Contemporary clinical trials最新文献

{"title":"Protocol for the implementation and referral of the ecofit physical activity intervention within Diabetes Alliance Program Plus among regional, rural and remote people with type 2 diabetes in a primary care setting","authors":"Anna K. Jansson ,&nbsp;Sam Beacroft ,&nbsp;Mitch J. Duncan ,&nbsp;Emily R. Cox ,&nbsp;Sara L. Robards ,&nbsp;Wendy Ferris ,&nbsp;Alexis Hure ,&nbsp;Shamasunder Acharya ,&nbsp;Ronald C. Plotnikoff","doi":"10.1016/j.cct.2024.107774","DOIUrl":"10.1016/j.cct.2024.107774","url":null,"abstract":"<div><h3>Background</h3><div>This paper outlines the protocol for the <em>ecofit</em> implementation-effectiveness trial, a multi-component mobile Health (mHealth) intervention that aims to increase resistance and aerobic physical activity in primary care-based adults with type 2 diabetes (T2D). This study will be conducted as part of the Diabetes Alliance Program Plus (DAP+), a large-scale integrated health service intervention in a large health district in Australia. The <em>ecofit</em> program has previously demonstrated efficacy and effectiveness in insufficiently active people with (or at risk of) T2D and community dwelling-adults, respectively. The aim of this study is to assess the reach (primary outcome), adoption, appropriateness, feasibility and fidelity of the implementation of <em>ecofit</em> and the overall effectiveness of the intervention.</div></div><div><h3>Research design and methods</h3><div>Prospective participants are adults diagnosed with T2D, who attend primary care settings enrolled in DAP+, and are identified and referred to <em>ecofit</em> by a primary care clinician. To support the implementation of <em>ecofit</em> a host of strategies will be utilised, which includes the education and upskilling of primary care clinicians enrolled in DAP+ using brief training sessions, the supply of an information package and access to professional development. The co-primary outcomes of reach will be defined as the number of participant registrations on the <em>ecofit</em> platform and the number of primary care clinicians who have been introduced to <em>ecofit</em>.</div></div><div><h3>Conclusion</h3><div>This study will evaluate the implementation of <em>ecofit</em> among adults with T2D within the primary care setting. The results may help improve T2D lifestyle interventions in primary care settings across Australia.</div></div>","PeriodicalId":10636,"journal":{"name":"Contemporary clinical trials","volume":"148 ","pages":"Article 107774"},"PeriodicalIF":2.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142791198","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Family history and cancer risk study (FOREST): A clinical trial assessing electronic patient-directed family history input for identifying patients at risk of hereditary cancer","authors":"Kathleen F. Mittendorf ,&nbsp;Harris T. Bland ,&nbsp;Justin Andujar ,&nbsp;Natasha Celaya-Cobbs ,&nbsp;Clasherrol Edwards ,&nbsp;Meredith Gerhart ,&nbsp;Gillian Hooker ,&nbsp;Mryia Hubert ,&nbsp;Sarah H. Jones ,&nbsp;Dana R. Marshall ,&nbsp;Rachel A. Myers ,&nbsp;Siddharth Pratap ,&nbsp;S. Trent Rosenbloom ,&nbsp;Azita Sadeghpour ,&nbsp;R. Ryanne Wu ,&nbsp;Lori A. Orlando ,&nbsp;Georgia L. Wiesner","doi":"10.1016/j.cct.2024.107714","DOIUrl":"10.1016/j.cct.2024.107714","url":null,"abstract":"<div><h3>Background</h3><div>Hereditary cancer syndromes cause a high lifetime risk of early, aggressive cancers. Early recognition of individuals at risk can allow risk-reducing interventions that improve morbidity and mortality. Family health history applications that gather data directly from patients could alleviate barriers to risk assessment in the clinical appointment, such as lack of provider knowledge of genetics guidelines and limited time in the clinical appointment. New approaches allow linking these applications to patient health portals and their electronic health records (EHRs), offering an end-to-end solution for patient-input family history information and risk result clinical decision support for their provider.