have been made, recent reports continue to demonstrate lower enrollment among African Americans, Asian Americans...

  14. National Cancer Patient Registry--a patient registry/clinical database to evaluate the health outcomes of patients undergoing treatment for cancers in Malaysia.

    Science.gov (United States)

    Lim, G C C; Azura, D

    2008-09-01

    Cancer burden in Malaysia is increasing. Although there have been improvements in cancer treatment, these new therapies may potentially cause an exponential increase in the cost of cancer treatment. Therefore, justification for the use of these treatments is mandated. Availability of local data will enable us to evaluate and compare the outcome of our patients. This will help to support our clinical decision making and local policy, improve access to treatment and improve the provision and delivery of oncology services in Malaysia. The National Cancer Patient Registry was proposed as a database for cancer patients who seek treatment in Malaysia. It will be a valuable tool to provide timely and robust data on the actual setting in oncology practice, safety and cost effectiveness of treatment and most importantly the outcome of these patients.

  15. Parents' perceived obstacles to pediatric clinical trial participation: Findings from the clinical trials transformation initiative.

    Science.gov (United States)

    Greenberg, Rachel G; Gamel, Breck; Bloom, Diane; Bradley, John; Jafri, Hasan S; Hinton, Denise; Nambiar, Sumathi; Wheeler, Chris; Tiernan, Rosemary; Smith, P Brian; Roberts, Jamie; Benjamin, Daniel K

    2018-03-01

    Enrollment of children into pediatric clinical trials remains challenging. More effective strategies to improve recruitment of children into trials are needed. This study used in-depth qualitative interviews with parents who were approached to enroll their children in a clinical trial in order to gain an understanding of the barriers to pediatric clinical trial participation. Twenty-four parents whose children had been offered the opportunity to participate in a clinical trial were interviewed: 19 whose children had participated in at least 1 clinical trial and 5 who had declined participation in any trial. Each study aspect, from the initial explanation of the study to the end of the study, can affect the willingness of parents to consent to the proposed study and future studies. Establishing trust, appropriate timing, a transparent discussion of risks and benefits oriented to the layperson, and providing motivation for children to participate were key factors that impacted parents' decisions. In order for clinical trial accrual to be successful, parents' priorities and considerations must be a central focus, beginning with initial trial design. The recommendations from the parents who participated in this study can be used to support budget allocations that ensure adequate training of study staff and improved staffing on nights and weekends. Studies of parent responses in outpatient settings and additional inpatient settings will provide valuable information on the consent process from the child's and parent's perspectives. Further studies are needed to explore whether implementation of such strategies will result in improved recruitment for pediatric clinical trials.

  16. Patient engagement in clinical trials: The Clinical Trials Transformation Initiative's leadership from theory to practical implementation.

    Science.gov (United States)

    Patrick-Lake, Bray

    2018-02-01

    Patient engagement is an increasingly important aspect of successful clinical trials. Over the past decade, as patient group involvement in clinical trials has continued to increase and diversify, the Clinical Trials Transformation Initiative has not only recognized the crucial role patients play in improving the clinical trial enterprise but also made a deep commitment to help grow and shape the emerging field of patient engagement. This article describes the evolution of patient engagement including the origins of the patient engagement movement; barriers to successful engagement and remaining challenges to full and valuable collaboration between patient groups and trial sponsors; and Clinical Trials Transformation Initiative's role in influencing the field through organizational practices, formal project work and resulting recommendations, and external advocacy efforts.

  17. An analysis of registered clinical trials in otolaryngology from 2007 to 2010: ClinicalTrials.gov.

    Science.gov (United States)

    Witsell, David L; Schulz, Kristine A; Lee, Walter T; Chiswell, Karen

    2013-11-01

    To describe the conditions studied, interventions used, study characteristics, and funding sources of otolaryngology clinical trials from the ClinicalTrials.gov database; compare this otolaryngology cohort of interventional studies to clinical visits in a health care system; and assess agreement between clinical trials and clinical activity. Database analysis. Trial registration data downloaded from ClinicalTrials.gov and administrative data from the Duke University Medical Center from October 1, 2007 to September 27, 2010. Data extraction from ClinicalTrials.gov was done using MeSH and non-MeSH disease condition terms. Studies were subcategorized to create the following groupings for descriptive analysis: ear, nose, allergy, voice, sleep, head and neck cancer, thyroid, and throat. Duke Health System visits were queried by using selected ICD-9 codes for otolaryngology and non-otolaryngology providers. Visits were grouped similarly to ClinicalTrials.gov for further analysis. Chi-square tests were used to explore differences between groups. A total of 1115 of 40,970 registered interventional trials were assigned to otolaryngology. Head and neck cancer trials predominated. Study models most frequently incorporated parallel design (54.6%), 2 study groups (46.6%), and randomization (69.1%). Phase 2 or 3 studies constituted 46.4% of the cohort. Comparison of the ClinicalTrials.gov database with administrative health system visit data by disease condition showed discordance between national research activity and clinical visit volume for patients with otolaryngology complaints. Analysis of otolaryngology-related clinical research as listed in ClinicalTrials.gov can inform patients, physicians, and policy makers about research focus areas. The relative burden of otolaryngology-associated conditions in our tertiary health system exceeds research activity within the field.

  18. The patient's safety and access to experimental drugs after the termination of clinical trials: regulations and trends.

    Science.gov (United States)

    da Silva, Ricardo Eccard; Amato, Angélica Amorim; Sousa, Thiago do Rego; de Carvalho, Marta Rodrigues; Novaes, Maria Rita Carvalho Garbi

    2018-05-12

    Participants' rights and safety must be guaranteed not only while a clinical trial is being conducted but also when a clinical trial finishes. The criteria for post-trial access to experimental drugs, however, are unclear in various countries. The objectives of this study were (i) to ascertain if there were regulations or guidelines related to patients' access to drugs after the end of clinical trials in the countries selected in the study and (ii) to analyze trends in post-trial access in countries classified by their level of economic development. This study is a retrospective review. The data are from the records of clinical trials from 2014 registered in the World Health Organization's International Clinical Trials Registry Platform (ICTRP) database. Among the countries selected, provision of drugs post-trial is mandatory only in Argentina, Brazil, Chile, Finland, and Peru. The plans for post-trial access tend to be more present in low- and middle-income and upper middle-income countries, in comparison with high-income countries. Studies involving vulnerable populations are 2.53 times more likely to have plans for post-trial access than studies which do not. The guaranteeing of post-trial access remains mandatory in few countries. Considering that individuals seen as vulnerable have been included in clinical trials without plans for post-trial access, stakeholders must discuss the need to develop regulations mandating the guaranteeing of post-trial access in specified situations.

  19. Diagnostic clinical and laboratory findings in response to predetermining bacterial pathogen: data from the Meningitis Registry.

    Directory of Open Access Journals (Sweden)

    Maria Karanika

    Full Text Available BACKGROUND: Childhood meningitis continues to be an important cause of mortality in many countries. The search for rapid diagnosis of acute bacterial meningitis has lead to the further exploration of prognostic factors. This study was scheduled in an attempt to analyze various clinical symptoms as well as rapid laboratory results and provide an algorithm for the prediction of specific bacterial aetiology of childhood bacterial meningitis. METHODOLOGY AND PRINCIPAL FINDINGS: During the 32 year period, 2477 cases of probable bacterial meningitis (BM were collected from the Meningitis Registry (MR. Analysis was performed on a total of 1331 confirmed bacterial meningitis cases of patients aged 1 month to 14 years. Data was analysed using EPI INFO (version 3.4.3-CDC-Atlanta and SPSS (version 15.0-Chicago software. Statistically significant (p or = 15000/microL (OR 2.19 with a PPV of 77.8% (95%CI 40.0-97.2. For the diagnosis of Haemophilus influenzae, the most significant group of diagnostic criteria included, absence of haemorrhagic rash (OR 13.61, age > or = 1 year (OR 2.04, absence of headache (OR 3.01, CSF Glu < 40 mg/dL (OR 3.62 and peripheral WBC < 15,000/microL (OR 1.74 with a PPV of 58.5% (95%CI 42.1-73.7. CONCLUSIONS: The use of clinical and laboratory predictors for the assessment of the causative bacterial pathogen rather than just for predicting outcome of mortality seems to be a useful tool in the clinical management and specific treatment of BM. These findings should be further explored and studied.

  20. Public information about clinical trials and research.

    Science.gov (United States)

    Plétan, Yannick; Zannad, Faïez; Jaillon, Patrice

    2003-01-01

    Be it to restore the confused image of clinical research in relation to the lay public, or to develop new ways of accruing healthy volunteers or patients for clinical trials, there is a need to draft some guidance on how best to provide information on research. Although the French legal and regulatory armamentarium in this area is essentially liberal, there is currently little-justified reluctance among study sponsors to advertise publicly. A group of academic and pharmaceutical industry researchers, assembled for a workshop, together with regulators, journalists, representatives from ethics committees, social security, patient and health consumer groups and other French institutional bodies, has suggested the following series of recommendations: there is no need for additional legal or regulatory constraints; sponsors should be aware of and make use of direct public information on trials; a 'good practice charter' on public communication about clinical trials should be developed; all professionals should be involved in this communication platform; communication in the patient's immediate vicinity should be preferred (primary-care physician, local press); clinical databases and websites accessible to professionals, but also to patients and non-professionals, should be developed; genuine instruction on clinical trials for physicians and health professionals unfamiliar with such trials should be developed and disseminated; media groups should receive at least some training in the fundamentals of clinical research.

  1. Clinical presentation of intraocular retinoblastoma; 5-year hospital-based registry in Egypt

    International Nuclear Information System (INIS)

    El Zomor, H.; Nour, R.; Alieldin, A.; Taha, H.; Montasr, M.M.; Moussa, E.; El Nadi, E.; Alfaar, A.S.; Alfaar, A.S.; El Zomor, H.; Taha, H.; Alieldin, A.; Montasr, M.M.; Moussa, E.; El Nadi, E.; Ezzat, S.

    2015-01-01

    Purpose: To study the presenting signs of Retinoblastoma in Egypt at Egypt’s main pediatric oncology referral center. Methods: This is a prospective descriptive study (hospital-based registry) conducted at Children’s Cancer Hospital Egypt between July 2007 and December 2012. Results: Out of 262 patients diagnosed with retinoblastoma, 244 were suffering from intra-ocular disease at presentation. One hundred thirty-nine (57%) patients presented with unilateral disease, while 105 (43%) suffered bilateral disease. The mean age at presentation was 20.6 ± 17 months, averaging 18.87 ± 11.76 months for bilateral and 25.72 ± 18.78 months for unilateral disease. The most common clinical presentation was leukocoria in 180 (73.8%) patients, strabismus in 32 (13.1%) patients and decreased visual acuity in 12 (4.9%) patients. Group D and E disease represented 62% of all affected eyes. Patients with advanced disease (Group C–E) had longer duration of symptoms.Conclusion: In Egypt, retinoblastoma patients present more frequently with advanced disease. There is an ever-increasing need to develop a national team dedicated to studying disease significance and formulating a national awareness program.

  2. Clinical Databases and Registries in Congenital and Pediatric Cardiac Surgery, Cardiology, Critical Care, and Anesthesiology Worldwide.

    Science.gov (United States)

    Vener, David F; Gaies, Michael; Jacobs, Jeffrey P; Pasquali, Sara K

    2017-01-01

    The growth in large-scale data management capabilities and the successful care of patients with congenital heart defects have coincidentally paralleled each other for the last three decades, and participation in multicenter congenital heart disease databases and registries is now a fundamental component of cardiac care. This manuscript attempts for the first time to consolidate in one location all of the relevant databases worldwide, including target populations, specialties, Web sites, and participation information. Since at least 1,992 cardiac surgeons and cardiologists began leveraging this burgeoning technology to create multi-institutional data collections addressing a variety of specialties within this field. Pediatric heart diseases are particularly well suited to this methodology because each individual care location has access to only a relatively limited number of diagnoses and procedures in any given calendar year. Combining multiple institutions data therefore allows for a far more accurate contemporaneous assessment of treatment modalities and adverse outcomes. Additionally, the data can be used to develop outcome benchmarks by which individual institutions can measure their progress against the field as a whole and focus quality improvement efforts in a more directed fashion, and there is increasing utilization combining clinical research efforts within existing data structures. Efforts are ongoing to support better collaboration and integration across data sets, to improve efficiency, further the utility of the data collection infrastructure and information collected, and to enhance return on investment for participating institutions.

  3. Involving South Asian patients in clinical trials.

    Science.gov (United States)

    Hussain-Gambles, M; Leese, B; Atkin, K; Brown, J; Mason, S; Tovey, P

    2004-10-01

    To investigate how South Asian patients conceptualise the notion of clinical trials and to identify key processes that impact on trial participation and the extent to which communication difficulties, perceptions of risk and attitudes to authority influence these decisions. Also to identify whether 'South Asian' patients are homogeneous in these issues, and which factors differ between different South Asian subgroups and finally how professionals regard the involvement of South Asian patients and their views on strategies to increase participation. A review of the literature on minority ethnic participation in clinical trials was followed by three qualitative interview studies. Interviews were taped and transcribed (and translated if required) and subjected to framework analysis. Face-to-face interviews were conducted with 25 health professionals; 60 South Asian lay people who had not taken part in a trial and 15 South Asian trial participants. Motivations for trial participation were identified as follows: to help society, to improve own health or that of family and friends, out of obligation to the doctor and to increase scientific knowledge. Deterrents were concerns about drug side-effects, busy lifestyles, language, previous bad experiences, mistrust and feelings of not belonging to British society. There was no evidence of antipathy amongst South Asians to the concept of clinical trials and, overall, the younger respondents were more knowledgeable than the older ones. Problems are more likely to be associated with service delivery. Lack of being approached was a common response. Lay-reported factors that might affect South Asian participation in clinical trials include age, language, social class, feeling of not belonging/mistrust, culture and religion. Awareness of clinical trials varied between each group. There are more similarities than differences in attitudes towards clinical trial participation between the South Asian and the general population

  4. The geographical distribution of leadership in globalized clinical trials.

    Directory of Open Access Journals (Sweden)

    Jarno Hoekman

    Full Text Available BACKGROUND: Pharmaceutical trials are mainly initiated by sponsors and investigators in the United States, Western Europe and Japan. However, more and more patients are enrolled in Central and Eastern Europe, Latin America and Asia. The involvement of patients in new geographical settings raises questions about scientific and ethical integrity, especially when experience with those settings is lacking at the level of trial management. We therefore studied to what extent the geographical shift in patient enrolment is anticipated in the composition of trial management teams using the author nationalities on the primary outcome publication as an indicator of leadership. METHODS AND FINDINGS: We conducted a cohort-study among 1,445 registered trials in www.clinicaltrials.gov that could be matched with a primary outcome publication using clinical trial registry numbers listed in publications. The name of the sponsor and the enrolment countries were extracted from all registrations. The author-addresses of all authors were extracted from the publications. We searched the author-address of all publications to determine whether enrolment countries and sponsors listed on registrations also appeared on a matched publication. Of all sponsors, 80.1% were listed with an author-address on the publication. Of all enrolment countries, 50.3% appeared with an author-address on the publication. The listing of enrolment countries was especially low for industry-funded trials (39.9% as compared to government (90.4% and not-for-profit funding (93.7%. We found that listing of enrolment countries in industry-funded trials was higher for traditional research locations such as the United States (98.2% and Japan (72.0% as compared to nontraditional research locations such as Poland (27.3% and Mexico (14.1%. CONCLUSIONS: Despite patient enrolment efforts, the involvement of researchers from nontraditional locations in trial management as measured by their contribution to

  5. The geographical distribution of leadership in globalized clinical trials.

    Science.gov (United States)

    Hoekman, Jarno; Frenken, Koen; de Zeeuw, Dick; Heerspink, Hiddo Lambers

    2012-01-01

    Pharmaceutical trials are mainly initiated by sponsors and investigators in the United States, Western Europe and Japan. However, more and more patients are enrolled in Central and Eastern Europe, Latin America and Asia. The involvement of patients in new geographical settings raises questions about scientific and ethical integrity, especially when experience with those settings is lacking at the level of trial management. We therefore studied to what extent the geographical shift in patient enrolment is anticipated in the composition of trial management teams using the author nationalities on the primary outcome publication as an indicator of leadership. We conducted a cohort-study among 1,445 registered trials in www.clinicaltrials.gov that could be matched with a primary outcome publication using clinical trial registry numbers listed in publications. The name of the sponsor and the enrolment countries were extracted from all registrations. The author-addresses of all authors were extracted from the publications. We searched the author-address of all publications to determine whether enrolment countries and sponsors listed on registrations also appeared on a matched publication. Of all sponsors, 80.1% were listed with an author-address on the publication. Of all enrolment countries, 50.3% appeared with an author-address on the publication. The listing of enrolment countries was especially low for industry-funded trials (39.9%) as compared to government (90.4%) and not-for-profit funding (93.7%). We found that listing of enrolment countries in industry-funded trials was higher for traditional research locations such as the United States (98.2%) and Japan (72.0%) as compared to nontraditional research locations such as Poland (27.3%) and Mexico (14.1%). Despite patient enrolment efforts, the involvement of researchers from nontraditional locations in trial management as measured by their contribution to manuscript writing is modest. This division of labor has

  6. Quality of clinical trials: A moving target

    Science.gov (United States)

    Bhatt, Arun

    2011-01-01

    Quality of clinical trials depends on data integrity and subject protection. Globalization, outsourcing and increasing complexicity of clinical trials have made the target of achieving global quality challenging. The quality, as judged by regulatory inspections of the investigator sites, sponsors/contract research organizations and Institutional Review Board, has been of concern to the US Food and Drug Administration, as there has been hardly any change in frequency and nature of common deficiencies. To meet the regulatory expectations, the sponsors need to improve quality by developing systems with specific standards for each clinical trial process. The quality systems include: personnel roles and responsibilities, training, policies and procedures, quality assurance and auditing, document management, record retention, and reporting and corrective and preventive action. With an objective to improve quality, the FDA has planned new inspection approaches such as risk-based inspections, surveillance inspections, real-time oversight, and audit of sponsor quality systems. The FDA has partnered with Duke University for Clinical Trials Transformation Initiative, which will conduct research projects on design principles, data quality and quantity including monitoring, study start-up, and adverse event reporting. These recent initiatives will go a long way in improving quality of clinical trials. PMID:22145122

  7. Quality of clinical trials: A moving target

    Directory of Open Access Journals (Sweden)

    Arun Bhatt

    2011-01-01

    Full Text Available Quality of clinical trials depends on data integrity and subject protection. Globalization, outsourcing and increasing complexicity of clinical trials have made the target of achieving global quality challenging. The quality, as judged by regulatory inspections of the investigator sites, sponsors/contract research organizations and Institutional Review Board, has been of concern to the US Food and Drug Administration, as there has been hardly any change in frequency and nature of common deficiencies. To meet the regulatory expectations, the sponsors need to improve quality by developing systems with specific standards for each clinical trial process. The quality systems include: personnel roles and responsibilities, training, policies and procedures, quality assurance and auditing, document management, record retention, and reporting and corrective and preventive action. With an objective to improve quality, the FDA has planned new inspection approaches such as risk-based inspections, surveillance inspections, real-time oversight, and audit of sponsor quality systems. The FDA has partnered with Duke University for Clinical Trials Transformation Initiative, which will conduct research projects on design principles, data quality and quantity including monitoring, study start-up, and adverse event reporting. These recent initiatives will go a long way in improving quality of clinical trials.

  8. One Year Clinical Outcomes of Renal Artery Stenting: The Results of ODORI Registry

    International Nuclear Information System (INIS)

    Sapoval, M.; Tamari, I.; Goffette, P.; Downes, M.; Senechal, Q.; Fanelli, F.; Reimer, P.; Negaiwi, Z.; Cassin, P. De; Heye, S.; Korobov, V.; Tsetis, D.; Abada, H.

