Contemporary Clinical Trials Communications最新文献

{"title":"Hydroxyurea therapy for neurological and cognitive protection in pediatric sickle cell anemia in Uganda (BRAIN SAFE II): Protocol for a single-arm open label trial","authors":"Vincent Mboizi ,&nbsp;Catherine Nabaggala ,&nbsp;Deogratias Munube ,&nbsp;John M. Ssenkusu ,&nbsp;Phillip Kasirye ,&nbsp;Samson Kamya ,&nbsp;Michael G. Kawooya ,&nbsp;Amelia Boehme ,&nbsp;Frank Minja ,&nbsp;Ezekiel Mupere ,&nbsp;Robert Opoka ,&nbsp;Caterina Rosano ,&nbsp;Richard Idro ,&nbsp;Nancy S. Green","doi":"10.1016/j.conctc.2024.101404","DOIUrl":"10.1016/j.conctc.2024.101404","url":null,"abstract":"<div><h3>Background</h3><div>Children with sickle cell anemia (SCA) in Sub-Saharan Africa are at high risk of sickle cerebrovascular injury (SCVI). Hydroxyurea, a commonly used disease-modifying therapy, may reduce SCVI resulting in potential impact on reducing stroke and cognitive dysfunction. We aim to test the impact of daily hydroxyurea therapy on these outcomes in Ugandan children with SCA. We hypothesized that hydroxyurea therapy over 36 months will prevent, stabilize or improve these complications of SCA.</div></div><div><h3>Methods</h3><div>The BRAIN SAFE II study is an open label, single arm trial of daily hydroxyurea in 270 children with SCA (HbSS) in Uganda, ages 3–9 years. Following baseline assessments, participants began hydroxyurea therapy and are followed according to local guidelines. Standard hydroxyurea dose is escalated to maximum tolerated dose (MTD). SCVI is assessed by cerebral arterial velocity using Doppler ultrasound, with cognitive function determined by formal neurocognitive testing (primary outcomes). Structural SCVI is assessed by magnetic resonance imaging (MRI) and angiography (MRA) in a sub-sample of 90 participants ages &gt;5 years. At trial midpoint (18 months) and completion (36 months), outcomes of age-specific assessments will be compared to baseline, as well as biomarkers of anemia, inflammation and malnutrition (secondary outcomes) to determine their relationships to primary outcomes.</div></div><div><h3>Conclusion</h3><div>This trial will examine the impact of hydroxyurea on preventing or ameliorating SCA SCVI in children, assessed by reducing incident stroke, stroke risk and neurocognitive dysfunction. Trial results will provide critical insight into the role of hydroxyurea therapy on critical manifestations of SCVI in children with SCA.</div></div><div><h3>Trial registration</h3><div><span><span>https://clinicaltrials.gov/ct2/show/NCT04750707</span><svg><path></path></svg></span> (registered 11 February 2021).</div></div><div><h3>Protocol version</h3><div>BRAIN SAFE II Protocol Version 3.0, Mar 02, 2022.</div></div>","PeriodicalId":37937,"journal":{"name":"Contemporary Clinical Trials Communications","volume":"42 ","pages":"Article 101404"},"PeriodicalIF":1.4,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11664125/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142883093","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"TEleRehabilitation foR Aphasia (TERRA) phase II trial design","authors":"Christy Cassarly ,&nbsp;Alexandra Basilakos ,&nbsp;Lisa Johnson ,&nbsp;Janina Wilmskoetter ,&nbsp;Jordan Elm ,&nbsp;Argye E. Hillis ,&nbsp;Leonardo Bonilha ,&nbsp;Chris Rorden ,&nbsp;Gregory Hickok ,&nbsp;Dirk-Bart den Ouden ,&nbsp;Julius Fridriksson","doi":"10.1016/j.conctc.2024.101406","DOIUrl":"10.1016/j.conctc.2024.101406","url":null,"abstract":"<div><h3>Background and purpose</h3><div>Despite comprehensive evidence that supports the utility of aphasia therapy in persons with chronic (≥6 months) stroke-induced aphasia, the amount of therapy provided to patients in the United States is typically far less than what is likely necessary to maximize recovery. Two potential contributors to this discrepancy are limited access to rehabilitation services due to the availability of providers and logistical difficulties with transportation. One way to increase access to aphasia therapy is to rely on telerehabilitation.