</div></div><div><h3>Methods</h3><div>We describe the design of the first large-scale evaluation of an EHR-integrable, patient-facing family history software platform based on the Substitutable Medical Applications and Reusable Technologies on Fast Healthcare Interoperability Resources (SMART on FHIR) standard. In our study, we leverage an established implementation science framework to evaluate the success of our model to facilitate scalable, systematic risk assessment for hereditary cancers in diverse clinical environments in a large pragmatic study at two sites. We will also evaluate the success of the approach to improve the efficiency of downstream genetic counseling resulting from pre-counseling pedigree generation.</div></div><div><h3>Conclusions</h3><div>Our research study will provide evidence regarding a new care delivery model that is scalable and sustainable for a variety of medical centers and clinics.</div></div><div><h3>Trial registration</h3><div>This study was registered on <span><span>ClinicalTrials.gov</span><svg><path></path></svg></span> under <span><span>NCT05079334</span><svg><path></path></svg></span> on 15 October 2021.</div></div>","PeriodicalId":10636,"journal":{"name":"Contemporary clinical trials","volume":"148 ","pages":"Article 107714"},"PeriodicalIF":2.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142459890","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Individualized Coordination and Empowerment for Care Partners of Persons with Dementia (ICECaP): Feasibility and acceptability","authors":"Ryan C. Thompson ,&nbsp;Virginia T. Gallagher ,&nbsp;Shannon E. Reilly ,&nbsp;Anna M. Arp ,&nbsp;Carol A. Manning","doi":"10.1016/j.cct.2024.107770","DOIUrl":"10.1016/j.cct.2024.107770","url":null,"abstract":"<div><h3>Background</h3><div>Informal care partners (CPs) of persons with dementia (PWDs), who are at risk of negative health outcomes, benefit from psychosocial interventions. Individualized Coordination and Empowerment for CPs of PWDs (ICECaP) is a year-long, multi-component intervention comprised of in-person and telehealth psychoeducation and emotional support from dementia care coordinators (DCCs). ICECaP feasibility and acceptability were examined during a pilot randomized controlled trial.</div></div><div><h3>Method</h3><div>Feasibility was determined by study enrollment (relative to screening) and study completion. Acceptability was examined with CP-rated intervention satisfaction and DCC-completed surveys of CP engagement, using one-sample Wilcoxon tests to evaluate differences between observed and null hypothesized medians. Implementation fidelity was also evaluated. Analyses of variance and Chi-square tests identified demographic differences in study completion and DCC-reported CP engagement.</div></div><div><h3>Results</h3><div>Study enrollment (91.4 %) and study completion (85.4 %) were both high when compared to findings from a recent meta-analysis of CP psychosocial interventions. Completion was similar across groups (ICECaP vs. control) and demographics. CPs and DCCs communicated twice monthly, most commonly via email. On average, DCCs spent 68 minutes total per CP monthly; the number and duration of contacts varied widely. CPs responded positively on the satisfaction survey, and DCCs mostly responded positively about CP communication, engagement, and responsiveness. Communication was reportedly more difficult with employed CPs and CPs with ≥16 years of education.</div></div><div><h3>Conclusion</h3><div>ICECaP was both feasible and acceptable. DCC time spent with CPs occurred primarily virtually and varied widely, reflecting both individualization within the intervention and the unpredictability of dementia care for CPs.</div><div>ClinicalTrials.govNCT04495686.</div></div>","PeriodicalId":10636,"journal":{"name":"Contemporary clinical trials","volume":"148 ","pages":"Article 107770"},"PeriodicalIF":2.