    2010-01-01

    The safety, efficacy and long term clinical benefits of renal artery revascularization by stenting are still a matter of debate. The aim of our study was to define the safety and efficacy of renal artery stenting with the Tsunami peripheral stent (Terumo Corporation, Tokyo, Japan). The ODORI was a prospective, multicentre registry which enrolled 251 consecutive patients, (276 renal arteries) in 36 centres across Europe. The primary endpoint was acute procedural success defined as <30% residual stenosis after stent placement. Secondary endpoints included major adverse events, blood pressure control, serum creatinine level, and target lesion revascularization (TLR) at 6 and 12 months. Patients were 70 ± 10 years old, 59% were male, 33% had diabetes, and 96% hypertension. The main indications for renal stent implantation were hypertension in 83% and renal salvage in 39%. Direct stent implantation was performed in 76% of the cases. Acute success rate was 100% with residual stenosis of 2.5 ± 5.4%. Systolic/diastolic blood pressure decreased from a mean of 171/89 at baseline to 142/78 mmHg at 6 months (p < 0.0001 vs. baseline), and 141/80 mmHg at 12 months (p < 0.0001 vs. baseline). Mean serum creatinine concentration did not change significantly in the total population. However, there was significant improvement in the highest tercile (from 283 μmol/l at baseline to 205 and 209 μmol/l at 6 and 12 months respectively). At 12-months, rates of restenosis and TLR were 6.6 and 0.8% respectively. The 12 month cumulative rate of all major clinical adverse events was 6.4% while the rate of device or procedure related events was 2.4%. In hypertensive patients with atherosclerotic renal artery stenosis Tsunami peripheral balloon-expandable stent provides a safe revascularization strategy, with a potential beneficial impact on hypertension control and renal function in the highest risk patients.

  9. Marketing and clinical trials: a case study

    OpenAIRE

    Entwistle Vikki A; Snowdon Claire; Garcia Jo; Knight Rosemary C; Shakur Haleema; Elbourne Diana R; Roberts Ian; Francis David; McDonald Alison M; Grant Adrian M; Campbell Marion K

    2007-01-01

    Abstract Background Publicly funded clinical trials require a substantial commitment of time and money. To ensure that sufficient numbers of patients are recruited it is essential that they address important questions in a rigorous manner and are managed well, adopting effective marketing strategies. Methods Using methods of analysis drawn from management studies, this paper presents a structured assessment framework or reference model, derived from a case analysis of the MRC's CRASH trial, o...

  10. Update on clinical trials in Dysphagia.

    Science.gov (United States)

    Logemann, Jeri A

    2006-04-01

    Randomized clinical trials (RCTs) are often known as the gold standard in treatment efficacy studies. This article defines the characteristics of RCTs and the factors that investigators must consider in designing clinical trials in dysphagia. Design issues unique to behavioral treatments often used in dysphagia are discussed. Ongoing RCTs in dysphagia are described including studies of (1) the effectiveness of the Shaker exercise versus standardized treatment in patients with severe dysphagia resulting from stroke or treatment for head and neck cancer who have been nonoral for at least three months; (2) the comparative effects of nectar- and honey-thickened liquids versus chin tuck posture and in patients with dementia or Parkinson's disease with or without dementia who aspirate on thin liquids; and (3) the comparative effects of muscle exercise versus sensory postural therapy for dysphagia resulting from treatment for head and neck cancer. Issues in generalizing from the results of clinical trials are also described.

  11. Industry funded clinical trials: bias and quality.

    Science.gov (United States)

    Del Parigi, Angelo

    2012-01-01

    The quality of the clinical data supporting the development and ultimately the approval for medical use of new drugs is often challenged. Many share the perception that the business goals of the pharmaceutical industry overrule the best scientific efforts to accrue critical knowledge on a new molecule, in order to inform investment of resources, regulatory approvals and appropriate use by patients. Despite this common belief, few scientists have attempted to assess objectively the quality of industry funded (IF) clinical trials by measuring it and comparing it with non-industry funded (NIF) clinical trials in a data-driven fashion. Overall, the average quality of IF clinical research has been reported to be higher than the quality of NIF clinical research.

  12. Control of anxiety through music in a head and neckoutpatient clinic: a randomized clinical trial

    Directory of Open Access Journals (Sweden)

    Mariana Alves Firmeza

    Full Text Available Abstract OBJECTIVE Evaluating the effectiveness of a musical intervention in reducing anxiety and vital parameters in people suffering from head and neck cancer. METHOD A randomized controlled clinical trial, performed in a head and neck outpatient clinic with 40 participants, subdivided into two groups (intervention and control.The classicalmusic“Spring” from The Four Seasons by Vivaldi was used as an intervention.The State-Trait Anxiety Inventory (STAI was used as the data collectioninstrument,along with an inventory of socio-demographic and clinical data. Student'st-test was used to verify intragroup and intergroup statistical significance. RESULTS Participants presented a statistically significant reduction in levels of perceived anxiety (t= 12.68; p<0.001,as well as blood pressure levels (t = 4.56; p<0.001; pulse (t = 6.15; p<0.001 and respiratory rate (t = 5.10; p<0.001. CONCLUSION Music has proven to be an effective non-pharmacological therapeutic resource in managinganxiety in an outpatient setting for people with cancer, as well as in reducing blood pressure, pulse and respiratory rate. Brazilian Registry of Clinical Trials: RBR-7W4YJJ

  13. Genomic sequencing in clinical trials

    OpenAIRE

    Mestan, Karen K; Ilkhanoff, Leonard; Mouli, Samdeep; Lin, Simon

    2011-01-01

    Abstract Human genome sequencing is the process by which the exact order of nucleic acid base pairs in the 24 human chromosomes is determined. Since the completion of the Human Genome Project in 2003, genomic sequencing is rapidly becoming a major part of our translational research efforts to understand and improve human health and disease. This article reviews the current and future directions of clinical research with respect to genomic sequencing, a technology that is just beginning to fin...

  14. Clinical trials in male hormonal contraception.

    Science.gov (United States)

    Nieschlag, Eberhard

    2010-11-01

    Research has established the principle of hormonal male contraception based on suppression of gonadotropins and spermatogenesis. All hormonal male contraceptives use testosterone, but only in East Asian men can testosterone alone suppress spermatogenesis to a level compatible with contraceptive protection. In Caucasians, additional agents are required of which progestins are favored. Clinical trials concentrate on testosterone combined with norethisterone, desogestrel, etonogestrel or depot-medroxyprogesterone acetate. The first randomized, placebo-controlled clinical trial performed by the pharmaceutical industry demonstrated the effectiveness of a combination of testosterone undecanoate and etonogestrel in suppressing spermatogenesis in volunteers. Copyright © 2010 Elsevier Inc. All rights reserved.

  15. Clinical and demographic features of vertigo: findings from the REVERT registry

    Directory of Open Access Journals (Sweden)

    Sam eAgus

    2013-05-01

    Full Text Available IntroductionDespite being a common disease, data on vertigo management in a real-world setting are scarce. AimsTo provide information on the vertigo and its management in a real-world setting.Materials and MethodsData were collected from 4,294 patients with vertigo in 13 countries over 28 months via a multi-national, non-interventional observational study (the so-called REVERT registry. Data included medical history and details of anti-vertigo therapy. ‘Clinical global impression’ (CGI of severity (CGI-S was assessed at baseline (V1 and then at 6 months follow-up (V2 along with CGI change (CGI-C. All variables were analysed descriptively. ResultsThe majority of patients were female, >40 years of age, and almost half had co-morbid cardiovascular disease. Diagnoses were split into 4 categories: 37.2% ‘other vertigo of peripheral vestibular origin’, 26.9% benign paroxysmal positional vertigo (BPPV, 20.5% ‘peripheral vestibular vertigo of unknown origin’ and 15.4% Menière’s disease (MD. Betahistine was the most commonly prescribed therapy prior to and after enrolment, and was followed by piracetam, ginkgo biloba and diuretics. MD had the highest proportion of betahistine treated patients. Almost half of patients were ‘moderately ill’ at V1 based on CGI-S. At V2, patient distribution moved towards ‘less severe illness’ (91.0% improved.The greatest improvements were in the more severely ill, and those with BPPV or ‘other vertigo of peripheral origin’. ConclusionsThere was a reduction in illness severity over the course of the study, some of which is likely to be due to pharmacological intervention. Further studies are needed to confirm these results.

  16. Spinal Cord Injury Clinical Registries: Improving Care across the SCI Care Continuum by Identifying Knowledge Gaps.

    Science.gov (United States)

    Dvorak, Marcel F; Cheng, Christiana L; Fallah, Nader; Santos, Argelio; Atkins, Derek; Humphreys, Suzanne; Rivers, Carly S; White, Barry A B; Ho, Chester; Ahn, Henry; Kwon, Brian K; Christie, Sean; Noonan, Vanessa K

    2017-10-15

    Timely access and ongoing delivery of care and therapeutic interventions is needed to maximize recovery and function after traumatic spinal cord injury (tSCI). To ensure these decisions are evidence-based, access to consistent, reliable, and valid sources of clinical data is required. The Access to Care and Timing Model used data from the Rick Hansen SCI Registry (RHSCIR) to generate a simulation of healthcare delivery for persons after tSCI and to test scenarios aimed at improving outcomes and reducing the economic burden of SCI. Through model development, we identified knowledge gaps and challenges in the literature and current health outcomes data collection throughout the continuum of SCI care. The objectives of this article were to describe these gaps and to provide recommendations for bridging them. Accurate information on injury severity after tSCI was hindered by difficulties in conducting neurological assessments and classifications of SCI (e.g., timing), variations in reporting, and the lack of a validated SCI-specific measure of associated injuries. There was also limited availability of reliable data on patient factors such as multi-morbidity and patient-reported measures. Knowledge gaps related to structures (e.g., protocols) and processes (e.g., costs) at each phase of care have prevented comprehensive evaluation of system performance. Addressing these knowledge gaps will enhance comparative and cost-effectiveness evaluations to inform decision-making and standards of care. Recommendations to do so were: standardize data element collection and facilitate database linkages, validate and adopt more outcome measures for SCI, and increase opportunities for collaborations with stakeholders from diverse backgrounds.

  17. Clinical and Demographic Features of Vertigo: Findings from the REVERT Registry

    Science.gov (United States)

    Agus, Sam; Benecke, Heike; Thum, Cornelia; Strupp, Michael

    2013-01-01

    Introduction: Despite being a common disease, data on vertigo management in a real-world setting are scarce. Aims: To provide information on the vertigo and its management in a real-world setting. Methods: Data were collected from 4,294 patients with vertigo in 13 countries over 28 months via a multi-national, non-interventional observational study (the so-called REVERT registry). Data included medical history and details of anti-vertigo therapy. “Clinical global impression” (CGI) of severity (CGI-S) was assessed at baseline (V1) and then at 6 months follow-up (V2) along with CGI change (CGI-C). All variables were analyzed descriptively. Results: The majority of patients were female, >40 years of age, and almost half had co-morbid cardio-vascular disease. Diagnoses were split into four categories: 37.2% “other vertigo of peripheral vestibular origin,” 26.9% benign paroxysmal positional vertigo (BPPV), 20.5% “peripheral vestibular vertigo of unknown origin,” and 15.4% Ménière’s disease (MD). Betahistine was the most commonly prescribed therapy prior to and after enrollment, and was followed by piracetam, ginkgo biloba, and diuretics. MD had the highest proportion of betahistine treated patients. Almost half of patients were “moderately ill” at V1 based on CGI-S. At V2, patient distribution moved toward “less severe illness” (91.0% improved). The greatest improvements were in the more severely ill, and those with BPPV or “other vertigo of peripheral origin.” Conclusion: There was a reduction in illness severity over the course of the study, some of which is likely to be due to pharmacological intervention. Further studies are needed to confirm these results. PMID:23675366

  18. Clinical trial optimization: Monte Carlo simulation Markov model for planning clinical trials recruitment.

    Science.gov (United States)

    Abbas, Ismail; Rovira, Joan; Casanovas, Josep

    2007-05-01

    The patient recruitment process of clinical trials is an essential element which needs to be designed properly. In this paper we describe different simulation models under continuous and discrete time assumptions for the design of recruitment in clinical trials. The results of hypothetical examples of clinical trial recruitments are presented. The recruitment time is calculated and the number of recruited patients is quantified for a given time and probability of recruitment. The expected delay and the effective recruitment durations are estimated using both continuous and discrete time modeling. The proposed type of Monte Carlo simulation Markov models will enable optimization of the recruitment process and the estimation and the calibration of its parameters to aid the proposed clinical trials. A continuous time simulation may minimize the duration of the recruitment and, consequently, the total duration of the trial.

  19. Utility of registries for post-marketing evaluation of medicines. A survey of Swedish health care quality registries from a regulatory perspective.

    Science.gov (United States)

    Feltelius, Nils; Gedeborg, Rolf; Holm, Lennart; Zethelius, Björn

    2017-06-01

    The aim of this study was to describe content and procedures in some selected Swedish health care quality registries (QRs) of relevance to regulatory decision-making. A workshop was organized with participation of seven Swedish QRs which subsequently answered a questionnaire regarding registry content on drug treatments and outcomes. Patient populations, coverage, data handling and quality control, as well as legal and ethical aspects are presented. Scientific publications from the QRs are used as a complementary measure of quality and scientific relevance. The registries under study collect clinical data of high relevance to regulatory and health technology agencies. Five out of seven registries provide information on the drug of interest. When applying external quality criteria, we found a high degree of fulfillment, although information on medication was not sufficient to answer all questions of regulatory interest. A notable strength is the option for linkage to the Prescribed Drug Registry and to information on education and socioeconomic status. Data on drugs used during hospitalization were also collected to some extent. Outcome measures collected resemble those used in relevant clinical trials. All registries collected patient-reported outcome measures. The number of publications from the registries was substantial, with studies of appropriate design, including randomized registry trials. Quality registries may provide a valuable source of post-marketing data on drug effectiveness, safety, and cost-effectiveness. Closer collaboration between registries and regulators to improve quality and usefulness of registry data could benefit both regulatory utility and value for health care providers.

  20. Comparison of Registered and Reported Outcomes in Randomized Clinical Trials Published in Anesthesiology Journals.

    Science.gov (United States)

    Jones, Philip M; Chow, Jeffrey T Y; Arango, Miguel F; Fridfinnson, Jason A; Gai, Nan; Lam, Kevin; Turkstra, Timothy P

    2017-10-01

    Randomized clinical trials (RCTs) provide high-quality evidence for clinical decision-making. Trial registration is one of the many tools used to improve the reporting of RCTs by reducing publication bias and selective outcome reporting bias. The purpose of our study is to examine whether RCTs published in the top 6 general anesthesiology journals were adequately registered and whether the reported primary and secondary outcomes corresponded to the originally registered outcomes. Following a prespecified protocol, an electronic database was used to systematically screen and extract data from RCTs published in the top 6 general anesthesiology journals by impact factor (Anaesthesia, Anesthesia & Analgesia, Anesthesiology, British Journal of Anaesthesia, Canadian Journal of Anesthesia, and European Journal of Anaesthesiology) during the years 2007, 2010, 2013, and 2015. A manual search of each journal's Table of Contents was performed (in duplicate) to identify eligible RCTs. An adequately registered trial was defined as being registered in a publicly available trials registry before the first patient being enrolled with an unambiguously defined primary outcome. For adequately registered trials, the outcomes registered in the trial registry were compared with the outcomes reported in the article, with outcome discrepancies documented and analyzed by the type of discrepancy. During the 4 years studied, there were 860 RCTs identified, with 102 RCTs determined to be adequately registered (12%). The proportion of adequately registered trials increased over time, with 38% of RCTs being adequately registered in 2015. The most common reason in 2015 for inadequate registration was registering the RCT after the first patient had already been enrolled. Among adequately registered trials, 92% had at least 1 primary or secondary outcome discrepancy. In 2015, 42% of RCTs had at least 1 primary outcome discrepancy, while 90% of RCTs had at least 1 secondary outcome discrepancy

  1. Biomarkers in T cell therapy clinical trials

    Directory of Open Access Journals (Sweden)

    Kalos Michael

    2011-08-01

    Full Text Available Abstract T cell therapy represents an emerging and promising modality for the treatment of both infectious disease and cancer. Data from recent clinical trials have highlighted the potential for this therapeutic modality to effect potent anti-tumor activity. Biomarkers, operationally defined as biological parameters measured from patients that provide information about treatment impact, play a central role in the development of novel therapeutic agents. In the absence of information about primary clinical endpoints, biomarkers can provide critical insights that allow investigators to guide the clinical development of the candidate product. In the context of cell therapy trials, the definition of biomarkers can be extended to include a description of parameters of the cell product that are important for product bioactivity. This review will focus on biomarker studies as they relate to T cell therapy trials, and more specifically: i. An overview and description of categories and classes of biomarkers that are specifically relevant to T cell therapy trials, and ii. Insights into future directions and challenges for the appropriate development of biomarkers to evaluate both product bioactivity and treatment efficacy of T cell therapy trials.

  2. Cross-sectional study of characteristics of clinical registries in Australia: a resource for clinicians and policy makers.

    Science.gov (United States)

    Md Emdadul Hoque, Dewan; Ruseckaite, Rasa; Lorgelly, Paula; McNeil, John J; Evans, Sue M

    2018-04-01

    To investigate the attributes of Australian clinical quality registries (CQR). Survey of 40 CQRs between September 2015 and April 2016. CQR lead investigators/project managers. None. Registry organization, geographical coverage, data quality, management, characteristics, output and outcomes. Of those who responded (34/40; 85.0%), 12 (34.3%) were binational (Australia and New Zealand); 22 (64.7%) were Australian-only registries; and 13 (38.2%) had national coverage. CQRs covered critical care, infection control, cardiovascular diseases, cancer, chronic diseases, procedures and devices, and transplants. Overall, 24/34 CQRs (70.6%) were public sector funded. In total, 14 (41.2%) scored >75% on a composite score developed to assess data quality. Overall, 29/34 (85.3%) produced an annual multi-centred report; only 15/34 (44.1%) produced provider-specific reports. Mortality/survival and quality of life were collected by 82.4 and 32.4% of CQRs, respectively. Most CQRs displayed data in bar/column charts (28/34, 82.4%) and funnel plots (17/34, 50%). Most CQRs adopted an opt-out consent process (n = 17/31; 54.8%). Linear regression indicated that longer duration of CQR was associated with higher data quality (>20 vs 0-5 years coefficient = 4.76, 95% CI: 0.26, 9.26). Opt-in consent was associated with lower data quality (no active consent vs opt-in approval method, coefficient = -5.22, 95% CI: -8.71, -1.72). Six CQRs self-reported having undertaken an economic evaluation of their registry. CQRs varied in geographical coverage; stage of development, approach to recruitment; method and frequency of reporting their output; and data quality assurance. An accreditation system for CQRs would likely assist in recognizing high-quality registries.

  3. Existing data sources for clinical epidemiology: the Danish National Pathology Registry and Data Bank

    DEFF Research Database (Denmark)

    Erichsen, Rune; Lash, Timothy L; Hamilton-Dutoit, Stephen J

    2010-01-01

    Diagnostic histological and cytological specimens are routinely stored in pathology department archives. These biobanks are a valuable research resource for many diseases, particularly if they can be linked to high quality population-based health registries, allowing large retrospective epidemiol......Diagnostic histological and cytological specimens are routinely stored in pathology department archives. These biobanks are a valuable research resource for many diseases, particularly if they can be linked to high quality population-based health registries, allowing large retrospective...... epidemiological studies to be carried out. Such studies are of significant importance, for example in the search for novel prognostic and predictive biomarkers in the era of personalized medicine. Denmark has a wealth of highly-regarded population-based registries that are ideally suited to conduct this type...

  4. Chinese herbal medicine for cancer-related fatigue: a systematic review of randomized clinical trials.

    Science.gov (United States)

    Su, Chun-Xiang; Wang, Li-Qiong; Grant, Suzanne J; Liu, Jian-Ping

    2014-06-01

    To assess the effectiveness and safety of Chinese herbal medicine for the treatment of cancer-related fatigue. We systematically searched seven electronic databases and two trial registries for randomized clinical trials of Chinese herbal medicine for cancer-related fatigue. Two authors independently extracted data and assessed the methodological quality of the included trials using the Cochrane risk of bias tool. Data were synthesized using RevMan 5.2 software. A total of 10 trials involving 751 participants with cancer-related fatigue were identified and the methodological quality of the included trials was generally poor. Chinese herbal medicine used alone or in combination with chemotherapy or supportive care showed significant relief in cancer-related fatigue compared to placebo, chemotherapy or supportive care based on single trials. Chinese herbal medicine plus chemotherapy or supportive care was superior to chemotherapy or supportive care in improving quality of life. Data from one trial demonstrated Chinese herbal medicine exerted a greater beneficial effect on relieving anxiety but no difference in alleviating depression. Seven trials reported adverse events and no severe adverse effects were found in Chinese herbal medicine groups. The findings from limited number of trials suggest that Chinese herbal medicine seems to be effective and safe in the treatment of cancer-related fatigue. However, the current evidence is insufficient to draw a confirmative conclusion due to the poor methodological quality of included trials. Thus, conducting rigorously designed trials on potential Chinese herbal medicine is warranted. Copyright © 2014 Elsevier Ltd. All rights reserved.