</div></div><div><h3>Methods</h3><div>The TEleRehabilitation foR Aphasia (TERRA) trial is a prospective, randomized, rater-blinded, multicenter phase II non-inferiority trial to evaluate telerehabilitation for aphasia therapy in persons with chronic post-stroke aphasia. Participants are randomized (1:1) to receive either aphasia remote therapy or in-clinic therapy for 30 total days of treatment (15 days of a semantically focused approach and 15 days of a phonologically focused approach) for 45 min per day. A total of 100 adults (ages 21–80) with a history of left hemisphere ischemic or hemorrhagic stroke incurred at least 12 months prior to study enrollment will be randomized. The trial will be conducted at the clinical research facilities at two sites: the Medical University of South Carolina and the University of South Carolina.</div></div><div><h3>Conclusions</h3><div>This paper details the design of the TERRA trial, which aims to test whether aphasia therapy delivered by a remote speech-language pathologist through videoconferencing (i.e., via telerehabilitation) is not clinically worse than in-clinic therapy for individuals with chronic post-stroke aphasia to provide an opportunity to move to a definitive phase III trial.</div></div>","PeriodicalId":37937,"journal":{"name":"Contemporary Clinical Trials Communications","volume":"42 ","pages":"Article 101406"},"PeriodicalIF":1.4,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142757564","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Clinical study of Jianpi Qingchang decoction in the treatment of ulcerative colitis patients with spleen deficiency and dampness-heat syndrome accompanied by fatigue: Study protocol for a randomized controlled trial","authors":"Zi-Xuan Liu ,&nbsp;Xiao-Yan Liu ,&nbsp;Wei-wei Tan ,&nbsp;Wei-Bing Zhang ,&nbsp;Ya-Li Zhang ,&nbsp;Lie Zheng ,&nbsp;Yan-Cheng Dai","doi":"10.1016/j.conctc.2024.101409","DOIUrl":"10.1016/j.conctc.2024.101409","url":null,"abstract":"<div><h3>Background</h3><div>Ulcerative colitis (UC) is a chronic non-specific inflammatory intestinal disease, categoried under \"dysentery\" and \"intestinal bleeding\" in Traditional Chinese Medicine (TCM). Jianpi Qingchang decoction (JPQC) is a combination formula specifically designed for the treatment of UC. The primary objective of this study is to examine the clinical efficacy of JPQC in individuals diagnosed with UC who exhibit both spleen deficiency and dampness-heat syndrome, along with the presence of fatigue. The investigation will focus on assessing the impact of JPQC on the gut microbiota and metabolites in these patients, aiming to elucidate the regulatory mechanism that JPQC exerts on the gut microbiota and metabolites in the context of UC-related fatigue.</div></div><div><h3>Methods</h3><div>In this randomized clinical trial, 140 subjects diagnosed with UC will be recruited and randomized into two groups. They will receive either JPQC combined with mesalazine or mesalazine alone for 12 weeks. Follow-up visits will be conducted every four weeks, with a post-treatment visit scheduled at 6 months. The primary outcome measures include the Inflammatory bowel disease fatigue scale(IBD-F). Secondary efficacy indicators comprise the assessment of TCM syndrome and individual syndrome efficacy before and after treatment, Modified Mayo score, Simple clinical colitis activity index (SCCAI), as well as the Inflammatory Bowel Disease Questionnaire (IBDQ) for each group. The other outcomes are the Intestinal microbial diversity and non-targeted metabonomics, which will be measured at baseline and 12 weeks after randomization.</div></div><div><h3>Discussion</h3><div>If effective, JPQC will provide substantial clinical evidence concerning the effectiveness and safety in the treatment of patients with UC experiencing spleen deficiency and dampness-heat syndrome accompanied by fatigue.</div></div><div><h3>Trial registration</h3><div>ChiCTR2300068348.