  5. Mitochondrial disease patient motivations and barriers to participate in clinical trials.

    Directory of Open Access Journals (Sweden)

    Zarazuela Zolkipli-Cunningham

    Full Text Available Clinical treatment trials are increasingly being designed in primary mitochondrial disease (PMD, a phenotypically and genetically heterogeneous collection of inherited multi- system energy deficiency disorders that lack effective therapy. We sought to identify motivating factors and barriers to clinical trial participation in PMD.A survey study was conducted in two independent mitochondrial disease subject cohorts. A discovery cohort invited subjects with well-defined biochemical or molecularly- confirmed PMD followed at a single medical center (CHOP, n = 30/67 (45% respondents. A replication cohort included self-identified PMD subjects in the Rare Disease Clinical Research Network (RDCRN national contact registry (n = 290/1119 (26% respondents. Five-point Likert scale responses were analyzed using descriptive and quantitative statistics. Experienced and prioritized symptoms for trial participation, and patient attitudes toward detailed aspects of clinical trial drug features and study design.PMD subjects experienced an average of 16 symptoms. Muscle weakness, chronic fatigue, and exercise intolerance were the lead symptoms encouraging trial participation. Motivating trial design factors included a self-administered study drug; vitamin, antioxidant, natural or plant-derivative; pills; daily treatment; guaranteed treatment access during and after study; short travel distances; and late-stage (phase 3 participation. Relative trial participation barriers included a new study drug; discontinuation of current medications; disease progression; daily phlebotomy; and requiring participant payment. Treatment trial type or design preferences were not influenced by population age (pediatric versus adult, prior research trial experience, or disease severity.These data are the first to convey clear PMD subject preferences and priorities to enable improved clinical treatment trial design that cuts across the complex diversity of disease. Partnering with rare

  6. Infrastructure for Clinical Trials in Duchenne Dystrophy

    Science.gov (United States)

    2010-09-13

    A Zimmerman, T Duong, J Florence and the CINRG Investigators. Pulmonary Function Characteristics of Boys with Duchenne and Becker Muscular Dystrophy ...designated CINRG site staff 1. Has the participant been clinically diagnosed with Limb-Girdle or Becker muscular dystrophy ? LGMD BMD 2. Was...Number: W81XWH-09-1-0592 TITLE: CINRG: Infrastructure for Clinical Trials in Duchenne Dystrophy PRINCIPAL INVESTIGATOR: Avital Cnaan, PhD

  7. The HOPE (Helping to Outline Paediatric Eating Disorders) Project: development and debut of a paediatric clinical eating disorder registry

    Science.gov (United States)

    2013-01-01

    Background The HOPE (Helping to Outline Paediatric Eating Disorders) Project is an ongoing registry study made up of a sequential cross-sectional sample prospectively recruited over 17 years, and is designed to answer empirical questions about paediatric eating disorders. This paper introduces the HOPE Project, describes the registry sample to-date, and discusses future directions and challenges and accomplishments. The project and clinical service were established in a tertiary academic hospital in Western Australia in 1996 with a service development grant. Research processes were inbuilt into the initial protocols and data collection was maintained in the following years. Recognisable progress with the research agenda accelerated only when dedicated research resources were obtained. The registry sample consists of consecutive children and adolescents assessed at the eating disorder program from 1996 onward. Standardised multidisciplinary data collected from family intake interview, parent and child clinical interviews, medical review, parent, child and teacher psychometric assessments, and inpatient admission records populate the HOPE Project database. Results The registry database to-date contains 941 assessments, of whom 685 met DSM-IV diagnostic criteria for an eating disorder at admission. The majority of the sample were females (91%) from metropolitan Perth (83%). The cases with eating disorders consist of eating disorders not otherwise specified (68%), anorexia nervosa (25%) and bulimia nervosa (7%). Among those with eating disorders, a history of weight loss since illness onset was almost universal (96%) with fear of weight gain (71%) common, and the median duration of illness was 8 months. Conclusions Over the next five years and more, we expect that the HOPE Project will make a strong scientific contribution to paediatric eating disorders research and will have important real-world applications to clinical practice and policy as the research unfolds

  8. Smart Technology in Lung Disease Clinical Trials.

    Science.gov (United States)

    Geller, Nancy L; Kim, Dong-Yun; Tian, Xin

    2016-01-01

    This article describes the use of smart technology by investigators and patients to facilitate lung disease clinical trials and make them less costly and more efficient. By "smart technology" we include various electronic media, such as computer databases, the Internet, and mobile devices. We first describe the use of electronic health records for identifying potential subjects and then discuss electronic informed consent. We give several examples of using the Internet and mobile technology in clinical trials. Interventions have been delivered via the World Wide Web or via mobile devices, and both have been used to collect outcome data. We discuss examples of new electronic devices that recently have been introduced to collect health data. While use of smart technology in clinical trials is an exciting development, comparison with similar interventions applied in a conventional manner is still in its infancy. We discuss advantages and disadvantages of using this omnipresent, powerful tool in clinical trials, as well as directions for future research. Published by Elsevier Inc.

  9. Lung Cancer Clinical Trials: Advances in Immunotherapy

    Science.gov (United States)

    New treatments for lung cancer and aspects of joining a clinical trial are discussed in this 30-minute Facebook Live event, hosted by NCI’s Dr. Shakun Malik, head of thoracic oncology therapeutics, and Janet Freeman-Daily, lung cancer patient activist and founding member of #LCSM.

  10. Quality assurance of asthma clinical trials.

    Science.gov (United States)

    Malmstrom, Kerstin; Peszek, Iza; Al Botto; Lu, Susan; Enright, Paul L; Reiss, Theodore F

    2002-04-01

    Accuracy and repeatability of spirometry measurements are essential to obtain reliable efficacy data in randomized asthma clinical trials. We report our experience with a centralized spirometry quality assurance program that we implemented in our phase III asthma trials. Six asthma trials of 4 to 21 weeks in duration were conducted at 232 clinical centers in 31 countries. Approximately 23,100 prebronchodilator and 13,700 postbronchodilator spirometry tests were collected from 2523 adult and 336 pediatric asthmatic patients. The program used a standard spirometer (the Renaissance spirometry system) with maneuver quality messages and automated quality grading of the spirometry tests. Each clinical center transmitted spirometry data weekly to a central database, where uniform monitoring of data quality was performed and feedback was provided in weekly quality reports. Seventy-nine percent of all patients performed spirometry sessions with quality that either met or exceeded American Thoracic Society standards and improved over time. Good-quality spirometry was associated with (1) less severe asthma; (2) active treatment; (3) infrequent nocturnal awakenings; (4) age above 15 years; and (5) low body weight. Maneuver-induced bronchospasm was rare. Good-quality spirometry was observed in multicenter asthma clinical trials that employed a standard spirometer and continuous monitoring. Both within- and between-patient variability decreased. Spirometry quality improved with time as study participants and technicians gained experience.

  11. ORIGINAL ARTICLES Pharmacologically active: clinical trials and ...

    African Journals Online (AJOL)

    2008-01-22

    Jan 22, 2008 ... companies to manufacture pharmaceuticals, 24 to carry out quality control and ... represents a 3% real growth from 2004/2005, it represents a slight decline from ... manufacturer for the pharmas, or can it leverage strengths in medical ... increased clinical trials activity, R&D investment is too low to make it a ...

  12. Pharmacoligaclly Active: Clinical Trials and the Pharmaceuticals ...

    African Journals Online (AJOL)

    Multinational pharmaceutical companies ('pharmas') import and produce pharmaceuticals and also conduct clinical trials which are an important aspect of research and development (R&D). This may raise the question: Is South Africa a guinea pig for the pharmas? The Department of Trade and Industry National Industrial ...

  13. Limited accessibility to designs and results of Japanese large-scale clinical trials for cardiovascular diseases.

    Science.gov (United States)

    Sawata, Hiroshi; Ueshima, Kenji; Tsutani, Kiichiro

    2011-04-14

    Clinical evidence is important for improving the treatment of patients by health care providers. In the study of cardiovascular diseases, large-scale clinical trials involving thousands of participants are required to evaluate the risks of cardiac events and/or death. The problems encountered in conducting the Japanese Acute Myocardial Infarction Prospective (JAMP) study highlighted the difficulties involved in obtaining the financial and infrastructural resources necessary for conducting large-scale clinical trials. The objectives of the current study were: 1) to clarify the current funding and infrastructural environment surrounding large-scale clinical trials in cardiovascular and metabolic diseases in Japan, and 2) to find ways to improve the environment surrounding clinical trials in Japan more generally. We examined clinical trials examining cardiovascular diseases that evaluated true endpoints and involved 300 or more participants using Pub-Med, Ichushi (by the Japan Medical Abstracts Society, a non-profit organization), websites of related medical societies, the University Hospital Medical Information Network (UMIN) Clinical Trials Registry, and clinicaltrials.gov at three points in time: 30 November, 2004, 25 February, 2007 and 25 July, 2009. We found a total of 152 trials that met our criteria for 'large-scale clinical trials' examining cardiovascular diseases in Japan. Of these, 72.4% were randomized controlled trials (RCTs). Of 152 trials, 9.2% of the trials examined more than 10,000 participants, and 42.8% examined between 1,000 and 10,000 participants. The number of large-scale clinical trials markedly increased from 2001 to 2004, but suddenly decreased in 2007, then began to increase again. Ischemic heart disease (39.5%) was the most common target disease. Most of the larger-scale trials were funded by private organizations such as pharmaceutical companies. The designs and results of 13 trials were not disclosed. To improve the quality of clinical

  14. Limited accessibility to designs and results of Japanese large-scale clinical trials for cardiovascular diseases

    Directory of Open Access Journals (Sweden)

    Tsutani Kiichiro

    2011-04-01

    Full Text Available Abstract Background Clinical evidence is important for improving the treatment of patients by health care providers. In the study of cardiovascular diseases, large-scale clinical trials involving thousands of participants are required to evaluate the risks of cardiac events and/or death. The problems encountered in conducting the Japanese Acute Myocardial Infarction Prospective (JAMP study highlighted the difficulties involved in obtaining the financial and infrastructural resources necessary for conducting large-scale clinical trials. The objectives of the current study were: 1 to clarify the current funding and infrastructural environment surrounding large-scale clinical trials in cardiovascular and metabolic diseases in Japan, and 2 to find ways to improve the environment surrounding clinical trials in Japan more generally. Methods We examined clinical trials examining cardiovascular diseases that evaluated true endpoints and involved 300 or more participants using Pub-Med, Ichushi (by the Japan Medical Abstracts Society, a non-profit organization, websites of related medical societies, the University Hospital Medical Information Network (UMIN Clinical Trials Registry, and clinicaltrials.gov at three points in time: 30 November, 2004, 25 February, 2007 and 25 July, 2009. Results We found a total of 152 trials that met our criteria for 'large-scale clinical trials' examining cardiovascular diseases in Japan. Of these, 72.4% were randomized controlled trials (RCTs. Of 152 trials, 9.2% of the trials examined more than 10,000 participants, and 42.8% examined between 1,000 and 10,000 participants. The number of large-scale clinical trials markedly increased from 2001 to 2004, but suddenly decreased in 2007, then began to increase again. Ischemic heart disease (39.5% was the most common target disease. Most of the larger-scale trials were funded by private organizations such as pharmaceutical companies. The designs and results of 13 trials were not

  15. Establishing Evidence-Based Indications for Proton Therapy: An Overview of Current Clinical Trials

    Energy Technology Data Exchange (ETDEWEB)

    Mishra, Mark V., E-mail: mmishra@umm.edu [Department of Radiation Oncology, University of Maryland School of Medicine, Baltimore, Maryland (United States); Aggarwal, Sameer [Department of Internal Medicine, University of Maryland School of Medicine, Baltimore, Maryland (United States); Bentzen, Soren M. [Department of Epidemiology and Public Health, University of Maryland School of Medicine, Baltimore, Maryland (United States); Knight, Nancy [Department of Radiation Oncology, University of Maryland School of Medicine, Baltimore, Maryland (United States); Mehta, Minesh P. [Miami Cancer Institute at Baptist Health South Florida, Miami, Florida (United States); Regine, William F. [Department of Radiation Oncology, University of Maryland School of Medicine, Baltimore, Maryland (United States)

    2017-02-01

    Purpose: To review and assess ongoing proton beam therapy (PBT) clinical trials and to identify major gaps. Methods and Materials: Active PBT clinical trials were identified from (clinicaltrials.gov) and the World Health Organization International Clinical Trials Platform Registry. Data on clinical trial disease site, age group, projected patient enrollment, expected start and end dates, study type, and funding source were extracted. Results: A total of 122 active PBT clinical trials were identified, with target enrollment of >42,000 patients worldwide. Ninety-six trials (79%), with a median planned sample size of 68, were classified as interventional studies. Observational studies accounted for 21% of trials but 71% (n=29,852) of planned patient enrollment. The most common PBT clinical trials focus on gastrointestinal tract tumors (21%, n=26), tumors of the central nervous system (15%, n=18), and prostate cancer (12%, n=15). Five active studies (lung, esophagus, head and neck, prostate, breast) will randomize patients between protons and photons, and 3 will randomize patients between protons and carbon ion therapy. Conclusions: The PBT clinical trial portfolio is expanding rapidly. Although the majority of ongoing studies are interventional, the majority of patients will be accrued to observational studies. Future efforts should focus on strategies to encourage optimal patient enrollment and retention, with an emphasis on randomized, controlled trials, which will require support from third-party payers. Results of ongoing PBT studies should be evaluated in terms of comparative effectiveness, as well as incremental effectiveness and value offered by PBT in comparison with conventional radiation modalities.

  16. Clinical trial quality: From supervision to collaboration and beyond.

    Science.gov (United States)

    Meeker-O'Connell, Ann; Glessner, Coleen

    2018-02-01

    Over the past decade, clinical trial quality has evolved from an after-the-fact, reactive activity to one focused on the important work of evidence generation from well-designed trials. This article explores the role the Clinical Trials Transformation Initiative has played in advancing quality as a core element of clinical trial design, through project work that initially focused on monitoring but evolved into a holistic, prospective, and comprehensive quality by design approach to clinical trial design and conduct.

  17. Quality of registration for clinical trials published in emergency medicine journals.

    Science.gov (United States)

    Jones, Christopher W; Platts-Mills, Timothy F

    2012-10-01

    In 2005, the International Committee of Medical Journal Editors established clinical trial registration as a requirement for articles submitted to member journals, with the goal of improving the transparency of clinical research. The objective of this study is to characterize the registration of clinical trials published in emergency medicine journals. Randomized trials involving human subjects and published between June 1, 2008, and May 31, 2011 in the 5 emergency medicine journals with the highest impact factors were included. We assessed the clarity of registered primary outcomes, timing of registration relative to patient enrollment, and consistency between registered and published outcomes. Of the 123 trials included, registry entries were identified for 57 (46%). Of the 57 registered studies, 45 (79%) were registered after the initiation of subject enrollment, 9 (16%) had registered outcomes that were unclear, and 26 (46%) had discrepancies between registered and published outcomes. Only 5 studies were registered before patient enrollment with a clear primary outcome that was consistent with the published primary outcome. Annals of Emergency Medicine was the only journal in which the majority of trials were registered. Current compliance with clinical trial registration guidelines is poor among trials published in emergency medicine journals. Copyright © 2012. Published by Mosby, Inc.

  18. Portfolio of prospective clinical trials including brachytherapy: an analysis of the ClinicalTrials.gov database.

    Science.gov (United States)

    Cihoric, Nikola; Tsikkinis, Alexandros; Miguelez, Cristina Gutierrez; Strnad, Vratislav; Soldatovic, Ivan; Ghadjar, Pirus; Jeremic, Branislav; Dal Pra, Alan; Aebersold, Daniel M; Lössl, Kristina

    2016-03-22

    To evaluate the current status of prospective interventional clinical trials that includes brachytherapy (BT) procedures. The records of 175,538 (100 %) clinical trials registered at ClinicalTrials.gov were downloaded on September 2014 and a database was established. Trials using BT as an intervention were identified for further analyses. The selected trials were manually categorized according to indication(s), BT source, applied dose rate, primary sponsor type, location, protocol initiator and funding source. We analyzed trials across 8 available trial protocol elements registered within the database. In total 245 clinical trials were identified, 147 with BT as primary investigated treatment modality and 98 that included BT as an optional treatment component or as part of the standard treatment. Academic centers were the most frequent protocol initiators in trials where BT was the primary investigational treatment modality (p < 0.01). High dose rate (HDR) BT was the most frequently investigated type of BT dose rate (46.3 %) followed by low dose rate (LDR) (42.0 %). Prostate was the most frequently investigated tumor entity in trials with BT as the primary treatment modality (40.1 %) followed by breast cancer (17.0 %). BT was rarely the primary investigated treatment modality for cervical cancer (6.8 %). Most clinical trials using BT are predominantly in early phases, investigator-initiated and with low accrual numbers. Current investigational activities that include BT mainly focus on prostate and breast cancers. Important questions concerning the optimal usage of BT will not be answered in the near future.

  19. Portfolio of prospective clinical trials including brachytherapy: an analysis of the ClinicalTrials.gov database

    International Nuclear Information System (INIS)

    Cihoric, Nikola; Tsikkinis, Alexandros; Miguelez, Cristina Gutierrez; Strnad, Vratislav; Soldatovic, Ivan; Ghadjar, Pirus; Jeremic, Branislav; Dal Pra, Alan; Aebersold, Daniel M.; Lössl, Kristina

    2016-01-01

    To evaluate the current status of prospective interventional clinical trials that includes brachytherapy (BT) procedures. The records of 175,538 (100 %) clinical trials registered at ClinicalTrials.gov were downloaded on September 2014 and a database was established. Trials using BT as an intervention were identified for further analyses. The selected trials were manually categorized according to indication(s), BT source, applied dose rate, primary sponsor type, location, protocol initiator and funding source. We analyzed trials across 8 available trial protocol elements registered within the database. In total 245 clinical trials were identified, 147 with BT as primary investigated treatment modality and 98 that included BT as an optional treatment component or as part of the standard treatment. Academic centers were the most frequent protocol initiators in trials where BT was the primary investigational treatment modality (p < 0.01). High dose rate (HDR) BT was the most frequently investigated type of BT dose rate (46.3 %) followed by low dose rate (LDR) (42.0 %). Prostate was the most frequently investigated tumor entity in trials with BT as the primary treatment modality (40.1 %) followed by breast cancer (17.0 %). BT was rarely the primary investigated treatment modality for cervical cancer (6.8 %). Most clinical trials using BT are predominantly in early phases, investigator-initiated and with low accrual numbers. Current investigational activities that include BT mainly focus on prostate and breast cancers. Important questions concerning the optimal usage of BT will not be answered in the near future. The online version of this article (doi:10.1186/s13014-016-0624-8) contains supplementary material, which is available to authorized users

  20. [Ethical principles of clinical trials in minors].

    Science.gov (United States)

    Koch, H J; Raschka, C

    2002-12-05

    Clinical trials in volunteers and patients are essential to ensure rational treatment of patients. As a rule, drugs are routinely developed for adults, but children are excluded. A major reason for this restriction are ethical justifications, in particular the lack of autonomy on the part of children. The principle of fairness, however, requires that everyone should benefit from progress. Industry, science and society are therefore called upon to find ways of making available safe and adequate treatment for children as quickly as possible, by defining the required conditions for pediatric clinical trials. Important principles are minimal risk, minimal invasivity, rapid decision-making, and careful documentation of trial results. Dynamic ethical principles, such as autonomy and competence in adolescents must be considered on equal footing with existing international GCP guidelines. Aspects of child psychology indicate that the autonomy of adolescents should be respected. Where economic incentives for such trials are absent, for example, in the case of non-pharmacological problems, pediatric trials must be considered a task for society as a whole.

  1. Existing data sources for clinical epidemiology: the Danish National Pathology Registry and Data Bank

    DEFF Research Database (Denmark)

    Erichsen, Rune; Lash, Timothy L; Hamilton-Dutoit, Stephen J

    2010-01-01

    of epidemiological research. We describe two recent additions to these databases: the Danish National Pathology Registry (DNPR) and its underlying national online registration database, the Danish Pathology Data Bank (DPDB). The DNPR and the DPDB contain detailed nationwide records of all pathology specimens...

  2. A machine learning approach to identify clinical trials involving nanodrugs and nanodevices from ClinicalTrials.gov.

    Directory of Open Access Journals (Sweden)

    Diana de la Iglesia

    Full Text Available Clinical Trials (CTs are essential for bridging the gap between experimental research on new drugs and their clinical application. Just like CTs for traditional drugs and biologics have helped accelerate the translation of biomedical findings into medical practice, CTs for nanodrugs and nanodevices could advance novel nanomaterials as agents for diagnosis and therapy. Although there is publicly available information about nanomedicine-related CTs, the online archiving of this information is carried out without adhering to criteria that discriminate between studies involving nanomaterials or nanotechnology-based processes (nano, and CTs that do not involve nanotechnology (non-nano. Finding out whether nanodrugs and nanodevices were involved in a study from CT summaries alone is a challenging task. At the time of writing, CTs archived in the well-known online registry ClinicalTrials.gov are not easily told apart as to whether they are nano or non-nano CTs-even when performed by domain experts, due to the lack of both a common definition for nanotechnology and of standards for reporting nanomedical experiments and results.We propose a supervised learning approach for classifying CT summaries from ClinicalTrials.gov according to whether they fall into the nano or the non-nano categories. Our method involves several stages: i extraction and manual annotation of CTs as nano vs. non-nano, ii pre-processing and automatic classification, and iii performance evaluation using several state-of-the-art classifiers under different transformations of the original dataset.The performance of the best automated classifier closely matches that of experts (AUC over 0.95, suggesting that it is feasible to automatically detect the presence of nanotechnology products in CT summaries with a high degree of accuracy. This can significantly speed up the process of finding whether reports on ClinicalTrials.gov might be relevant to a particular nanoparticle or nanodevice

  3. New EORTC clinical trials for BNCT

    International Nuclear Information System (INIS)

    Hideghety, K.; Moss, R.; Vries, M. de

    2000-01-01

    Due to ethical reasons, a separated optimization of the two components of BNCT in the frame of clinical investigations can only be performed applying the whole binary system. The ongoing trial at HFR (High Flux Reactor Petten) has proven the feasibility of BNCT under defined conditions. On that basis the European Commission supported a comprehensive research project on boron imaging including three further clinical studies. In the first trial the boron uptake related to the blood boron concentration and surrounding normal tissue in various solid tumours will be examined using BSH (Sodiumborocaptate), BPA (Boronophenylalanine) or both in order to explore tumour entities, which may gain benefit from BNCT. The major objectives of the second trial are to define the maximum tolerated single and cumulative dose, and the dose limiting toxicity of BSH. The third clinical trial, a phase II study is designed to evaluate the anti-tumour effect of fractionated BNCT at the Petten treatment facility against cerebral metastasis of malignant melanoma using BPA. (author)

  4. Using e-technologies in clinical trials.

    Science.gov (United States)

    Rosa, Carmen; Campbell, Aimee N C; Miele, Gloria M; Brunner, Meg; Winstanley, Erin L

    2015-11-01

    Clinical trials have been slow to incorporate e-technology (digital and electronic technology that utilizes mobile devices or the Internet) into the design and execution of studies. In the meantime, individuals and corporations are relying more on electronic platforms and most have incorporated such technology into their daily lives. This paper provides a general overview of the use of e-technologies in clinical trials research, specifically within the last decade, marked by rapid growth of mobile and Internet-based tools. Benefits of and challenges to the use of e-technologies in data collection, recruitment and retention, delivery of interventions, and dissemination are provided, as well as a description of the current status of regulatory oversight of e-technologies in clinical trials research. As an example of ways in which e-technologies can be used for intervention delivery, a summary of e-technologies for treatment of substance use disorders is presented. Using e-technologies to design and implement clinical trials has the potential to reach a wide audience, making trials more efficient while also reducing costs; however, researchers should be cautious when adopting these tools given the many challenges in using new technologies, as well as threats to participant privacy/confidentiality. Challenges of using e-technologies can be overcome with careful planning, useful partnerships, and forethought. The role of web- and smartphone-based applications is expanding, and the increasing use of those platforms by scientists and the public alike make them tools that cannot be ignored. Published by Elsevier Inc.

  5. Clinical phenotypes and outcome of patients hospitalized for acute heart failure: the ESC Heart Failure Long-Term Registry.

    Science.gov (United States)

    Chioncel, Ovidiu; Mebazaa, Alexandre; Harjola, Veli-Pekka; Coats, Andrew J; Piepoli, Massimo Francesco; Crespo-Leiro, Maria G; Laroche, Cecile; Seferovic, Petar M; Anker, Stefan D; Ferrari, Roberto; Ruschitzka, Frank; Lopez-Fernandez, Silvia; Miani, Daniela; Filippatos, Gerasimos; Maggioni, Aldo P

    2017-10-01

    To identify differences in clinical epidemiology, in-hospital management and 1-year outcomes among patients hospitalized for acute heart failure (AHF) and enrolled in the European Society of Cardiology Heart Failure Long-Term (ESC-HF-LT) Registry, stratified by clinical profile at admission. The ESC-HF-LT Registry is a prospective, observational study collecting hospitalization and 1-year follow-up data from 6629 AHF patients. Among AHF patients enrolled in the registry, 13.2% presented with pulmonary oedema (PO), 2.9% with cardiogenic shock (CS), 61.1% with decompensated heart failure (DHF), 4.8% with hypertensive heart failure (HT-HF), 3.5% with right heart failure (RHF) and 14.4% with AHF and associated acute coronary syndromes (ACS-HF). The 1-year mortality rate was 28.1% in PO, 54.0% in CS, 27.2% in DHF, 12.8% in HT-HF, 34.0% in RHF and 20.6% in ACS-HF patients. When patients were classified by systolic blood pressure (SBP) at initial presentation, 1-year mortality was 34.8% in patients with SBP 140 mmHg. These differences tended to diminish in the months post-discharge, and 1-year mortality for the patients who survived at least 6 months post-discharge did not vary significantly by either clinical profile or SBP classification. Rates of adverse outcomes in AHF remain high, and substantial differences have been found when patients were stratified by clinical profile or SBP. However, patients who survived at least 6 months post-discharge represent a more homogeneous group and their 1-year outcome is less influenced by clinical profile or SBP at admission. © 2017 The Authors. European Journal of Heart Failure © 2017 European Society of Cardiology.

  6. Global women's health: current clinical trials in low- and middle-income countries.

    Science.gov (United States)

    Merriel, A; Harb, H M; Williams, H; Lilford, R; Coomarasamy, A

    2015-01-01

    Clinical trials in low- and middle-income countries (LMICs) are necessary to develop evidence-based approaches to improve women's health. Understanding what research is currently being conducted will allow the identification of research gaps, avoidance of duplication, planning of future studies, collaboration amongst research groups, and geographical targeting for research investments. To provide an overview of active women's health trials in LMICs. The World Health Organization's International Clinical Trials Registry Platform was searched for trials registered between 1 April 2012 and 31 March 2014. Selected trials were randomised, conducted in LMICs, active, and with a women's health intervention or a significant outcome for the woman. Two reviewers extracted data. Analysis included geographical spread, speciality areas, pre-enrolment registration, study size, and funders. Of the 8966 records, 509 were eligible for inclusion. Gynaecology trials made up 57% of the research, whereas the remaining 43% of trials were in obstetrics. Research activity focused on fertility (17%), the antenatal period (15%), benign gynaecology (14%), intrapartum care (9%), and pre-invasive disease and cancers (8%). The majority of trials (84%) took place in middle-income countries (MICs). In low-income countries (LICs) 83% of research investigated obstetrics, and in MICs 60% of research investigated gynaecology. Most trials (80%) had a sample size of 500 or fewer participants. The median size of trials in LICs was 815 compared with 128 in MICs. Pre-enrolment registration occurred in 54% of trials. The majority (62%) of trials were funded locally. Many LMICs are active in women's health research. The majority of registered trials are located in MICs; however, the trials in LICs are often larger. The focus of research in MICs may be driven by local priorities and funding, with fertility being highly researched. In LICs, pregnancy is the focus, perhaps reflecting the international

  7. Changes in the clinical presentation of immunoglobulin A nephropathy: data from the Spanish Registry of Glomerulonephritis.

    Science.gov (United States)

    Gutiérrez, Eduardo; Praga, Manuel; Rivera, Francisco; Sevillano, Angel; Yuste, Claudia; Goicoechea, Marian; López-Gómez, Juan M

    2018-03-01

    Immunoglobulin A nephropathy (IgAN) is the most common glomerulonephritis in the world, but there is little epidemiological data about possible changes in its presentation over the years. Available information about the influence of age on the form of clinical presentation is also scarce. The aim of the study was to analyse all renal biopsies performed between 1994 and 2013 and recorded in the Spanish Registry of Glomerulonephritis with a histological diagnosis of IgAN. The study was divided into five 4-year periods (1994-97, 1998-2001, 2002-05, 2006-09 and 2010-13) and patients were divided into four age groups: ≤16, 17-44, 45-64 and ≥65 years. From 20.974 renal biopsies recorded, 2961 (14.1%) corresponded to IgAN. The prevalence of IgAN remained stable, but a significant increase in age [from 37.6 (SD 17.7) in 1994-97 to 44.9 (SD 16.8) years in 2010-13; P = 0.001] and worse renal function at presentation [from serum creatinine (SCr) 1.9 (SD 1.9) in 1994-97 to 2.3 (SD 2.1) mg/dL in 2010-13; P = 0.001] were observed over the years. Nephrotic-range proteinuria and acute kidney injury (AKI) as forms of presentation were significantly more common among patients ≥65 years (17.7% and 43.2%, respectively) as compared with the other age groups [≤16 (11.4% and 13.1%, respectively), 17-44 (13.1% and 13%, respectively) and 45-64 (12.1% and 21.3%, respectively)]. Blood pressure, SCr and proteinuria were also significantly higher at presentation among elderly patients. Although the prevalence of IgAN in Spain has remained stable over the years, patients are significantly older and present with significantly worse renal function in the last years. The incidence of nephrotic-range proteinuria (17.7%) and AKI (43.2%) as forms of presentation is remarkable among patients ≥65 years of age. © The Author 2017. Published by Oxford University Press on behalf of ERA-EDTA. All rights reserved.

  8. Privacy and confidentiality in pragmatic clinical trials.

    Science.gov (United States)

    McGraw, Deven; Greene, Sarah M; Miner, Caroline S; Staman, Karen L; Welch, Mary Jane; Rubel, Alan

    2015-10-01

    With pragmatic clinical trials, an opportunity exists to answer important questions about the relative risks, burdens, and benefits of therapeutic interventions. However, concerns about protecting the privacy of this information are significant and must be balanced with the imperative to learn from the data gathered in routine clinical practice. Traditional privacy protections for research uses of identifiable information rely disproportionately on informed consent or authorizations, based on a presumption that this is necessary to fulfill ethical principles of respect for persons. But frequently, the ideal of informed consent is not realized in its implementation. Moreover, the principle of respect for persons—which encompasses their interests in health information privacy—can be honored through other mechanisms. Data anonymization also plays a role in protecting privacy but is not suitable for all research, particularly pragmatic clinical trials. In this article, we explore both the ethical foundation and regulatory framework intended to protect privacy in pragmatic clinical trials. We then review examples of novel approaches to respecting persons in research that may have the added benefit of honoring patient privacy considerations. © The Author(s) 2015.

  9. Activating clinical trials: a process improvement approach.

    Science.gov (United States)

    Martinez, Diego A; Tsalatsanis, Athanasios; Yalcin, Ali; Zayas-Castro, José L; Djulbegovic, Benjamin

    2016-02-24

    The administrative process associated with clinical trial activation has been criticized as costly, complex, and time-consuming. Prior research has concentrated on identifying administrative barriers and proposing various solutions to reduce activation time, and consequently associated costs. Here, we expand on previous research by incorporating social network analysis and discrete-event simulation to support process improvement decision-making. We searched for all operational data associated with the administrative process of activating industry-sponsored clinical trials at the Office of Clinical Research of the University of South Florida in Tampa, Florida. We limited the search to those trials initiated and activated between July 2011 and June 2012. We described the process using value stream mapping, studied the interactions of the various process participants using social network analysis, and modeled potential process modifications using discrete-event simulation. The administrative process comprised 5 sub-processes, 30 activities, 11 decision points, 5 loops, and 8 participants. The mean activation time was 76.6 days. Rate-limiting sub-processes were those of contract and budget development. Key participants during contract and budget development were the Office of Clinical Research, sponsors, and the principal investigator. Simulation results indicate that slight increments on the number of trials, arriving to the Office of Clinical Research, would increase activation time by 11 %. Also, incrementing the efficiency of contract and budget development would reduce the activation time by 28 %. Finally, better synchronization between contract and budget development would reduce time spent on batching documentation; however, no improvements would be attained in total activation time. The presented process improvement analytic framework not only identifies administrative barriers, but also helps to devise and evaluate potential improvement scenarios. The strength

  10. Clinical trials integrity: a CRO perspective.

    Science.gov (United States)

    Beach, J E

    2001-01-01

    When contract research organizations (CROs) were first formed, pharmaceutical companies outsourced to them only certain aspects of the conduct of their clinical trials. At first CROs were highly specialized entities, providing, for example, either biostatistical advice, clinical research associates who monitored investigational sites for regulatory compliance, or regulatory support. Gradually, full service CROs emerged, offering a full range of services for clinical trials, including the selection of investigators and investigational sites, assistance with patient recruitment, safety surveillance and reporting, site audits, and data management and biostatistics. This evolving relationship between CROs and the pharmaceutical and medical device industries has resulted in CROs assuming more and more of the regulatory and ethical risks and responsibilities inherent in the conduct of clinical trials. In this full service role, CROs, unlike sponsors, are not interested in the outcome of study, but like sponsors, are subject to heavy regulation by the federal government, must follow applicable state laws, must respect international guidelines, and are obliged to follow their own operating procedures. Moreover, they are judged by the industry on the basis of the scope and quality of services provided, including the degree of adherence to the research protocol, regulatory requirements, and timelines; the quality of the professional working relationships with investigators and institutions, both academic and community-based; and the validity of the data. Further, CROs are subject to comprehensive audits by sponsoring companies, FDA, and other regulatory authorities. For all these reasons, CROs are being tasked with strict vigilance of all stages of the clinical trial process to ensure that the laws, regulations, and industry standards designed for the protection of human subjects and data integrity are maintained.

  11. High Mortality without ESCAPE: The Registry of Heart Failure Patients Receiving Pulmonary Artery Catheters without Randomization

    Science.gov (United States)

    Allen, Larry A.; Rogers, Joseph G.; Warnica, J. Wayne; DiSalvo, Thomas G.; Tasissa, Gudaye; Binanay, Cynthia; O’Connor, Christopher M.; Califf, Robert M.; Leier, Carl V.; Shah, Monica R.; Stevenson, Lynne W.

    2008-01-01

    Background In ESCAPE, there was no difference in days alive and out of the hospital for patients with decompensated heart failure (HF) randomly assigned to therapy guided by pulmonary artery catheter (PAC) plus clinical assessment versus clinical assessment alone. The external validity of these findings is debated. Methods and Results ESCAPE sites enrolled 439 patients receiving PAC without randomization in a prospective registry. Baseline characteristics, pertinent trial exclusion criteria, reasons for PAC use, hemodynamics, and complications were collected. Survival was determined from the National Death Index and the Alberta Registry. On average, registry patients had lower blood pressure, worse renal function, less neurohormonal antagonist therapy, and higher use of intravenous inotropes as compared with trial patients. Although clinical assessment anticipated less volume overload and greater hypoperfusion among the registry population, measured filling pressures were similarly elevated in the registry and trial, while measured perfusion was slightly higher among registry patients. Registry patients had longer hospitalization (13 vs. 6 days, p <0.001) and higher 6-month mortality (34% vs. 20%, p < 0.001) than trial patients. Conclusions The decision to use PAC without randomization identified a population with higher disease severity and risk of mortality. This prospective registry highlights the complex context of patient selection for randomized trials. PMID:18926438

  12. The EuroMyositis registry

    DEFF Research Database (Denmark)

    Lilleker, James B; Vencovsky, Jiri; Wang, Guochun

    2018-01-01

    AIMS: The EuroMyositis Registry facilitates collaboration across the idiopathic inflammatory myopathy (IIM) research community. This inaugural report examines pooled Registry data. METHODS: Cross-sectional analysis of IIM cases from 11 countries was performed. Associations between clinical subtyp...

  13. Disclosure of investigators' recruitment performance in multicenter clinical trials

    DEFF Research Database (Denmark)

    Dal-Ré, Rafael; Moher, David; Gluud, Christian

    2011-01-01

    Rafael Dal-Ré and colleagues argue that the recruitment targets and performance of all site investigators in multi-centre clinical trials should be disclosed in trial registration sites before a trial starts, and when it ends.......Rafael Dal-Ré and colleagues argue that the recruitment targets and performance of all site investigators in multi-centre clinical trials should be disclosed in trial registration sites before a trial starts, and when it ends....

  14. Clinical trials recruitment planning: A proposed framework from the Clinical Trials Transformation Initiative.

    Science.gov (United States)

    Huang, Grant D; Bull, Jonca; Johnston McKee, Kelly; Mahon, Elizabeth; Harper, Beth; Roberts, Jamie N

    2018-03-01

    Patient recruitment is widely recognized as a key determinant of success for clinical trials. Yet a substantial number of trials fail to reach recruitment goals-a situation that has important scientific, financial, ethical, and policy implications. Further, there are important effects on stakeholders who directly contribute to the trial including investigators, sponsors, and study participants. Despite efforts over multiple decades to identify and address barriers, recruitment challenges persist. To advance a more comprehensive approach to trial recruitment, the Clinical Trials Transformation Initiative (CTTI) convened a project team to examine the challenges and to issue actionable, evidence-based recommendations for improving recruitment planning that extend beyond common study-specific strategies. We describe our multi-stakeholder effort to develop a framework that delineates three areas essential to strategic recruitment planning efforts: (1) trial design and protocol development, (2) trial feasibility and site selection, and (3) communication. Our recommendations propose an upstream approach to recruitment planning that has the potential to produce greater impact and reduce downstream barriers. Additionally, we offer tools to help facilitate adoption of the recommendations. We hope that our framework and recommendations will serve as a guide for initial efforts in clinical trial recruitment planning irrespective of disease or intervention focus, provide a common basis for discussions in this area and generate targets for further analysis and continual improvement. Copyright © 2018 The Authors. Published by Elsevier Inc. All rights reserved.

  15. Parents' perceived obstacles to pediatric clinical trial participation: Findings from the clinical trials transformation initiative

    Directory of Open Access Journals (Sweden)

    Rachel G. Greenberg

    2018-03-01

    In order for clinical trial accrual to be successful, parents' priorities and considerations must be a central focus, beginning with initial trial design. The recommendations from the parents who participated in this study can be used to support budget allocations that ensure adequate training of study staff and improved staffing on nights and weekends. Studies of parent responses in outpatient settings and additional inpatient settings will provide valuable information on the consent process from the child's and parent's perspectives. Further studies are needed to explore whether implementation of such strategies will result in improved recruitment for pediatric clinical trials.

  16. Recent clinical trials in valvular heart disease.

    Science.gov (United States)

    Kiss, Daniel; Anwaruddin, Saif

    2017-07-01

    With widespread adoption of transcatheter aortic valve replacement, there has been a change in the approach to management of valvular heart disease. New interest has taken hold in transcatheter therapies for valvular heart disease, as well as research into pathophysiology and progression of disease. Additionally, several key trials have further refined our understanding of surgical management of valvular heart disease. This review will elucidate recent clinical trial data leading to changes in practice. There have been several landmark trials expanding the indications for transcatheter aortic valve replacement. Additionally, although still early, trials are beginning to demonstrate the feasibility and safety of transcatheter mitral valves. Options for transcatheter management of right-sided valvular disease continue to evolve, and these are areas of active investigation. The emergence of novel therapies for valvular heart disease has expanded the management options available, allowing physicians to better individualize treatment of patients with valvular heart disease. This review will focus on the recent (within 2 years) trials in this field of interest.

  17. Type II diabetes and its therapy in clinical practice - results from the standardised non-interventional registry SIRTA.