</div></div>","PeriodicalId":37937,"journal":{"name":"Contemporary Clinical Trials Communications","volume":"42 ","pages":"Article 101409"},"PeriodicalIF":1.4,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11681875/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142903724","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Involving youth with intellectual and/or developmental disabilities as collaborators in a comparative effectiveness trial: A community-engaged research approach","authors":"K.L. Berg ,&nbsp;D Herrman ,&nbsp;L Bernard ,&nbsp;C.S Shiu ,&nbsp;I Mihaila ,&nbsp;C Arnold ,&nbsp;K Acharya ,&nbsp;T.R.G Gladstone ,&nbsp;C Danguilan ,&nbsp;H Gussin ,&nbsp;P Perez ,&nbsp;A Herrman ,&nbsp;S Aaron ,&nbsp;A Thornton ,&nbsp;M Gerges ,&nbsp;C Patriarca ,&nbsp;J.J Pak ,&nbsp;B.W Van Voorhees","doi":"10.1016/j.conctc.2024.101395","DOIUrl":"10.1016/j.conctc.2024.101395","url":null,"abstract":"<div><h3>Background</h3><div>Practices to include youth with intellectual and/or developmental disabilities (IDD) are necessary to design and implement research that specifically meets the behavioral health needs of this population. This article describes a protocol for engaging youth with IDD as collaborators in a comparative effectiveness clinical trial using a community-engaged research (CEnR) approach.</div></div><div><h3>Methods</h3><div>Our engagement protocol, guided by the Community Engaged Research (CEnR) Framework, emphasized harm avoidance, accessibility, demonstrated value, capacity bridging and co-learning, shared power and equity in decision-making, accountability and respect, and transparent communication. We involved seven youth with IDD in a Youth Advisory Committee (YAC) and four youth with IDD in a Summer Scholars program, ensuring consistent and structured engagement throughout the study.</div></div><div><h3>Results</h3><div>Youth with IDD maintained high levels of engagement in both the YAC and Summer Scholars Program with 100 % retention across two years. Youth used multiple modalities to provide feedback on aspects of the research project, resulting in study modifications, the co-development of products, and tangible improvements in the accessibility and relevance of the study for youth with IDD.</div></div><div><h3>Conclusion</h3><div>Researchers and clinicians seeking to engage the historically underserved population of disabled youth in clinical trial research can leverage our findings to enhance the accessibility and inclusivity of their studies.</div></div>","PeriodicalId":37937,"journal":{"name":"Contemporary Clinical Trials Communications","volume":"42 ","pages":"Article 101395"},"PeriodicalIF":1.4,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142744572","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Comparison of crisis response planning and treatment as usual for active duty service members at risk for suicide: Study protocol for a stepped-wedge cluster randomized trial in a military treatment facility","authors":"Kristen H. Walter ,&nbsp;Pia R. Khandekar ,&nbsp;Alexander C. Kline ,&nbsp;Erin L. Miggantz ,&nbsp;Nicholas P. Otis ,&nbsp;Lisa H. Glassman ,&nbsp;Cynthia J. Thomsen ,&nbsp;Guy Brock ,&nbsp;Craig J. Bryan","doi":"10.1016/j.conctc.2024.101407","DOIUrl":"10.1016/j.conctc.2024.101407","url":null,"abstract":"<div><h3>Background</h3><div>Suicide is one of the leading causes of death among U.S. service members, and rates of suicide among military personnel have increased over the past two decades. To address this serious issue, effective preventive treatments are needed in settings where at-risk service members are frequently seen, such as emergency departments and inpatient psychiatric units. This study will compare the longitudinal effects of crisis response planning (CRP) and treatment as usual (TAU) on suicidal thoughts and behaviors among active duty service members seeking emergent care for suicidality at a military treatment facility.</div></div><div><h3>Methods</h3><div>The current study is conducted through a consortium, Augmenting Suicide Prevention Interventions for Service Members. This article details an ongoing stepped-wedge cluster randomized clinical trial that compares rates of suicidal thoughts and behaviors among service members at risk for suicide following care from CRP-trained providers versus untrained providers (i.e., TAU). Participants complete assessments at pretreatment and every 3 months up to 1 year. Primary outcomes include suicide attempts and behaviors, and suicidal ideation is a secondary outcome. Moderators of treatment effects will also be examined. The methodological development of this trial is discussed, along with clinical and ethical considerations for suicide prevention research in emergency, inpatient, and military treatment settings.