    Science.gov (United States)

    Gallwitz, B; Kusterer, K; Hildemann, S; Fresenius, K

    2014-12-01

    Modern antidiabetic therapies should achieve low HbA1c values and avoid hypoglycaemic complications. The registry SIRTA included 1522 patients with type II diabetes mellitus (T2DM) from 306 German medical practices. Patients had an HbA1c > 6.5% under the maximum tolerated metformin dose. If required, they received combination therapy with other antidiabetics according to the guideline of the German Diabetes Society [Deutsche Diabetes Gesellschaft (DDG)] or usual medical practice. Patients were followed up for 6 months. The target criteria included the achievement of HbA1c target values and the emergence of severe hypoglycaemic episodes. Most patients (64.0%) were planned to achieve an HbA1c target < 6.5%, the standard target recommended by the 2009 DDG guideline valid throughout the registry. Primarily to reduce the individual risk for hypoglycaemia, 32.4% of patients had a less strict HbA1c-target of 6.5-7.0%. These targets were achieved by 31.3% and 44.3% of patients, respectively. Combination therapies increased from 45% to 56% over the 6 months registry. Four patients had severe hypoglycaemias (0.26%). The registry confirms results from other epidemiologic studies on the therapy of T2DM in everyday practice. The treatment strategies applied effectively reduced blood glucose and avoided severe hypoglycaemias. An early therapy of insufficiently controlled patients with T2DM is important, as lower baseline values facilitated achieving HbA1c targets. © 2014 The Authors. International Journal of Clinical Practice Published by John Wiley & Sons Ltd.

  18. A Clinical Trial on Weight Loss among Truck Drivers

    Directory of Open Access Journals (Sweden)

    MS Thiese

    2015-04-01

    Full Text Available Background: The high prevalence of obesity among commercial truck drivers may be related to sedentary nature of the job, lack of healthy eating choices, and lack of exercise. There may be a link between obesity and crash risk, therefore an intervention to reduce obesity in this population is needed. Objective: To assess feasibility of a 12-week weight loss intervention for truck drivers with a weight loss goal of 10% of initial body weight. Methods: Drivers were selected based on age (≥21 years and body mass index (≥30 kg/m2. The drivers participated in a before-after clinical trial. The intervention included a 12-week program that provided information on healthy diet and increasing exercise, and telephone-based coaching using SMART goals. Outcomes included change from baseline in reported energy intake, measured weight, waist, hip, and neck circumference, blood pressure, and point of care capillary blood lipids and hemoglobin A1c. Exit interviews were conducted to gain insight into driver opinions on the program features and usefulness. This study was registered with the NIH Clinical Trials Registry, number NCT02348983. Results: 12 of 13 drivers completed the study. Weight loss was statistically significant (p=0.03. Reported energy (p=0.005, total fat consumption (p=0.04, and saturated fat consumption (p=0.02 intake were also lower after the 12-week intervention. Drivers attributed their weight loss to health coaching and suggested a longer intervention so that they could reach their goal and become accustomed to the changes. Conclusion: This weight loss intervention is feasible for this difficult population. Additional research is needed to compare this intervention with a control group.

  19. Current concepts in clinical research: web-based, automated, arthroscopic surgery prospective database registry.

    Science.gov (United States)

    Lubowitz, James H; Smith, Patrick A

    2012-03-01

    In 2011, postsurgical patient outcome data may be compiled in a research registry, allowing comparative-effectiveness research and cost-effectiveness analysis by use of Health Insurance Portability and Accountability Act-compliant, institutional review board-approved, Food and Drug Administration-approved, remote, Web-based data collection systems. Computerized automation minimizes cost and minimizes surgeon time demand. A research registry can be a powerful tool to observe and understand variations in treatment and outcomes, to examine factors that influence prognosis and quality of life, to describe care patterns, to assess effectiveness, to monitor safety, and to change provider practice through feedback of data. Registry of validated, prospective outcome data is required for arthroscopic and related researchers and the public to advocate with governments and health payers. The goal is to develop evidence-based data to determine the best methods for treating patients. Copyright © 2012 Arthroscopy Association of North America. Published by Elsevier Inc. All rights reserved.

  20. Differential Globalization of Industry- and Non-Industry-Sponsored Clinical Trials.

    Science.gov (United States)

    Atal, Ignacio; Trinquart, Ludovic; Porcher, Raphaël; Ravaud, Philippe

    2015-01-01

    Mapping the international landscape of clinical trials may inform global health research governance, but no large-scale data are available. Industry or non-industry sponsorship may have a major influence in this mapping. We aimed to map the global landscape of industry- and non-industry-sponsored clinical trials and its evolution over time. We analyzed clinical trials initiated between 2006 and 2013 and registered in the WHO International Clinical Trials Registry Platform (ICTRP). We mapped single-country and international trials by World Bank's income groups and by sponsorship (industry- vs. non- industry), including its evolution over time from 2006 to 2012. We identified clusters of countries that collaborated significantly more than expected in industry- and non-industry-sponsored international trials. 119,679 clinical trials conducted in 177 countries were analysed. The median number of trials per million inhabitants in high-income countries was 100 times that in low-income countries (116.0 vs. 1.1). Industry sponsors were involved in three times more trials per million inhabitants than non-industry sponsors in high-income countries (75.0 vs. 24.5) and in ten times fewer trials in low- income countries (0.08 vs. 1.08). Among industry- and non-industry-sponsored trials, 30.3% and 3.2% were international, respectively. In the industry-sponsored network of collaboration, Eastern European and South American countries collaborated more than expected; in the non-industry-sponsored network, collaboration among Scandinavian countries was overrepresented. Industry-sponsored international trials became more inter-continental with time between 2006 and 2012 (from 54.8% to 67.3%) as compared with non-industry-sponsored trials (from 42.4% to 37.2%). Based on trials registered in the WHO ICTRP we documented a substantial gap between the globalization of industry- and non-industry-sponsored clinical research. Only 3% of academic trials but 30% of industry trials are

  1. Differential Globalization of Industry- and Non-Industry–Sponsored Clinical Trials

    Science.gov (United States)

    Atal, Ignacio; Trinquart, Ludovic; Porcher, Raphaël; Ravaud, Philippe

    2015-01-01

    Background Mapping the international landscape of clinical trials may inform global health research governance, but no large-scale data are available. Industry or non-industry sponsorship may have a major influence in this mapping. We aimed to map the global landscape of industry- and non-industry–sponsored clinical trials and its evolution over time. Methods We analyzed clinical trials initiated between 2006 and 2013 and registered in the WHO International Clinical Trials Registry Platform (ICTRP). We mapped single-country and international trials by World Bank's income groups and by sponsorship (industry- vs. non- industry), including its evolution over time from 2006 to 2012. We identified clusters of countries that collaborated significantly more than expected in industry- and non-industry–sponsored international trials. Results 119,679 clinical trials conducted in 177 countries were analysed. The median number of trials per million inhabitants in high-income countries was 100 times that in low-income countries (116.0 vs. 1.1). Industry sponsors were involved in three times more trials per million inhabitants than non-industry sponsors in high-income countries (75.0 vs. 24.5) and in ten times fewer trials in low- income countries (0.08 vs. 1.08). Among industry- and non-industry–sponsored trials, 30.3% and 3.2% were international, respectively. In the industry-sponsored network of collaboration, Eastern European and South American countries collaborated more than expected; in the non-industry–sponsored network, collaboration among Scandinavian countries was overrepresented. Industry-sponsored international trials became more inter-continental with time between 2006 and 2012 (from 54.8% to 67.3%) as compared with non-industry–sponsored trials (from 42.4% to 37.2%). Conclusions Based on trials registered in the WHO ICTRP we documented a substantial gap between the globalization of industry- and non-industry–sponsored clinical research. Only 3% of

  2. COMBINED ANTIHYPERTENSIVE THERAPY IN REAL CLINICAL PRACTICE. FOCUS ON FIXED COMBINATIONS OF ANTIHYPERTENSIVE DRUGS (According to the Data of Outpatient Registries RECVASA and PROFILE

    Directory of Open Access Journals (Sweden)

    S. Yu. Martsevich

    2017-01-01

    prescribed original AHD; 75% of patients from RECVASA registry received generics. The most popular combinations in both registries were combinations of angiotensin-converting enzyme inhibitors or angiotensin II receptor blockers with thiazide/thiazide-like diuretics. The target level of BP was more often achieved in the patients of the PROFILE registry: 37.6% vs 26.1% in the RECVASA registry.Conclusion. The results of the analysis of the presented registries demonstrate the low frequency of prescribing fixed combinations in real clinical practice, the inertness of physicians in achieving the target BP levels and the low efficiency of antihypertensive therapy.

  3. Registry Assessment of Peripheral Interventional Devices (RAPID) - Registry Assessment of Peripheral Interventional Devices Core Data Elements.

    Science.gov (United States)

    Jones, W Schuyler; Krucoff, Mitchell W; Morales, Pablo; Wilgus, Rebecca W; Heath, Anne H; Williams, Mary F; Tcheng, James E; Marinac-Dabic, J Danica; Malone, Misti L; Reed, Terrie L; Fukaya, Rie; Lookstein, Robert; Handa, Nobuhiro; Aronow, Herbert D; Bertges, Daniel J; Jaff, Michael R; Tsai, Thomas T; Smale, Joshua A; Zaugg, Margo J; Thatcher, Robert J; Cronenwett, Jack L; Nc, Durham; Md, Silver Spring; Japan, Tokyo; Ny, New York; Ri, Providence; Vt, Burlington; Mass, Newton; Colo, Denver; Ariz, Tempe; Calif, Santa Clara; Minn, Minneapolis; Nh, Lebanon

    2018-01-25

    The current state of evaluating patients with peripheral artery disease and more specifically of evaluating medical devices used for peripheral vascular intervention (PVI) remains challenging because of the heterogeneity of the disease process, the multiple physician specialties that perform PVI, the multitude of devices available to treat peripheral artery disease, and the lack of consensus about the best treatment approaches. Because PVI core data elements are not standardized across clinical care, clinical trials, and registries, aggregation of data across different data sources and physician specialties is currently not feasible.Methods and Results:Under the auspices of the U.S. Food and Drug Administration's Medical Device Epidemiology Network initiative-and its PASSION (Predictable and Sustainable Implementation of the National Registries) program, in conjunction with other efforts to align clinical data standards-the Registry Assessment of Peripheral Interventional Devices (RAPID) workgroup was convened. RAPID is a collaborative, multidisciplinary effort to develop a consensus lexicon and to promote interoperability across clinical care, clinical trials, and national and international registries of PVI. The current manuscript presents the initial work from RAPID to standardize clinical data elements and definitions, to establish a framework within electronic health records and health information technology procedural reporting systems, and to implement an informatics-based approach to promote the conduct of pragmatic clinical trials and registry efforts in PVI. Ultimately, we hope this work will facilitate and improve device evaluation and surveillance for patients, clinicians, health outcomes researchers, industry, policymakers, and regulators.

  4. OARSI Clinical Trials Recommendations: Design and conduct of clinical trials for hand osteoarthritis.

    Science.gov (United States)

    Kloppenburg, M; Maheu, E; Kraus, V B; Cicuttini, F; Doherty, M; Dreiser, R-L; Henrotin, Y; Jiang, G-L; Mandl, L; Martel-Pelletier, J; Nelson, A E; Neogi, T; Pelletier, J-P; Punzi, L; Ramonda, R; Simon, L S; Wang, S

    2015-05-01

    Hand osteoarthritis (OA) is a very frequent disease, but yet understudied. However, a lot of works have been published in the past 10 years, and much has been done to better understand its clinical course and structural progression. Despite this new knowledge, few therapeutic trials have been conducted in hand OA. The last OARSI recommendations for the conduct of clinical trials in hand OA dates back to 2006. The present recommendations aimed at updating previous recommendations, by incorporating new data. The purpose of this expert opinion, consensus driven exercise is to provide evidence-based guidance on the design, execution and analysis of clinical trials in hand OA, where published evidence is available, supplemented by expert opinion, where evidence is lacking, to perform clinical trials in hand OA, both for symptom and for structure-modification. They indicate core outcome measurement sets for studies in hand OA, and list the methods and instruments that should be used to measure symptoms or structure. For both symptom- and structure-modification, at least pain, physical function, patient global assessment, HR-QoL, joint activity and hand strength should be assessed. In addition, for structure-modification trials, structural progression should be measured by radiographic changes. We also provide a research agenda listing many unsolved issues that seem to most urgently need to be addressed from the perspective of performing "good" clinical trials in hand OA. These updated OARSI recommendations should allow for better standardizing the conduct of clinical trials in hand OA in the next future. Copyright © 2015 Osteoarthritis Research Society International. Published by Elsevier Ltd. All rights reserved.

  5. Mexican registry of pulmonary hypertension: REMEHIP.

    Science.gov (United States)

    Sandoval Zarate, Julio; Jerjes-Sanchez, Carlos; Ramirez-Rivera, Alicia; Zamudio, Tomas Pulido; Gutierrez-Fajardo, Pedro; Elizalde Gonzalez, Jose; Leon, Mario Seoane Garcia De; Gamez, Miguel Beltran; Abril, Francisco Moreno Hoyos; Michel, Rodolfo Parra; Aguilar, Humberto Garcia

    REMEHIP is a prospective, multicentre registry on pulmonary hypertension. The main objective will be to identify the clinical profile, medical care, therapeutic trends and outcomes in adult and pediatric Mexican patients with well-characterized pulmonary hypertension. REMEHIP a multicenter registry began in 2015 with a planned recruitment time of 12 months and a 4-year follow-up. The study population will comprise a longitudinal cohort study, collecting data on patients with prevalent and incident pulmonary hypertension. Will be included patients of age >2 years and diagnosis of pulmonary hypertension by right heart catheterization within Group 1 and Group 4 of the World Health Organization classification. The structure, data collection and data analysis will be based on quality current recommendations for registries. The protocol has been approved by institutional ethics committees in all participant centers. All patients will sign an informed consent form. Currently in Mexico, there is a need of observational registries that include patients with treatment in the everyday clinical practice so the data could be validated and additional information could be obtained versus the one from the clinical trials. In this way, REMEHIP emerges as a link among randomized clinical trials developed by experts and previous Mexican experience. Copyright © 2016 Instituto Nacional de Cardiología Ignacio Chávez. Publicado por Masson Doyma México S.A. All rights reserved.

  6. OARSI Clinical Trials Recommendations: Soluble biomarker assessments in clinical trials in osteoarthritis.

    Science.gov (United States)

    Kraus, V B; Blanco, F J; Englund, M; Henrotin, Y; Lohmander, L S; Losina, E; Önnerfjord, P; Persiani, S

    2015-05-01

    The objective of this work was to describe requirements for inclusion of soluble biomarkers in osteoarthritis (OA) clinical trials and progress toward OA-related biomarker qualification. The Guidelines for Biomarkers Working Group, representing experts in the field of OA biomarker research from both academia and industry, convened to discuss issues related to soluble biomarkers and to make recommendations for their use in OA clinical trials based on current knowledge and anticipated benefits. This document summarizes current guidance on use of biomarkers in OA clinical trials and their utility at five stages, including preclinical development and phase I to phase IV trials. As demonstrated by this summary, biomarkers can provide value at all stages of therapeutics development. When resources permit, we recommend collection of biospecimens in all OA clinical trials for a wide variety of reasons but in particular, to determine whether biomarkers are useful in identifying those individuals most likely to receive clinically important benefits from an intervention; and to determine whether biomarkers are useful for identifying individuals at earlier stages of OA in order to institute treatment at a time more amenable to disease modification. Copyright © 2015 Osteoarthritis Research Society International. Published by Elsevier Ltd. All rights reserved.

  7. Clinical trials in hospitalized heart failure patients: targeting interventions to optimal phenotypic subpopulations.

    Science.gov (United States)

    Vaduganathan, Muthiah; Butler, Javed; Roessig, Lothar; Fonarow, Gregg C; Greene, Stephen J; Metra, Marco; Cotter, Gadi; Kupfer, Stuart; Zalewski, Andrew; Sato, Naoki; Filippatos, Gerasimos; Gheorghiade, Mihai

    2015-07-01

    With one possible exception, the last decade of clinical trials in hospitalized heart failure (HHF) patients has failed to demonstrate improvement in long-term clinical outcomes. This trend necessitates a need to evaluate optimal drug development strategies and standards of trial conduct. It has become increasingly important to recognize the heterogeneity among HHF patients and the differential characterization of novel drug candidates. Targeting these agents to specific subpopulations may afford optimal net response related to the particular mode of action of the drug. Analyses of previous trials demonstrate profound differences in the baseline characteristics of patients enrolled across global regions and participating sites. Such differences may influence risks for events and interpretation of results. Therefore, the actual execution of trials and the epidemiology of HHF populations at the investigative sites must be taken into consideration. Collaboration among participating sites including the provision of registry data tailored to the planned development program will optimize trial conduct. Observational data prior to study initiation may enable sites to feedback and engage in protocol development to allow for feasible and valid clinical trial conduct. This site-centered, epidemiology-based network environment may facilitate studies in specific patient populations and promote optimal data collection and clear interpretation of drug safety and efficacy. This review summarizes the roundtable discussion held by a multidisciplinary team of representatives from academia, National Institutes of Health, industry, regulatory agencies, payers, and contract and academic research organizations to answer the question: Who should be targeted for novel therapies in HHF?

  8. Pulmonary hypertension registry of Kerala, India (PRO-KERALA) - Clinical characteristics and practice patterns.

    Science.gov (United States)

    Harikrishnan, S; Sanjay, G; Ashishkumar, M; Menon, Jaideep; Rajesh, Gopalan Nair; Kumar, Raman Krishna; George Koshy, A; Attacheril, Thankachan V; George, Raju; Punnoose, Eapen; Ashraf, S M; Arun, S R; Cholakkal, Mohammed; Jeemon, Panniyammakal

    2018-08-15

    Epidemiological data on pulmonary hypertension (PH) are scarce from developing countries including India. We established a multi-center registry of PH, the PRO-KERALA registry, in Kerala, India. Fifty hospitals enrolled consecutive adult (>18 years) patients for one year. Echocardiographic criteria (right ventricular systolic pressure - RVSP > 50 mmHg) or invasively obtained mean pulmonary artery pressure > 25 mmHg was the criteria for entry. There were 2003 patients (52% Women, mean age 56 ± 16.1 years) enrolled. The mean RVSP was 68.2 (SD = 17.9) mmHg. Majority of the study participants (59%) belonged to group 2 of the WHO Nice Classification 2013 (PH secondary to left heart disease). One-fifth (21.2%) belonged to group 1, while 13.3%, 3.8% and 2.4% of the study population belonged to groups 3, 4 and 5 respectively. More than a quarter (27%) reported PH due to left heart disease with valvular disease etiology; while 20.7% had coronary artery disease. The other common etiological factors were chronic obstructive pulmonary disease (10.6%), congenital heart disease (14.6%), idiopathic pulmonary hypertension (5.8%), and chronic thromboembolic pulmonary hypertension (3.8%). Only one of two patients with pulmonary artery hypertension was receiving PH specific therapies. The use of combination therapy was negligible and PH-specific therapies were prescribed off-label to a small proportion of patients too. PRO-KERALA is the first PH registry from South Asia and the second largest globally. Left heart diseases attribute to three fifths of patients with PH. Utilization rates of PH specific drug therapies are remarkably lower than the Western population. Copyright © 2018 Elsevier B.V. All rights reserved.

  9. Interpreting clinical trial results by deductive reasoning: In search of improved trial design.

    Science.gov (United States)

    Kurbel, Sven; Mihaljević, Slobodan

    2017-10-01

    Clinical trial results are often interpreted by inductive reasoning, in a trial design-limited manner, directed toward modifications of the current clinical practice. Deductive reasoning is an alternative in which results of relevant trials are combined in indisputable premises that lead to a conclusion easily testable in future trials. © 2017 WILEY Periodicals, Inc.

  10. Clinical trials attitudes and practices of Latino physicians.

    Science.gov (United States)

    Ramirez, Amelie G; Wildes, Kimberly; Talavera, Greg; Nápoles-Springer, Anna; Gallion, Kipling; Pérez-Stable, Eliseo J

    2008-07-01

    Ethnic differences in physicians' attitudes and behaviors related to clinical trials might partially account for disparities in clinical trial participation among Latino patients. Literature regarding Latino physicians' clinical trials attitudes and practices, in comparison to White physicians, was lacking. Cross-sectional data from randomly selected physicians (N=695), stratified by ethnicity, were analyzed to test associations of ethnicity with physicians' participation in and attitudes toward referral of patients to clinical trials. Chi-square analyses showed significant (pLatino physicians were significantly less involved in clinical trials than White physicians and found less scientific value in them, highlighting areas for future education and intervention.