</div></div><div><h3>Conclusion</h3><div>Providing evidence-based treatment for suicidality that addresses service members’ unique needs is crucial to reduce suicide rates and facilitate mental health recovery in this population. This study aims to inform future implementation and dissemination of CRP in healthcare systems to ultimately decrease suicide among service members.</div></div><div><h3>Clinical trials identifier</h3><div>NCT05795764.</div></div>","PeriodicalId":37937,"journal":{"name":"Contemporary Clinical Trials Communications","volume":"42 ","pages":"Article 101407"},"PeriodicalIF":1.4,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11681889/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142903734","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The effects of alpha-lipoic acid transdermal patch for local subcutaneous fat reduction: A randomized, placebo-controlled, clinical trial in overweight volunteers","authors":"Kanidta Sooklert ,&nbsp;Sasin Thamakaison ,&nbsp;Siwaporn Nilyai ,&nbsp;Sarocha Cherdchom ,&nbsp;Rojrit Rojanathanes ,&nbsp;Amornpun Sereemaspun","doi":"10.1016/j.conctc.2024.101402","DOIUrl":"10.1016/j.conctc.2024.101402","url":null,"abstract":"<div><h3>Background</h3><div>Combating obesity is challenging, as anti-obesity compounds lose effectiveness or cause severe side effects when delivered via conventional routes. Thus, there is a need for new, effective treatment routes that are home-based and safe for long-term use. This double-blind, placebo-controlled clinical trial aimed to investigate the efficacy of a biocellulose transdermal patch containing α-lipoic acid (ALA), an anti-obesity compound, in reducing subcutaneous fat accumulation.</div></div><div><h3>Methods</h3><div>One hundred and sixteen overweight participants (average age 37.96 ± 7.80 years) were recruited for the study. They were randomly assigned to apply either the calcium citrate nanoparticle-encapsulated ALA transdermal patch or a placebo on their arm. The participants’ body weight, height, blood lipid profile (cholesterol, triglyceride, low-density lipoprotein, and high-density lipoprotein), arm circumference, triceps skin fold, and subcutaneous fat thickness were recorded at baseline and at the 2-week follow-up.</div></div><div><h3>Results</h3><div>The mean arm circumference did not show any significant difference from baseline, whereas the triceps skinfold and subcutaneous fat thickness showed a significant reduction. The 2-week treatment did not significantly alter the plasma LDL, HDL, and triglyceride levels of the participants, but it significantly reduced the total cholesterol level.</div></div><div><h3>Conclusion</h3><div>This study reports the successful reduction of subcutaneous fat of the calcium citrate nanoparticle-encapsulated ALA transdermal patches. The transdermal patches could be used as a safe and effective home-based solution for combating obesity.</div></div>","PeriodicalId":37937,"journal":{"name":"Contemporary Clinical Trials Communications","volume":"42 ","pages":"Article 101402"},"PeriodicalIF":1.4,"publicationDate":"2024-11-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142721253","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Noninvasive anatomical assessment for ruling out hemodynamically relevant coronary artery anomalies in adults – A comparison of coronary-CT to invasive coronary angiography: The NARCO study design","authors":"Marius R. Bigler ,&nbsp;Anselm W. Stark ,&nbsp;Isaac Shiri ,&nbsp;Joel Illi ,&nbsp;Matthias Siepe ,&nbsp;Federico Caobelli ,&nbsp;Andreas A. Giannopoulos ,&nbsp;Ronny R. Buechel ,&nbsp;Andreas Haeberlin ,&nbsp;Dominik Obrist ,&nbsp;Lorenz Räber ,&nbsp;Christoph Gräni","doi":"10.1016/j.conctc.2024.101394","DOIUrl":"10.1016/j.conctc.2024.101394","url":null,"abstract":"<div><h3>Background</h3><div>Anomalous aortic origin of a coronary artery (AAOCA) is a rare congenital heart disease, potentially leading to myocardial ischemia and adverse cardiac events. As the sole presence of AAOCA does not always imply a revascularization, a detailed anatomical and functional analysis is crucial for clinical decision-making. Currently, invasive coronary angiography is the gold-standard method for a thorough hemodynamic assessment of AAOCA. However, due to its invasive nature, the development of noninvasive diagnostic alternatives is desired.</div></div><div><h3>Methods</h3><div>In the NARCO trial, patients with AAOCA will undergo coronary computed tomography angiography (CCTA) to assess anatomical high-risk features followed by a vessel-based (i.e. invasive measurement with fractional flow reserve and intravascular imaging under a dobutamine-volume challenge) and a myocardium-based (i.e. nuclear imaging) ischemia testing. Comparison of noninvasive and invasive imaging will be performed. Additionally, explorative analysis of post-processing advanced computational fluid dynamics (CFD) and 3D printing will be performed to unravel the pathophysiologic mechanism of myocardial ischemia in AAOCA.</div></div><div><h3>Aims</h3><div>Our primary aim is to define characteristics of anatomical high-risk features (using CCTA) to rule out noninvasively hemodynamically relevant anomalous vessels in AAOCA patients. The secondary aim is to investigate the underlying pathophysiology of AAOCA-related hemodynamic relevance using advanced techniques such as CFD and 3D printing.</div></div><div><h3>Conclusions</h3><div>The NARCO trial will help to optimize AAOCA patient selection for revascularization by improving risk stratification and ruling out hemodynamic relevance noninvasively and, therefore, preventing unnecessary downstream testing and/or costly interventions in patients with AAOCA.</div></div>","PeriodicalId":37937,"journal":{"name":"Contemporary Clinical Trials Communications","volume":"42 ","pages":"Article 101394"},"PeriodicalIF":1.4,"publicationDate":"2024-11-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142702607","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Defining methods to improve eSource site start-up practices","authors":"Amy E. Cramer ,&nbsp;Linda S. King ,&nbsp;Michael T. Buckley ,&nbsp;Peter Casteleyn ,&nbsp;Cory Ennis ,&nbsp;Muayad Hamidi ,&nbsp;Gonçalo M.C. Rodrigues ,&nbsp;Denise C. Snyder ,&nbsp;Aruna Vattikola ,&nbsp;Eric L. Eisenstein","doi":"10.1016/j.conctc.2024.101391","DOIUrl":"10.1016/j.conctc.2024.101391","url":null,"abstract":"<div><h3>Background</h3><div>eSource software that transfers patient electronic health record data into a clinical trial electronic case report form holds promise for increasing data quality while reducing data collection, monitoring and source document verification costs. Integrating eSource into multicenter clinical trial start-up procedures could facilitate the use of eSource technologies in clinical trials.</div></div><div><h3>Methods</h3><div>We conducted a qualitative integrative analysis to identify eSource site start-up key steps, challenges that might occur in executing those steps, and potential solutions to those challenges. We then conducted a value analysis to determine the challenges and solutions with the greatest impacts for eSource implementation teams.</div></div><div><h3>Results</h3><div>There were 16 workshop participants: 10 pharmaceutical sponsor, 3 academic site, and 1 eSource vendor representative. Participants identified 36 Site Start-Up Key Steps, 11 Site Start-Up Challenges, and 14 Site Start-Up Solutions for eSource-enabled studies. Participants also identified 77 potential impacts of the Challenges upon the Site Start-Up Key Steps and 70 ways in which the Solutions might impact Site Start-Up Challenges. The most important Challenges were: [1] not being able to identify a site eSource champion and [2] not agreeing on an eSource approach. The most important Solutions were: [1] eSource vendors accepting electronic data in the Health Level 7 Fast Healthcare Interoperability Resources (HL7® FHIR®) standard, [2] creating standard content for eSource-related legal documents, and [3] creating a common eSource site readiness checklist.</div></div><div><h3>Conclusions</h3><div>Site start-up for eSource-enabled multi-center clinical trials is a complex socio-technical problem. This study's Start-Up Solutions provide initial steps for scalable eSource implementation.