  11. Meta-analysis in clinical trials revisited.

    Science.gov (United States)

    DerSimonian, Rebecca; Laird, Nan

    2015-11-01

    In this paper, we revisit a 1986 article we published in this Journal, Meta-Analysis in Clinical Trials, where we introduced a random-effects model to summarize the evidence about treatment efficacy from a number of related clinical trials. Because of its simplicity and ease of implementation, our approach has been widely used (with more than 12,000 citations to date) and the "DerSimonian and Laird method" is now often referred to as the 'standard approach' or a 'popular' method for meta-analysis in medical and clinical research. The method is especially useful for providing an overall effect estimate and for characterizing the heterogeneity of effects across a series of studies. Here, we review the background that led to the original 1986 article, briefly describe the random-effects approach for meta-analysis, explore its use in various settings and trends over time and recommend a refinement to the method using a robust variance estimator for testing overall effect. We conclude with a discussion of repurposing the method for Big Data meta-analysis and Genome Wide Association Studies for studying the importance of genetic variants in complex diseases. Published by Elsevier Inc.

  12. A New Ethical Challenge for Institutional Review Boards (IRBs/Ethics Committees (ECs in the Assessment of Pediatric Clinical Trials

    Directory of Open Access Journals (Sweden)

    Klaus Rose

    2015-05-01

    Full Text Available Both the US and EU have introduced pediatric pharmaceutical legislation to facilitate clinical trials in children and development of better medicines for children. The first concerns were published in 2014 that the European Medicines Agency (EMA’s Pediatric Committee (PDCO may be over-enthusiastic and has compelled questionable pediatric clinical trials from pharmaceutical companies. Numerous clinical trials are mandated in rare conditions for which not enough patients exist for even one trial. Furthermore, where these trials are mandated in adolescent patients, the legal age limit of the 18th birthday is confused with a medical age limit and can result in separate clinical trials in adolescent patients that neither make medical nor scientific sense nor will ever recruit enough patients for a meaningful outcome. To confirm our concerns we searched the registry clinicaltrials.gov and found examples for PDCO-triggered unethical trials. We conclude that such trials should not be accepted by institutional review boards (IRBs/ethics committees (ECs and that clinical trials resulting from negotiations with EMA’s PDCO need extra careful scrutiny by IRBs/ECs in order to prevent unethical studies and damage to pediatric research and unnecessary risks to pediatric patients.

  13. Future clinical trials in DIPG: bringing epigenetics to the clinic

    Directory of Open Access Journals (Sweden)

    Andres E. Morales La Madrid

    2015-07-01

    Full Text Available In spite of major recent advances in DIPG molecular characterization, this body of knowledge has not yet translated into better treatments.To date,more than 250 clinical trials evaluating radiotherapy along with conventional cytotoxic chemotherapy as well as newer biologic agents,have failed to improve the dismal outcome when compared to palliative radiation alone.The biology of DIPG remained unknown until recently when the neurosurgical expertise along with the recognition by the scientific and clinical community of the importance of tissue sampling at diagnosis;ideally in the context of a clinical trial and by trained neurosurgical teams to maximize patient safety.These pre-treatment tumor samples,and others coming from tissue obtained post-mortem,have yielded new insights into DIPG molecular biology.We now know that DIPG comprises a heterogeneous disease with variable molecular phenotypes, different from adult high grade glioma,other non-pontine pediatric high grade gliomas and even between pontine gliomas.The discovery of histone H3.3 or H3.1 mutations has been an important step forward in understanding tumor formation,maintenance and progression.Pharmacologic reversal of DIPG histone demethylation therefore offers an important potential intervention strategy for the treatment of DIPG.To date,clinical trials of newly diagnosed or progressive DIPG with epigenetic modifiers have been unsuccessful.Whether this failure represents limited activity of the agents used,their CNS penetration,redundant pathways within the tumor,or the possibility that histone mutations are necessary only to initiate DIPGs but not maintain their growth,suggest that a great deal still needs to be elucidated in both the underlying biology of these pathways,and the drugs designed to target them.In this review, we discuss the role of both epigenetic and genetic mutations within DIPG and the development of treatment strategies directed against the unique abnormalities

  14. Clinical trials for stem cell therapies

    Directory of Open Access Journals (Sweden)

    Lomax Geoff

    2011-05-01

    Full Text Available Abstract In recent years, clinical trials with stem cells have taken the emerging field in many new directions. While numerous teams continue to refine and expand the role of bone marrow and cord blood stem cells for their vanguard uses in blood and immune disorders, many others are looking to expand the uses of the various types of stem cells found in bone marrow and cord blood, in particular mesenchymal stem cells, to uses beyond those that could be corrected by replacing cells in their own lineage. Early results from these trials have produced mixed results often showing minor or transitory improvements that may be attributed to extracellular factors. More research teams are accelerating the use of other types of adult stem cells, in particular neural stem cells for diseases where beneficial outcome could result from either in-lineage cell replacement or extracellular factors. At the same time, the first three trials using cells derived from pluripotent cells have begun.

  15. When clinical trials compete: prioritising study recruitment.

    Science.gov (United States)

    Gelinas, Luke; Lynch, Holly Fernandez; Bierer, Barbara E; Cohen, I Glenn

    2017-12-01

    It is not uncommon for multiple clinical trials at the same institution to recruit concurrently from the same patient population. When the relevant pool of patients is limited, as it often is, trials essentially compete for participants. There is evidence that such a competition is a predictor of low study accrual, with increased competition tied to increased recruitment shortfalls. But there is no consensus on what steps, if any, institutions should take to approach this issue. In this article, we argue that an institutional policy that prioritises some trials for recruitment ahead of others is ethically permissible and indeed prima facie preferable to alternative means of addressing recruitment competition. We motivate this view by appeal to the ethical importance of minimising the number of studies that begin but do not complete, thereby exposing their participants to unnecessary risks and burdens in the process. We then argue that a policy of prioritisation can be fair to relevant stakeholders, including participants, investigators and funders. Finally, by way of encouraging and helping to frame future debate, we propose some questions that would need to be addressed when identifying substantive ethical criteria for prioritising between studies. Published by the BMJ Publishing Group Limited. For permission to use (where not already granted under a licence) please go to http://www.bmj.com/company/products-services/rights-and-licensing/.

  16. Does information from ClinicalTrials.gov increase transparency and reduce bias? Results from a five-report case series.

    Science.gov (United States)

    Adam, Gaelen P; Springs, Stacey; Trikalinos, Thomas; Williams, John W; Eaton, Jennifer L; Von Isenburg, Megan; Gierisch, Jennifer M; Wilson, Lisa M; Robinson, Karen A; Viswanathan, Meera; Middleton, Jennifer Cook; Forman-Hoffman, Valerie L; Berliner, Elise; Kaplan, Robert M

    2018-04-16

    We investigated whether information in ClinicalTrials.gov would impact the conclusions of five ongoing systematic reviews. We considered five reviews that included 495 studies total. Each review team conducted a search of ClinicalTrials.gov up to the date of the review's last literature search, screened the records using the review's eligibility criteria, extracted information, and assessed risk of bias and applicability. Each team then evaluated the impact of the evidence found in ClinicalTrials.gov on the conclusions in the review. Across the five reviews, the number of studies that had both a registry record and a publication varied widely, from none in one review to 43% of all studies identified in another. Among the studies with both a record and publication, there was also wide variability in the match between published outcomes and those listed in ClinicalTrials.gov. Of the 173 total ClinicalTrials.gov records identified across the five projects, between 11 and 43% did not have an associated publication. In the 14% of records that contained results, the new data provided in the ClinicalTrials.gov records did not change the results or conclusions of the reviews. Finally, a large number of published studies were not registered in ClinicalTrials.gov, but many of these were published before ClinicalTrials.gov's inception date of 2000. Improved prospective registration of trials and consistent reporting of results in ClinicalTrials.gov would help make ClinicalTrials.gov records more useful in finding unpublished information and identifying potential biases. In addition, consistent indexing in databases, such as MEDLINE, would allow for better matching of records and publications, leading to increased utility of these searches for systematic review projects.

  17. Neurosurgery clinical registry data collection utilizing Informatics for Integrating Biology and the Bedside and electronic health records at the University of Rochester.

    Science.gov (United States)

    Pittman, Christine A; Miranpuri, Amrendra S

    2015-12-01

    In a population health-driven health care system, data collection through the use of clinical registries is becoming imperative to continue to drive effective and efficient patient care. Clinical registries rely on a department's ability to collect high-quality and accurate data. Currently, however, data are collected manually with a high risk for error. The University of Rochester's Department of Neurosurgery in conjunction with the university's Clinical and Translational Science Institute has implemented the integrated use of the Informatics for Integrating Biology and the Bedside (i2b2) informatics framework with the Research Electronic Data Capture (REDCap) databases.

  18. Talking About Trials: Overcoming Bottlenecks in Clinical Communication

    Science.gov (United States)

    Participation in clinical trials by adult patients is dismally low. No one knows how many patients are offered the opportunity to enroll in trials. NCI researchers are studying how patients hear about trials, whether they discuss enrollment with their providers, and the roles they play in deciding to participate in a trial.

  19. Clinical trials for stem cell transplantation: when are they needed?

    Science.gov (United States)

    Van Pham, Phuc

    2016-04-27

    In recent years, both stem cell research and the clinical application of these promising cells have increased rapidly. About 1000 clinical trials using stem cells have to date been performed globally. More importantly, more than 10 stem cell-based products have been approved in some countries. With the rapid growth of stem cell applications, some countries have used clinical trials as a tool to diminish the rate of clinical stem cell applications. However, the point at which stem cell clinical trials are essential remains unclear. This commentary discusses when stem cell clinical trials are essential for stem cell transplantation therapies.

  20. The thyroid registry: Clinical and hormonal characteristics of adult indian patients with hypothyroidism

    Directory of Open Access Journals (Sweden)

    Bipin Sethi

    2017-01-01

    Full Text Available Objectives: Appropriate treatment of hypothyroidism requires accurate diagnosis. This registry aimed to study the disease profile and treatment paradigm in hypothyroid patients in India. Materials and Methods: We registered 1500 newly diagnosed, treatment-naïve, adult hypothyroid males and nonpregnant females across 33 centers and collected relevant data from medical records. The first analysis report on baseline data is presented here. Results: The mean age of the study population was 41.1 ± 14.01 years with a female to male ratio of 7:3. The most frequently reported symptoms and signs were fatigue (60.17% and weight gain with poor appetite (36.22%. Menstrual abnormalities were reported in all women (n = 730 who had not attained menopause. Grades 1 and 2 goiter (as per the WHO were observed in 15.41% and 3.27% patients, respectively. Comorbidities were reported in 545 patients (36.36%, type 2 diabetes mellitus being the most prevalent (13.54% followed by hypertension (11.34%. Total serum thyroxine (T4 and thyroid-stimulating hormone (TSH levels were assessed in 291 (19.47% patients only. In majority of patients (81%, treatment was based on serum TSH levels alone. The dose of levothyroxine ranged from 12.5 to 375 mcg. Conclusions: Guidelines suggest a diagnosis of hypothyroidism based on TSH and T4 levels. However, most of the patients as observed in this registry received treatment with levothyroxine based on TSH levels alone, thus highlighting the need for awareness and scientific education among clinicians in India. The use of standard doses (100, 75, and 25 mcg of levothyroxine may point toward empirical management practices.

  1. Organisation of a clinical trial unit--a proposal

    DEFF Research Database (Denmark)

    Gluud, C; Sørensen, T I

    1998-01-01

    to cover investments, core staff, and running costs, but excluding housing costs and costs of randomised clinical trials that do not originate from trial coordination. In return, such a unit should be able to mount and launch 6-7 multicenter randomised clinical trials during a 5 year period, corresponding...

  2. Real-time enrollment dashboard for multisite clinical trials

    Directory of Open Access Journals (Sweden)

    William A. Mattingly

    2015-10-01

    Conclusion: We have designed and implemented a visualization dashboard for managing multi-site clinical trial enrollment in two community acquired pneumonia studies. Information dashboards are useful for clinical trial management. They can be used in a standalone trial or can be included into a larger management system.

  3. Citation Sentiment Analysis in Clinical Trial Papers.

    Science.gov (United States)

    Xu, Jun; Zhang, Yaoyun; Wu, Yonghui; Wang, Jingqi; Dong, Xiao; Xu, Hua

    2015-01-01

    In scientific writing, positive credits and negative criticisms can often be seen in the text mentioning the cited papers, providing useful information about whether a study can be reproduced or not. In this study, we focus on citation sentiment analysis, which aims to determine the sentiment polarity that the citation context carries towards the cited paper. A citation sentiment corpus was annotated first on clinical trial papers. The effectiveness of n-gram and sentiment lexicon features, and problem-specified structure features for citation sentiment analysis were then examined using the annotated corpus. The combined features from the word n-grams, the sentiment lexicons and the structure information achieved the highest Micro F-score of 0.860 and Macro-F score of 0.719, indicating that it is feasible to use machine learning methods for citation sentiment analysis in biomedical publications. A comprehensive comparison between citation sentiment analysis of clinical trial papers and other general domains were conducted, which additionally highlights the unique challenges within this domain.

  4. [Clinical trials: vulnerability and ethical relativism].

    Science.gov (United States)

    Lima, Cristina

    2005-01-01

    Research in human beings is an important chapter of medical ethics. In recent years, investigation has been taken over by profit driven corporations that must guarantee the medical and commercial application of results. This new model of investigation has generated conflicts of interest in doctor-patient, researcher-subject relationship. The inevitable debate and media reaction has led. These trials of controversial design to regions of the globe where the vulnerability of the populations continues to allow their undertaking. This article includes a historical perspective on experimentation in human beings and the conditions that led to its regulation: the Nuremberg CODE, followed by the Helsinky Declaration in its different versions, and the Belmont Report, that defend the subject according to the ethic of principles used in western medicine. There is then a review of the attempts to change international regulation to reintroduce clinical trials with placebo--which since 1996 is only permitted where there are no therapeutic or diagnostic methods--on populations that would otherwise have no access to treatment. This then leads on to the issue of double standards in medical investigation defended by many investigators and some official entities. The article concludes that it may be prudent to allow local ethical commissions to approve deviation from the established norm if such is necessary to resolve urgent questions of health in the country, but it is unacceptable that any such emergency is used as a reason to reduce the ethical prerequisites, in clinical trials. It also concludes that true urgency is in making available to all who need it the effective products already in existence. Furthermore, that the acceptance of ethical relativism can result in the exploitation of vulnerable third world populations for research programmes that cannot be undertaken in their sponsoring countries due to the ethical restrictions in place.

  5. Usefulness of ambulatory blood pressure monitoring (ABPM) in daily clinical practice: Data from the Spanish ABPM registry.

    Science.gov (United States)

    Segura, Julian; Banegas, Jose R; Ruilope, Luis M

    2014-01-01

    1. Hypertension is one of the most important challenges for public health systems because of its high prevalence and its association with the risk of cardiovascular and renal diseases. 2. Adequate control of hypertension is low in population and medical settings, with physicians frequently misclassifying patients' blood pressure status based on readings taken in the clinic rather than ambulatory blood pressure measurements (ABPM). 3. Data from the Spanish Society of Hypertension ABPM registry support ABPM as a feasible option in the primary care setting, providing valuable information for the diagnosis and management of hypertension. By using ABPM rather than office BP monitoring, BP control can be doubled. This is an encouraging message to clinicians, although there is still a relatively large degree of undetected controlled and uncontrolled hypertension. 4. This short review describes the design, development and main results of the Spanish Society of Hypertension ABPM registry, a project based on a large-scale network of Spanish physicians trained in ABPM. © 2013 Wiley Publishing Asia Pty Ltd.

  6. Danish Hip Arthroscopy Registry

    DEFF Research Database (Denmark)

    Mygind-Klavsen, Bjarne; Grønbech Nielsen, Torsten; Maagaard, Niels

    2016-01-01

    Danish Hip Arthroscopy Registry (DHAR) was initiated in 2012 as a web-based prospective registry. The purpose of this study was to evaluate and report the epidemiologic and perioperative data of the first 2000 procedures in a Danish hip arthroscopy population and to describe the development of DHAR...... was 0.65 and HAGOS sub-scores were 51 (pain), 49 (symptoms), 53 (ADL), 35 (sport), 20 (physical activity) and 29, respectively. We conclude that patients undergoing hip arthroscopy report considerable pain, loss of function, reduced level of activity and reduced quality-of-life prior to surgery....... The problems with development and maintaining a large clinical registry are described and further studies are needed to validate data completeness. We consider the development of a national clinical registry for hip arthroscopy as a successful way of developing and maintaining a valuable clinical...

  7. Dental hygiene work in a clinical trial.

    Science.gov (United States)

    Luís, H S; Morgado, I; Assunção, V; Bernardo, M F; Leroux, B; Martin, M D; DeRouen, T A; Leitão, J

    2008-08-01

    Dental hygiene activities were developed as part of a randomized clinical trial designed to assess the safety of low-level mercury exposure from dental amalgam restorations. Along with dental-hygiene clinical work, a community programme was implemented after investigators noticed the poor oral hygiene habits of participants, and the need for urgent action to minimize oral health problems in the study population. Clinical and community activity goal was to promote oral health and prevent new disease. Community activities involved participants and their fellow students and were aimed at providing education on oral health in a school environment. Dental hygienists developed clinical work with prophylaxis, sealants application and topical fluoride and implemented the community programme with in-class sessions on oral health themes. Twice a month fluoride mouthrinses and bi-annual tooth brushing instructional activity took place. Participation at dental-hygiene activities, sealed teeth with no need of restoration and dental-plaque-index were measures used to evaluate success of the programme for the participants. Improvement in dental hygiene is shown by the decrease in dental plaque index scores (P dental hygiene activities. Teachers became aware of the problem and included oral-health in school curricula. Dental hygiene activities have shown to be helpful to promote dental hygiene, promote oral health and to provide school-age children with education on habits that will be important for their future good health.

  8. The Italian Registry of Antiphospholipid Antibodies.

    Science.gov (United States)

    Finazzi, G

    1997-01-01

    The clinical importance of antiphospholipid antibodies (APA) derives from their association with a syndrome of venous and arterial thrombosis, recurrent fetal loss and thrombocytopenia known as the antiphospholipid syndrome (APS). The Italian Registry of Antiphospholipid Antibodies was set up in 1989 for the purpose of collecting a large number of patients with lupus anticoagulant (LA) or anticardiolipin antibodies (ACA) for clinical studies in order to obtain more information on the clinical features of APS. The Italian Registry has completed two clinical studies and proposed an international trial on the treatment of APS patients. These activities of the Registry are reviewed herein. Additional information has been obtained from pertinent articles and abstracts published in journals covered by the Science Citation Index and Medline. The first study of the Registry was a retrospective analysis of enrolled patients which showed that: a) the prevalence of thrombosis and thrombocytopenia was similar in cases with idiopathic APA or APA secondary to systemic lupus erythematosus, and b) the rate of thrombosis was significantly reduced in patients with severe thrombocytopenia but not in those with only a mild reduction of the platelet count. The second study was a prospective survey of the natural history of the disease, showing that a) previous thrombosis and ACA titer > 40 units were independent predictors of subsequent vascular complications; b) a history of miscarriage or thrombosis is significantly associated with adverse pregnancy outcome; c) hematological malignancies can develop during follow-up and patients with APA should be considered at increased risk of developing NHL. Thus the possibility of a hematologic neoplastic disease should be borne in mind in the initial evaluation and during the follow-up of these patients. The latest initiative of the Registry was the proposal of an international, randomized clinical trial (WAPS study) aimed at assessing the

  9. Operative technique at caesarean delivery and risk of complete uterine rupture in a subsequent trial of labour at term. A registry case-control study

    DEFF Research Database (Denmark)

    Thisted, Dorthe L. A.; Mortensen, Laust H.; Hvidman, Lone

    2017-01-01

    Objective: To estimate the relation of single-layer closure at previous caesarean delivery, and other pre-labour and intra-partum risk factors for complete uterine rupture in trial of vaginal birth after a caesarean (TOLAC) at term. Study design: Population-based case-control study. We identified...... all women (n = 39 742) recorded in the Danish Medical Birth Registry (DMBR) during a 12-year period (1997–2008) with a singleton pregnancy at term and TOLAC. Among these, all women with a complete uterine rupture were identified (cases). Information from the registry was validated against medical...... of uterine rupture. Conclusion: Single-layer uterine closure did not remain significantly associated to uterine rupture during TOLAC at term after adjustment for confounding factors. Induction of labour with an unfavourable cervix, birth weight ≥ 4000g and indicators of prolonged labour were all major risk...

  10. Contemporary issues in clinical trials for medulloblastoma

    International Nuclear Information System (INIS)

    Kun, Larry E.

    1996-01-01

    Medulloblastoma is the seminal pediatric brain tumor providing opportunities for clinical investigation to define improved treatment strategies for both disease control and ultimate functional integrity. Recent studies addressing neuraxis radiation dose provide a 'standard' for conventional therapy while establishing 5-year disease control rates for 'favorable' or 'low risk' presentations approximating 60% following surgery and irradiation. A highly visible recent report of combined post-operative irradiation and chemotherapy incorporating a platinum- and alkylator-based regimen indicates 5-year disease control approaching 90% in localized medulloblastoma. Despite unfavorable outcome with reduced-dose neuraxis irradiation in earlier trials, further data from recent studies suggest the addition of post-operative chemotherapy to similarly reduced-dose neuraxis irradiation (23.4 Gy) in 'favorable' presentations may result in progression-free survival rates at least equivalent to those achieved with full-dose neuraxis irradiation (36 Gy) absent chemotherapy. The panel will (1) provide updated information regarding the major clinical trials that form the basis for current and planned protocols and (2) debate the therapeutic modifications appropriate for contemporary clinical investigations. Critical in planning future studies in the analysis of risk factors that may identify 'favorable' patients versus 'high risk' patients. Risk-related studies appropriately address maintaining or improving current disease control rates in the context of diminishing late treatment sequelae for 'favorable' presentations. For those identified as 'high risk' (e.g., patients with disease beyond the primary site), studies are in development that increase the intensity of chemotherapy and explore modifications of radiation delivery. Study designs that permit assessment of innovations in surgical, radiotherapeutic, and chemotherapeutic approaches will be presented and debated by the panelists

  11. Is Mandatory Prospective Trial Registration Working to Prevent Publication of Unregistered Trials and Selective Outcome Reporting? An Observational Study of Five Psychiatry Journals That Mandate Prospective Clinical Trial Registration.

    Directory of Open Access Journals (Sweden)

    Amelia Scott

    Full Text Available To address the bias occurring in the medical literature associated with selective outcome reporting, in 2005, the International Committee of Medical Journal Editors (ICMJE introduced mandatory trial registration guidelines and member journals required prospective registration of trials prior to patient enrolment as a condition of publication. No research has examined whether these guidelines are impacting psychiatry publications. Our objectives were to determine the extent to which articles published in psychiatry journals adhering to ICMJE guidelines were correctly prospectively registered, whether there was evidence of selective outcome reporting and changes to participant numbers, and whether there was a relationship between registration status and source of funding.Any clinical trial (as defined by ICMJE published between 1 January 2009 and 31 July 2013 in the top five psychiatry journals adhering to ICMJE guidelines (The American Journal of Psychiatry, Archives of General Psychiatry/JAMA Psychiatry, Biological Psychiatry, Journal of the American Academy of Child and Adolescent Psychiatry, and The Journal of Clinical Psychiatry and conducted after July 2005 (or 2007 for two journals was included. For each identified trial, where possible we extracted trial registration information, changes to POMs between publication and registry to assess selective outcome reporting, changes to participant numbers, and funding type.Out of 3305 articles, 181 studies were identified as clinical trials requiring registration: 21 (11.6% were deemed unregistered, 61 (33.7% were retrospectively registered, 37 (20.4% had unclear POMs either in the article or the registry and 2 (1.1% were registered in an inaccessible trial registry. Only 60 (33.1% studies were prospectively registered with clearly defined POMs; 17 of these 60 (28.3% showed evidence of selective outcome reporting and 16 (26.7% demonstrated a change in participant numbers of 20% or more; only 26 (14

  12. Need for Outcome Scenario Analysis of Clinical Trials in Diabetes.

    Science.gov (United States)

    Garcia-Verdugo, Rosa; Erbach, Michael; Schnell, Oliver

    2017-03-01

    Since the FDA requirement for cardiovascular safety of all new antihyperglycemic drugs to enter the market, the number and extent of phase 3 clinical trials has markedly increased. Unexpected trial results imply an enormous economic, personal and time cost and has deleterious effects over R&D. To prevent unforeseen developments in clinical trials, we recommend performing a comprehensive prospective outcome scenario analysis before launching the trial. In this commentary, we discuss the most important factors to take in consideration for prediction of clinical trial outcome scenarios and propose a theoretical model for decision making.

  13. The Southern French registry of genetic hemochromatosis: a tool for determining clinical prevalence of the disorder and genotype penetrance.

    Science.gov (United States)

    Aguilar-Martinez, Patricia; Bismuth, Michael; Blanc, François; Blanc, Pierre; Cunat, Severine; Dereure, Olivier; Dujols, Pierre; Giansily-Blaizot, Muriel; Jorgensen, Christian; Konate, Amadou; Larrey, Dominique; Le Quellec, Alain; Mura, Thibault; Raingeard, Isabelle; Ramos, Jeanne; Renard, Eric; Rousseau, Florence; Schved, Jean-François; Picot, Marie-Christine

    2010-04-01

    Despite great progress in understanding the mechanisms underlying genetic hemochromatosis, data on the prevalence and the penetrance of the disorder are conflicting. A registry of patients with genetic hemochromatosis was established in the South of France and a regional health network was developed to allow the inclusion of all the diagnosed patients. C282Y homozygous patients classified in stages 2 (biological iron overload), 3 and 4 (clinical manifestations of iron overload, stage 4 being the more severe) according to the classification of the French National Authority for Health were included in the registry over a 6-year period. A total of 352 symptomatic C282Y homozygotes were identified, resulting in a total prevalence of 1.83 per 10,000 (95% CI: 1.63 to 2.02) in subjects over 20 years and 2.40 per 10,000 (95% CI, 2.15 to 2.65) among subjects of European descent. Among Europeans, the total calculated penetrance was 15.8% in stage 2 or higher, 12.1% in stage 3 or 4 and 2.9% in stage 4. The penetrance was slightly higher in males (18.7%) than in females (13.2%). It was 19.9% for individuals over 40 years of age (24.1% and 16.3% in males and females, respectively) with a maximum of 31% in subjects between 50 and 54 years old. Among 249 patients with complete records, 24% were in stage 2, the majority (58%) were in stage 3, and 18% in stage 4. There was a higher proportion of males, and excessive alcohol intake was more prevalent in stage 4 than in stages 2 and 3 combined. A French Mediterranean regional hemochromatosis registry with strict inclusion criteria is a useful tool for characterizing the history of this disease, particularly for the most severely affected patients, as defined by the disease severity classification. The total prevalence of symptomatic C282Y homozygotes in the region was found to be low. However, clinical penetrance (stages 3 and 4) was not negligible.

  14. Advancing the educational and career pathway for clinical trials nurses.

    Science.gov (United States)

    Scott, Kathleen; White, Kathryn; Roydhouse, Jessica K

    2013-04-01

    Clinical trials nurses play a pivotal role in the conduct of clinical research, but the educational and career pathway for these nurses remains unclear. This article reports findings from a survey of nurses working in cancer clinical trials research in Australia. Most participants held postgraduate qualifications (42 of 61); however, clinical trials education was primarily attained through short professional development courses. Interest in pursuing trial-specific postgraduate education was high, but barriers were identified, including cost, time, and unclear benefit for career advancement. Job titles varied substantially, which is indicative of an unclear employment pathway. These findings suggest that initiatives to improve the educational and career pathway for clinical trials nurses are needed and should include the following: formal educational preparation, greater consistency in employment status, and clearer career progression. These strategies should be underpinned by broad professional recognition of the clinical trials nurse as a specialized nursing role. Copyright 2013, SLACK Incorporated.

  15. Randomized Clinical Trials on Deep Carious Lesions

    DEFF Research Database (Denmark)

    Bjørndal, Lars; Fransson, Helena; Bruun, Gitte

    2017-01-01

    nonselective carious removal to hard dentin with or without pulp exposure. The aim of this article was to report the 5-y outcome on these previously treated patients having radiographically well-defined carious lesions extending into the pulpal quarter of the dentin but with a well-defined radiodense zone...... pulp exposures per se were included as failures. Pulp exposure rate was significantly lower in the stepwise carious removal group (21.2% vs. 35.5%; P = 0.014). Irrespective of pulp exposure status, the difference (13.3%) was still significant when sustained pulp vitality without apical radiolucency......) in deep carious lesions in adults. In conclusion, the stepwise carious removal group had a significantly higher proportion of pulps with sustained vitality without apical radiolucency versus nonselective carious removal of deep carious lesions in adult teeth at 5-y follow-up (ClinicalTrials.gov NCT...

  16. Motivators for Alzheimer's disease clinical trial participation.

    Science.gov (United States)

    Bardach, Shoshana H; Holmes, Sarah D; Jicha, Gregory A

    2018-02-01

    Alzheimer's disease (AD) research progress is impeded due to participant recruitment challenges. This study seeks to better understand, from the perspective of individuals engaged in clinical trials (CTs), research motivations. Participants, or their caregivers, from AD treatment and prevention CTs were surveyed about research motivators. The 87 respondents had a mean age of 72.2, were predominantly Caucasian, 55.2% were male, and 56.3% had cognitive impairment. An overwhelming majority rated the potential to help themselves or a loved one and the potential to help others in the future as important motivators. Relatively few respondents were motivated by free healthcare, monetary rewards, or to make others happy. Recruitment efforts should focus on the potential benefit for the individual, their loved ones, and others in the future rather than free healthcare or monetary rewards.

  17. Malignant mesothelioma clinical trial combines immunotherapy drugs.

    Science.gov (United States)

    Chatwal, Monica S; Tanvetyanon, Tawee

    2018-04-01

    Immunotherapy by checkpoint inhibitor is effective for a number of solid tumors including malignant mesothelioma. Studies utilizing single-agent PD-1 or PD-L1 inhibitor for mesothelioma have reported tumor response rates in approximately 10-20% of patients treated. Given the success of combining these agents with CTLA-4 inhibitor in melanoma, there is a strong rationale to study it in mesothelioma. Recently results from clinical trials investigating this approach have been released. Though limited by small sample size, the studies conclusively demonstrated feasibility and suggested a modestly higher tumor response rate than one would expect from treatment with single-agent PD-1 or PD-L1 inhibitor. Nevertheless, toxicity was also increased. Immunotherapy-related deaths due to encephalitis, renal failure and hepatitis were observed. Further studies are warranted.

  18. A data grid for imaging-based clinical trials

    Science.gov (United States)

    Zhou, Zheng; Chao, Sander S.; Lee, Jasper; Liu, Brent; Documet, Jorge; Huang, H. K.

    2007-03-01

    Clinical trials play a crucial role in testing new drugs or devices in modern medicine. Medical imaging has also become an important tool in clinical trials because images provide a unique and fast diagnosis with visual observation and quantitative assessment. A typical imaging-based clinical trial consists of: 1) A well-defined rigorous clinical trial protocol, 2) a radiology core that has a quality control mechanism, a biostatistics component, and a server for storing and distributing data and analysis results; and 3) many field sites that generate and send image studies to the radiology core. As the number of clinical trials increases, it becomes a challenge for a radiology core servicing multiple trials to have a server robust enough to administrate and quickly distribute information to participating radiologists/clinicians worldwide. The Data Grid can satisfy the aforementioned requirements of imaging based clinical trials. In this paper, we present a Data Grid architecture for imaging-based clinical trials. A Data Grid prototype has been implemented in the Image Processing and Informatics (IPI) Laboratory at the University of Southern California to test and evaluate performance in storing trial images and analysis results for a clinical trial. The implementation methodology and evaluation protocol of the Data Grid are presented.

  19. Recruiting older people at nutritional risk for clinical trials: what have we learned?

    Science.gov (United States)

    Piantadosi, Cynthia; Chapman, Ian M; Naganathan, Vasi; Hunter, Peter; Cameron, Ian D; Visvanathan, Renuka

    2015-04-15

    The difficulty of recruiting older people to clinical trials is well described, but there is limited information about effective ways to screen and recruit older people into trials, and the reasons for their reluctance to enrol. This paper examines recruitment efforts for a community-based health intervention study that targeted older adults. One year randomized control trial. Undernourished men and women, aged ≥ 65 years and living independently in the community were recruited in three Australian states. Participants were allocated to either oral testosterone undecanoate and high calorie oral nutritional supplement or placebo medication and low calorie oral nutritional supplementation. Hospital admissions, functional status, nutritional health, muscle strength, and other variables were assessed. 4023 potential participants were identified and 767 were screened by a variety of methods: hospital note screening, referrals from geriatric health services, advertising and media segments/appearances. 53 participants (7% of total screened) were recruited. The majority of potentially eligible participants declined participation in the trial after reading the information sheet. Media was the more successful method of recruiting, whereas contacting people identified by screening a large number of hospital records was not successful in recruiting any participants. Recruitment of frail and older participants is difficult and multiple strategies are required to facilitate participation. Australian Clinical Trial Registry: ACTRN 12610000356066 date registered 4/5/2010.

  20. Clinical outcomes according to symptom presentation in patients with acute myocardial infarction: Results from the FAST-MI 2010 registry.

    Science.gov (United States)

    Puymirat, Etienne; Aissaoui, Nadia; Bonello, Laurent; Cayla, Guillaume; Labèque, Jean-Noel; Nallet, Olivier; Motreff, Pascal; Varenne, Olivier; Schiele, François; Ferrières, Jean; Simon, Tabassome; Danchin, Nicolas

    2017-12-01

    Atypical clinical presentation in acute myocardial infarction (AMI) patients is not uncommon; most studies suggest that it is associated with unfavorable prognosis. Long-term clinical impact differs according to predominant symptom presentation (typical chest pain, atypical chest pain, syncope, cardiac arrest, or dyspnea) in AMI patients. FAST-MI 2010, a nationwide French registry, included 4169 patients with AMI in 213 centers at the end of 2010 (76% of active centers). Demographics, medical history, hospital management, and outcomes were compared according to predominant symptom presentation. Typical chest pain with no other symptom was reported in 3020 patients (68% in STEMI patients, 76% in NSTEMI patients). Atypical chest pain, dyspnea, syncope, and cardiac arrest were reported in 11%, 11%, 5%, and 1%, respectively. Patients with atypical clinical presentation had a higher cardiovascular risk profile and received fewer medications and a less invasive strategy. Using Cox multivariate analysis, atypical chest pain was not associated with higher death rate at 3 years (HR: 0.96, 95% CI: 0.69-1.33, P = 0.78), whereas cardiac arrest (HR: 2.44, 95% CI: 1.00-5.97, P = 0.05), syncope (HR: 1.70, 95% CI: 1.18-2.46, P = 0.005), and dyspnea (HR: 1.66, 95% CI: 1.31-2.10, P clinical presentation is observed in about 20% of AMI patients. Cardiac arrest, dyspnea, and syncope represent independent predictors of long-term mortality in STEMI and NSTEMI populations. © 2017 Wiley Periodicals, Inc.

  1. OARSI Clinical Trials Recommendations: Hand imaging in clinical trials in osteoarthritis.

    Science.gov (United States)

    Hunter, D J; Arden, N; Cicuttini, F; Crema, M D; Dardzinski, B; Duryea, J; Guermazi, A; Haugen, I K; Kloppenburg, M; Maheu, E; Miller, C G; Martel-Pelletier, J; Ochoa-Albíztegui, R E; Pelletier, J-P; Peterfy, C; Roemer, F; Gold, G E

    2015-05-01

    Tremendous advances have occurred in our understanding of the pathogenesis of hand osteoarthritis (OA) and these are beginning to be applied to trials targeted at modification of the disease course. The purpose of this expert opinion, consensus driven exercise is to provide detail on how one might use and apply hand imaging assessments in disease modifying clinical trials. It includes information on acquisition methods/techniques (including guidance on positioning for radiography, sequence/protocol recommendations/hardware for MRI); commonly encountered problems (including positioning, hardware and coil failures, sequences artifacts); quality assurance/control procedures; measurement methods; measurement performance (reliability, responsiveness, validity); recommendations for trials; and research recommendations. Copyright © 2015 Osteoarthritis Research Society International. Published by Elsevier Ltd. All rights reserved.

  2. Biopharmaceutical industry-sponsored global clinical trials in emerging countries.

    Science.gov (United States)

    Alvarenga, Lenio Souza; Martins, Elisabeth Nogueira

    2010-01-01

    To evaluate biopharmaceutical industry-sponsored clinical trials placed in countries previously described as emerging regions for clinical research, and potential differences for those placed in Brazil. Data regarding recruitment of subjects for clinical trials were retrieved from www.clinicaltrials.gov on February 2nd 2009. Proportions of sites in each country were compared among emerging countries. Multiple logistic regressions were performed to evaluate whether trial placement in Brazil could be predicted by trial location in other countries and/or by trial features. A total of 8,501 trials were then active and 1,170 (13.8%) included sites in emerging countries (i.e., Argentina, Brazil, China, Czech Republic, Hungary, India, Mexico, Poland, Russia, South Korea, and South Africa). South Korea and China presented a significantly higher proportion of sites when compared to other countries (pattractiveness for biopharmaceutical industry-sponsored clinical trials.

  3. Contribution of clinical trials to gross domestic product in Hungary.

    Science.gov (United States)

    Kaló, Zoltán; Antal, János; Pénzes, Miklós; Pozsgay, Csilla; Szepezdi, Zsuzsanna; Nagyjánosi, László

    2014-10-01

    To determine the contribution of clinical trials to the gross domestic product (GDP) in Hungary. An anonymous survey of pharmaceutical companies and clinical research organizations (CROs) was conducted to estimate their clinical trial-related employment and revenues. Clinical trial documents at the National Institute of Pharmacy (NIP) were analyzed to estimate trial-related revenues at health care institutions and the value of investigational medical products (IMPs) based on avoided drug costs. Financial benefits were calculated as 2010 US $ purchasing power parity (PPP) values. Clinical trials increased the revenue of Hungarian health care providers by 1 US $65.6 million. The value of IMPs was US $67.0 million. Clinical trial operation and management activities generated 900 jobs and US $166.9 million in revenue among CROs and pharmaceutical companies. The contribution of clinical trials to the Hungarian GDP in 2010 amounted to 0.2%. Participation in international clinical trials may result in health, financial, and intangible benefits that contribute to the sustainability of health care systems, especially in countries with severe resource constraints. Although a conservative approach was employed to estimate the economic benefits of clinical trials, further research is necessary to improve the generalizability of our findings.

  4. Development in the number of clinical trial applications in Western Europe from 2007 to 2015

    DEFF Research Database (Denmark)

    Dombernowsky, Tilde; Hædersdal, Merete; Lassen, Ulrik

    2017-01-01

    OBJECTIVE: To investigate the development in the number of applications for authorisation of clinical trials of medicines (CTAs) submitted annually to national competent authorities in 10 Western European member states of the European Union from 2007 to 2015. DESIGN: Registry study. SETTING: Data...... from national competent authorities. PARTICIPANTS: Germany, Italy, Spain, France, the UK, Belgium, the Netherlands, Austria, Denmark and Sweden. Inclusion criteria were Western European member states of the European Union, receiving more than 200 CTAs per year. OUTCOME MEASURES: Summarised number...... of CTAs and distribution of CTAs by type of sponsor (commercial or non-commercial) and trial phase (I-IV). Average annual growth rates (AAGRs) based on linear regressions. Data were evaluated 2007-2011 and 2012-2015 to compare findings with the European Commission's statement of a 25% decrease in CTAs...