</div></div>","PeriodicalId":37937,"journal":{"name":"Contemporary Clinical Trials Communications","volume":"42 ","pages":"Article 101391"},"PeriodicalIF":1.4,"publicationDate":"2024-11-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142702692","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A Bayesian adaptive feasibility design for rare diseases","authors":"Maureen M. Churipuy ,&nbsp;Shirin Golchi ,&nbsp;Marie Hudson ,&nbsp;Sabrina Hoa","doi":"10.1016/j.conctc.2024.101392","DOIUrl":"10.1016/j.conctc.2024.101392","url":null,"abstract":"<div><div>It is important for researchers to carefully assess the feasibility of a clinical trial prior to the launch of the study. One feasibility aspect that needs to be considered includes whether investigators can expect to successfully achieve the sample size needed for their trial. In this manuscript, we present a Bayesian design in which data collected during a pilot study is used to predict the feasibility of a planned phase III trial. Specifically, we outline a model that predicts a target sample size obtained from the Gamma-Poisson distribution. In a simulation study, we showcase the utility of the proposed design by applying it to a phase III trial designed to assess the efficacy of mycophenolate mofetil in individuals with mild systemic sclerosis. We demonstrate that the predictive nature of the proposed design is particularly useful for rare disease clinical trials and has the potential to greatly increase their efficiency.</div></div>","PeriodicalId":37937,"journal":{"name":"Contemporary Clinical Trials Communications","volume":"42 ","pages":"Article 101392"},"PeriodicalIF":1.4,"publicationDate":"2024-11-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142652494","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Reasons for declining participation in inpatient research among historically minoritized racial and ethnic communities: A scoping review","authors":"Poyani Bavishi ,&nbsp;Alyssa A. Grimshaw ,&nbsp;Oscar F. Rojas Perez ,&nbsp;Brian D. Kiluk ,&nbsp;E. Jennifer Edelman","doi":"10.1016/j.conctc.2024.101386","DOIUrl":"10.1016/j.conctc.2024.101386","url":null,"abstract":"<div><h3>Background</h3><div>To promote equitable recruitment for studies conducted in the inpatient hospital setting, we sought to characterize reasons why individuals, both from historically minoritized racial and ethnic groups and the broader patient population, refuse participation in clinical trials within inpatient settings.</div></div><div><h3>Methods</h3><div>An exhaustive search of the literature was conducted in Cochrane Library, Google Scholar, Embase, MEDLINE, PubMed, Scopus, and Web of Science databases to find relevant articles published from the inception of each database to April 30, 2023. Studies recruiting patients during their inpatient stay and reporting reasons for refusing participation in clinical trials met the inclusion criteria.</div></div><div><h3>Results</h3><div>The search resulted in 2264 citations, of which 22 were included. Fourteen did not report data related to race, while 19 reported no ethnicity data. Reasons for refusal across trials included study burden and inconvenience (n = 16), transportation and logistical issues (n = 13), lack of interest in research (n = 12), and refusal to be randomized (n = 10). Prominent concepts included the importance of incorporating social support systems in consenting processes, lack of efforts to include data or recruitment efforts for individuals from minoritized groups, and physician involvement in recruitment.</div></div><div><h3>Discussion</h3><div>To enhance participation among historically minoritized communities in clinical trials, greater efforts must be made to collect demographic information and document refusal reasons to inform future recruitment methods. Strategies include proactively accounting for culture and language differences in study design and recruitment and intentionally engaging social support networks. Limiting study burden and logistics and optimizing collaborations between clinical and research teams would promote accessibility and foster patient trust.</div></div>","PeriodicalId":37937,"journal":{"name":"Contemporary Clinical Trials Communications","volume":"42 ","pages":"Article 101386"},"PeriodicalIF":1.4,"publicationDate":"2024-11-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142702608","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}