  5. The Alzheimer's Disease Neuroimaging Initiative 3: Continued innovation for clinical trial improvement

    Energy Technology Data Exchange (ETDEWEB)

    Weiner, Michael W. [Dept. of Veterans Affairs Medical Center, San Francisco, CA (United States); Univ. of California, San Francisco, CA (United States); Veitch, Dallas P. [Dept. of Veterans Affairs Medical Center, San Francisco, CA (United States); Aisen, Paul S. [Univ. of Southern California, San Diego, CA (United States); Beckett, Laurel A. [Univ. of California, Davis, CA (United States); Cairns, Nigel J. [Washington Univ. School of Medicine, St. Louis, MO (United States); Green, Robert C. [Brigham and Women' s Hospital and Harvard Medical School, Boston, MA (United States); Harvey, Danielle [Univ. of California, Davis, CA (United States); Jack, Clifford R. [Mayo Clinic, Rochester, MN (United States); Jagust, William [Univ. of California, Berkeley, CA (United States); Morris, John C. [Univ. of Southern California, San Diego, CA (United States); Petersen, Ronald C. [Mayo Clinic, Rochester, MN (United States); Salazar, Jennifer [Univ. of Southern California, San Diego, CA (United States); Saykin, Andrew J. [Indiana Univ. School of Medicine, Indianapolis, IN (United States); Shaw, Leslie M. [Eli Lilly and Company, Indianapolis, IN (United States); Toga, Arthur W. [Univ. of Southern California, Los Angeles, CA (United States); Trojanowski, John Q. [Univ. of Pennsylvania, Philadelphia, PA (United States)

    2016-12-05

    Overall, the goal of the Alzheimer's Disease Neuroimaging Initiative (ADNI) is to validate biomarkers for Alzheimer's disease (AD) clinical trials. ADNI-3, which began on August 1, 2016, is a 5-year renewal of the current ADNI-2 study. ADNI-3 will follow current and additional subjects with normal cognition, mild cognitive impairment, and AD using innovative technologies such as tau imaging, magnetic resonance imaging sequences for connectivity analyses, and a highly automated immunoassay platform and mass spectroscopy approach for cerebrospinal fluid biomarker analysis. A Systems Biology/pathway approach will be used to identify genetic factors for subject selection/enrichment. Amyloid positron emission tomography scanning will be standardized using the Centiloid method. The Brain Health Registry will help recruit subjects and monitor subject cognition. Multimodal analyses will provide insight into AD pathophysiology and disease progression. Finally, ADNI-3 will aim to inform AD treatment trials and facilitate development of AD disease-modifying treatments.

  6. Planning and analyzing clinical trials with composite endpoints

    CERN Document Server

    Rauch, Geraldine; Kieser, Meinhard

    2017-01-01

    This book addresses the most important aspects of how to plan and evaluate clinical trials with a composite primary endpoint to guarantee a clinically meaningful and valid interpretation of the results. Composite endpoints are often used as primary efficacy variables for clinical trials, particularly in the fields of oncology and cardiology. These endpoints combine several variables of interest within a single composite measure, and as a result, all variables that are of major clinical relevance can be considered in the primary analysis without the need to adjust for multiplicity. Moreover, composite endpoints are intended to increase the size of the expected effects thus making clinical trials more powerful. The book offers practical advice for statisticians and medical experts involved in the planning and analysis of clinical trials. For readers who are mainly interested in the application of the methods, all the approaches are illustrated with real-world clinical trial examples, and the software codes requ...

  7. Timing of oral anticoagulant therapy in acute ischemic stroke with atrial fibrillation: study protocol for a registry-based randomised controlled trial.

    Science.gov (United States)

    Åsberg, Signild; Hijazi, Ziad; Norrving, Bo; Terént, Andreas; Öhagen, Patrik; Oldgren, Jonas

    2017-12-02

    Oral anticoagulation therapy is recommended for the prevention of recurrent ischemic stroke in patients with atrial fibrillation (AF). Current guidelines do not provide evidence-based recommendations on optimal time-point to start anticoagulation therapy after an acute ischemic stroke. Non-vitamin K antagonist oral anticoagulants (NOACs) may offer advantages compared to warfarin because of faster and more predictable onset of action and potentially a lower risk of intracerebral haemorrhage also in the acute phase after an ischemic stroke. The TIMING study aims to establish the efficacy and safety of early vs delayed initiation of NOACs in patients with acute ischemic stroke and AF. The TIMING study is a national, investigator-led, registry-based, multicentre, open-label, randomised controlled study. The Swedish Stroke Register is used for enrolment, randomisation and follow-up of 3000 patients, who are randomised (1:1) within 72 h from ischemic stroke onset to either early (≤ 4 days) or delayed (≥ 5-10 days) start of NOAC therapy. The primary outcome is the composite of recurrent ischemic stroke, symptomatic intracerebral haemorrhage, or all-cause mortality within 90 days after randomisation. Secondary outcomes include: individual components of the primary outcome at 90 and 365 days; major haemorrhagic events; functional outcome by the modified Rankin Scale at 90 days; and health economics. In an optional biomarker sub-study, blood samples will be collected after randomisation from approximately half of the patients for central analysis of cardiovascular biomarkers after study completion. The study is funded by the Swedish Medical Research Council. Enrolment of patients started in April 2017. The TIMING study addresses the ongoing clinical dilemma of when to start NOAC after an acute ischemic stroke in patients with AF. By the inclusion of a randomisation module within the Swedish Stroke Register, the advantages of a prospective randomised study design

  8. The clinically-integrated randomized trial: proposed novel method for conducting large trials at low cost

    Directory of Open Access Journals (Sweden)

    Scardino Peter T

    2009-03-01

    Full Text Available Abstract Introduction Randomized controlled trials provide the best method of determining which of two comparable treatments is preferable. Unfortunately, contemporary randomized trials have become increasingly expensive, complex and burdened by regulation, so much so that many trials are of doubtful feasibility. Discussion Here we present a proposal for a novel, streamlined approach to randomized trials: the "clinically-integrated randomized trial". The key aspect of our methodology is that the clinical experience of the patient and doctor is virtually indistinguishable whether or not the patient is randomized, primarily because outcome data are obtained from routine clinical data, or from short, web-based questionnaires. Integration of a randomized trial into routine clinical practice also implies that there should be an attempt to randomize every patient, a corollary of which is that eligibility criteria are minimized. The similar clinical experience of patients on- and off-study also entails that the marginal cost of putting an additional patient on trial is negligible. We propose examples of how the clinically-integrated randomized trial might be applied in four distinct areas of medicine: comparisons of surgical techniques, "me too" drugs, rare diseases and lifestyle interventions. Barriers to implementing clinically-integrated randomized trials are discussed. Conclusion The proposed clinically-integrated randomized trial may allow us to enlarge dramatically the number of clinical questions that can be addressed by randomization.

  9. Lumbar spinal stenosis: comparison of surgical practice variation and clinical outcome in three national spine registries.

    Science.gov (United States)

    Lønne, Greger; Fritzell, Peter; Hägg, Olle; Nordvall, Dennis; Gerdhem, Paul; Lagerbäck, Tobias; Andersen, Mikkel; Eiskjaer, Søren; Gehrchen, Martin; Jacobs, Wilco; van Hooff, Miranda L; Solberg, Tore K

    2018-05-21

    Decompression surgery for lumbar spinal stenosis (LSS) is the most common spinal procedure in the elderly. To avoid persisting low back pain, adding arthrodesis has been recommended, especially if there is a coexisting degenerative spondylolisthesis. However, this strategy remains controversial, resulting in practice-based variation. The present study aimed to evaluate in a pragmatic study if surgical selection criteria and variation in use of arthrodesis in three Scandinavian countries can be linked to variation in treatment effectiveness. This is an observational study based on a combined cohort from the national spine registries of Norway, Sweden, and Denmark. Patients aged 50 and older operated during 2011-2013 for LSS were included. Patient-Reported Outcome Measures (PROMs): Oswestry Disability Index (ODI) (primary outcome), Numeric Rating Scale (NRS) for leg pain and back pain, and health-related quality of life (Euro-Qol-5D) were reported. Analysis included case-mix adjustment. In addition, we report differences in hospital stay. Analyses of baseline data were done by analysis of variance (ANOVA), chi-square, or logistic regression tests. The comparisons of the mean changes of PROMs at 1-year follow-up between the countries were done by ANOVA (crude) and analysis of covariance (case-mix adjustment). Out of 14,223 included patients, 10,890 (77%) responded at 1-year follow-up. Apart from fewer smokers in Sweden and higher comorbidity rate in Norway, baseline characteristics were similar. The rate of additional fusion surgery (patients without or with spondylolisthesis) was 11% (4%, 47%) in Norway, 21% (9%, 56%) in Sweden, and 28% (15%, 88%) in Denmark. At 1-year follow-up, the mean improvement for ODI (95% confidence interval) was 18 (17-18) in Norway, 17 (17-18) in Sweden, and 18 (17-19) in Denmark. Patients operated with arthrodesis had prolonged hospital stay. Real-life data from three national spine registers showed similar indications for decompression

  10. Phase II cancer clinical trials for biomarker-guided treatments.

    Science.gov (United States)

    Jung, Sin-Ho

    2018-01-01

    The design and analysis of cancer clinical trials with biomarker depend on various factors, such as the phase of trials, the type of biomarker, whether the used biomarker is validated or not, and the study objectives. In this article, we demonstrate the design and analysis of two Phase II cancer clinical trials, one with a predictive biomarker and the other with an imaging prognostic biomarker. Statistical testing methods and their sample size calculation methods are presented for each trial. We assume that the primary endpoint of these trials is a time to event variable, but this concept can be used for any type of endpoint.

  11. Detecting dissonance in clinical and research workflow for translational psychiatric registries.

    Science.gov (United States)

    Cofiel, Luciana; Bassi, Débora U; Ray, Ryan Kumar; Pietrobon, Ricardo; Brentani, Helena

    2013-01-01

    The interplay between the workflow for clinical tasks and research data collection is often overlooked, ultimately making it ineffective. To the best of our knowledge, no previous studies have developed standards that allow for the comparison of workflow models derived from clinical and research tasks toward the improvement of data collection processes. In this study we used the term dissonance for the occurrences where there was a discord between clinical and research workflows. We developed workflow models for a translational research study in psychiatry and the clinic where its data collection was carried out. After identifying points of dissonance between clinical and research models we derived a corresponding classification system that ultimately enabled us to re-engineer the data collection workflow. We considered (1) the number of patients approached for enrollment and (2) the number of patients enrolled in the study as indicators of efficiency in research workflow. We also recorded the number of dissonances before and after the workflow modification. We identified 22 episodes of dissonance across 6 dissonance categories: actor, communication, information, artifact, time, and space. We were able to eliminate 18 episodes of dissonance and increase the number of patients approached and enrolled in research study trough workflow modification. The classification developed in this study is useful for guiding the identification of dissonances and reveal modifications required to align the workflow of data collection and the clinical setting. The methodology described in this study can be used by researchers to standardize data collection process.

  12. Comparison of reporting phase I trial results in ClinicalTrials.gov and matched publications.

    Science.gov (United States)

    Shepshelovich, D; Goldvaser, H; Wang, L; Abdul Razak, A R; Bedard, P L

    2017-12-01

    Background Data on completeness of reporting of phase I cancer clinical trials in publications are lacking. Methods The ClinicalTrials.gov database was searched for completed adult phase I cancer trials with reported results. PubMed was searched for matching primary publications published prior to November 1, 2016. Reporting in primary publications was compared with the ClinicalTrials.gov database using a 28-point score (2=complete; 1=partial; 0=no reporting) for 14 items related to study design, outcome measures and safety profile. Inconsistencies between primary publications and ClinicalTrials.gov were recorded. Linear regression was used to identify factors associated with incomplete reporting. Results After a review of 583 trials in ClinicalTrials.gov , 163 matching primary publications were identified. Publications reported outcomes that did not appear in ClinicalTrials.gov in 25% of trials. Outcomes were upgraded, downgraded or omitted in publications in 47% of trials. The overall median reporting score was 23/28 (interquartile range 21-25). Incompletely reported items in >25% publications were: inclusion criteria (29%), primary outcome definition (26%), secondary outcome definitions (53%), adverse events (71%), serious adverse events (80%) and dates of study start and database lock (91%). Higher reporting scores were associated with phase I (vs phase I/II) trials (ppublication in journals with lower impact factor (p=0.004). Conclusions Reported results in primary publications for early phase cancer trials are frequently inconsistent or incomplete compared with ClinicalTrials.gov entries. ClinicalTrials.gov may provide more comprehensive data from new cancer drug trials.

  13. Laboratory research at the clinical trials of Veterinary medicinal Products

    OpenAIRE

    ZHYLA M.I.

    2011-01-01

    The article analyses the importance of laboratory test methods, namely pathomorfological at conduct of clinical trials. The article focuses on complex laboratory diagnostics at determination of clinical condition of animals, safety and efficacy of tested medicinal product.

  14. Clinical Trials: A Crucial Key to Human Health Research

    Science.gov (United States)

    ... Past Issues Clinical Trials: A Crucial Key to Human Health Research Past Issues / Summer 2006 Table of Contents ... Javascript on. Photo: PhotoDisc At the forefront of human health research today are clinical trials—studies that use ...

  15. Rationale, Design, and Methodology of the APOLLON trial: A comPrehensive, ObservationaL registry of heart faiLure with midrange and preserved ejectiON fraction.

    Science.gov (United States)

    Özlek, Bülent; Özlek, Eda; Çelik, Oğuzhan; Çil, Cem; Doğan, Volkan; Tekinalp, Mehmet; Zencirkıran Ağuş, Hicaz; Kahraman, Serkan; Ösken, Altuğ; Rencüzoğulları, İbrahim; Tanık, Veysel Ozan; Bekar, Lütfü; Çakır, Mustafa Ozan; Kaya, Bedri Caner; Tibilli, Hakan; Çelik, Yunus; Başaran, Özcan; Mert, Kadir Uğur; Sevinç, Samet; Demirci, Erkan; Dondurmacı, Engin; Biteker, Murat

    2018-05-01

    Although almost half of chronic heart failure (HF) patients have mid-range (HFmrEF) and preserved left-ventricular ejection fraction (HFpEF), no studies have been carried out with these patients in our country. This study aims to determine the demographic characteristics and current status of the clinical background of HFmrEF and HFpEF patients in a multicenter trial. A comPrehensive, ObservationaL registry of heart faiLure with mid range and preserved ejectiON fraction (APOLLON) trial will be an observational, multicenter, and noninterventional study conducted in Turkey. The study population will include 1065 patients from 12 sites in Turkey. All data will be collected at one point in time and the current clinical practice will be evaluated (ClinicalTrials.gov number NCT03026114). We will enroll all consecutive patients admitted to the cardiology clinics who were at least 18 years of age and had New York Heart Association class II, III, or IV HF, elevated brain natriuretic peptide levels within the last 30 days, and an left ventricular ejection fraction (LVEF) of at least 40%. Patients fulfilling the exclusion criteria will not be included in the study. Patients will be stratified into two categories according to LVEF: mid-range EF (HFmrEF, LVEF 40%-49%) and preserved EF (HFpEF, LVEF ≥50%). Regional quota sampling will be performed to ensure that the sample was representative of the Turkish population. Demographic, lifestyle, medical, and therapeutic data will be collected by this specific survey. The APOLLON trial will be the largest and most comprehensive study in Turkey evaluating HF patients with a LVEF ≥40% and will also be the first study to specifically analyze the recently designated HFmrEF category.

  16. Pancreatic cancer clinical trials and accrual in the United States.

    Science.gov (United States)

    Hoos, William A; James, Porsha M; Rahib, Lola; Talley, Anitra W; Fleshman, Julie M; Matrisian, Lynn M

    2013-09-20

    Pancreatic cancer clinical trials open in the United States and their accrual were examined to identify opportunities to accelerate progress in the treatment of pancreatic cancer. Pancreatic cancer-specific clinical trials open in the United States in the years 2011 and 2012 were obtained from the Pancreatic Cancer Action Network database. Accrual information was obtained from trial sponsors. The portfolio of pancreatic cancer clinical trials identified by type (adenocarcinoma or neuroendocrine), phase, disease stage, and treatment approach is reported. More than half of trials for patients with pancreatic ductal adenocarcinoma applied biologic insights to new therapeutic approaches, and 38% focused on optimization of radiation or chemotherapy delivery or regimens. In 2011, pancreatic cancer trials required total enrollment of 11,786 patients. Actual accrual to 93.2% of trials was 1,804 patients, an estimated 4.57% of the patients with pancreatic cancer alive in that year. The greatest need was for patients with resectable cancer. Trials open in 2011 enrolled an average of 15% of their total target accrual. Physician recommendations greatly influenced patients' decision to enroll or not enroll onto a clinical trial. Matching to a clinical trial within a 50-mile radius and identifying trials for recurrent/refractory disease were documented as challenges for patient accrual. Overall trial enrollment indicates that pancreatic cancer trials open in 2011 would require 6.7 years on average to complete accrual. These results suggest that harmonizing patient supply and demand for clinical trials is required to accelerate progress toward improving survival in pancreatic cancer.

  17. Reinventing clinical trials: a review of innovative biomarker trial designs in cancer therapies.

    Science.gov (United States)

    Lin, Ja-An; He, Pei

    2015-06-01

    Recently, new clinical trial designs involving biomarkers have been studied and proposed in cancer clinical research, in the hope of incorporating the rapid growing basic research into clinical practices. Journal articles related to various biomarkers and their role in cancer clinical trial, articles and books about statistical issues in trial design, and regulatory website, documents, and guidance for submission of targeted cancer therapies. The drug development process involves four phases. The confirmatory Phase III is essential in regulatory approval of a special treatment. Regulatory agency has restrictions on confirmatory trials 'using adaptive designs'. No rule of thumb to pick the most appropriate design for biomarker-related trials. Statistical issues to solve in new designs. Regulatory acceptance of the 'newly proposed trial designs'. Biomarker-related trial designs that can resolve the statistical issues and satisfy the regulatory requirement. © The Author 2015. Published by Oxford University Press. All rights reserved. For permissions, please e-mail: journals.permissions@oup.com.

  18. Unreported links between trial registrations and published articles were identified using document similarity measures in a cross-sectional analysis of ClinicalTrials.gov.

    Science.gov (United States)

    Dunn, Adam G; Coiera, Enrico; Bourgeois, Florence T

    2018-03-01

    Trial registries can be used to measure reporting biases and support systematic reviews, but 45% of registrations do not provide a link to the article reporting on the trial. We evaluated the use of document similarity methods to identify unreported links between ClinicalTrials.gov and PubMed. We extracted terms and concepts from a data set of 72,469 ClinicalTrials.gov registrations and 276,307 PubMed articles and tested methods for ranking articles across 16,005 reported links and 90 manually identified unreported links. Performance was measured by the median rank of matching articles and the proportion of unreported links that could be found by screening ranked candidate articles in order. The best-performing concept-based representation produced a median rank of 3 (interquartile range [IQR] 1-21) for reported links and 3 (IQR 1-19) for the manually identified unreported links, and term-based representations produced a median rank of 2 (1-20) for reported links and 2 (IQR 1-12) in unreported links. The matching article was ranked first for 40% of registrations, and screening 50 candidate articles per registration identified 86% of the unreported links. Leveraging the growth in the corpus of reported links between ClinicalTrials.gov and PubMed, we found that document similarity methods can assist in the identification of unreported links between trial registrations and corresponding articles. Copyright © 2017 Elsevier Inc. All rights reserved.

  19. Evaluation of eligibility and recruitment in breast cancer clinical trials.

    Science.gov (United States)

    Lemieux, Julie; Forget, Geneviève; Brochu, Olyvia; Provencher, Louise; Cantin, Guy; Desbiens, Christine; Doyle, Catherine; Poirier, Brigitte; Camden, Stéphanie; Durocher, Martin

    2014-08-01

    Objectives of the study were to measure recruitment rates in clinical trials and to identify patients, physicians or trials characteristics associated with higher recruitment rates. Among patients who had a clinical trial available for their cancer, 83.5% (345/413) met the eligibility criteria to at least one clinical trial. At least one trial was proposed to 33.1% (113/341) of the eligible patients and 19.7% (68/345) were recruited. Overall recruitment was 16.5% (68/413). In multivariate analyses, trial proposal and enrollment were lower for elderly patients and higher in high cancer stages. Trials from pharmaceutical industry had higher recruitment rates and trials testing hormonal therapy enrolled more patients. Breast cancer patients' accrual to a clinical trial could be improved by trying to systematically identify all eligible patients and propose a trial to those eligible and to whom the treatment is planned to be equivalent to the standard arm of the trial. Copyright © 2014 Elsevier Ltd. All rights reserved.

  20. Monitoring additive manufacturing based products in clinical trials

    NARCIS (Netherlands)

    Marinakis, Yorgos; Harms, Rainer; Walsh, Steven Thomas

    2017-01-01

    Under U.S. federal regulation 31 CFR §312, medical interventions must report on a series of clinical trials phases before being submitted for approval for release to the U.S. market. Clinical trials are now being performed on medical interventions that were constructed through additive