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142778945","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Recruiting sexual minority youth for a drug abuse prevention trial: Comparing Instagram and Facebook","authors":"Traci M. Schwinn ,&nbsp;Margaret Weisblum ,&nbsp;Emma Trussell ,&nbsp;Rachel Yamshon ,&nbsp;Dina Sheira","doi":"10.1016/j.cct.2024.107772","DOIUrl":"10.1016/j.cct.2024.107772","url":null,"abstract":"<div><h3>Background</h3><div>Despite youth's shift from Facebook to Instagram, the literature on how to use Instagram to recruit youth for clinical trials is scant. This paper reports procedures and comparative metrics on the use of Facebook and Instagram to recruit a nationwide sample (<em>N =</em> 1216) of LGBQ youth, aged 15 and 16 years, for an online drug abuse prevention trial.</div></div><div><h3>Methods</h3><div>Our recruitment campaign used ads on Facebook and promoted posts on Instagram. Ads and posts shared common images, headlines, and captions. Ads and posts directed youth to a study recruitment website for informed consent and enrollment procedures.</div></div><div><h3>Results</h3><div>Our campaign ran for 48 non-consecutive days, yielded <em>N =</em> 1216 participants, had a total cost of $25,400.31, and an average cost per participant of $20.89. Facebook ads and Instagram promoted posts ran for a similar number of days. However, compared to Instagram, Facebook ads cost more than twice as much (115 %), had 51 % fewer clicks, and had an average cost-per-click that was 338 % higher. Furthermore, despite being shown to users more than 4 times as often and garnering more than twice as many unique views, Facebook ads had a click-through-rate that was 90 % lower than Instagram promoted posts.</div></div><div><h3>Conclusion</h3><div>Instagram promoted posts outperformed ads on Facebook by driving more potential participants to the study recruitment website and for less money.</div></div>","PeriodicalId":10636,"journal":{"name":"Contemporary clinical trials","volume":"148 ","pages":"Article 107772"},"PeriodicalIF":2.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142778985","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Evaluation of patient and encounter decision aid interventions for atrial fibrillation: Baseline characteristics of the RED-AF study - A Randomized Controlled Trial","authors":"Tanvi Nayak BA ,&nbsp;Joshua T. Christensen PhD ,&nbsp;Tyler Bardsley MS ,&nbsp;Geoffrey D. Barnes MD, MSc ,&nbsp;Kenzie A. Cameron PhD, MPH ,&nbsp;Rod Passman MD, MSCE ,&nbsp;Preeti Kansal MD ,&nbsp;Daniel M. Witt PharmD, FCCP, BCPS ,&nbsp;Kerri L. Cavanaugh MD, MHS ,&nbsp;Angela Fagerlin PhD ,&nbsp;Elissa M. Ozanne PhD ,&nbsp;the STEP-UP AF Writing Group","doi":"10.1016/j.cct.2024.107773","DOIUrl":"10.1016/j.cct.2024.107773","url":null,"abstract":"<div><h3>Background</h3><div>The Randomized Evaluation of Decision Support Interventions for Atrial Fibrillation (RED-AF) trial is a multi-site, randomized controlled clinical trial examining the effectiveness of a patient decision aid and an encounter decision aid in promoting shared decision-making (SDM) during a clinical encounter for patients with atrial fibrillation (AF). We sought to describe baseline characteristics of patients and clinicians in the trial and compare them to the demographics of the larger AF population. We also conducted an analysis of possible predictors of attrition rates at baseline, 6 and 12 months.</div></div><div><h3>Methods</h3><div>This study was a multi-center randomized controlled trial conducted at six academic centers across the U.S. Patients with non-valvular AF who qualify for anticoagulation therapy were eligible for enrollment. Patient demographics and characteristics were evaluated via questionnaires after their baseline clinical encounter. Participating clinicians completed demographic surveys, reporting educational background, specialty, and years of experience. Patient characteristics were analyzed via univariate logistic regression to identify potential trends among those lost to follow-up at each timepoint.</div></div><div><h3>Findings</h3><div>A total of 1117 patients were enrolled in the RED-AF trial, with an average age of 69 (SD 9.3). Patients were predominantly male (61.7 %) and white (89.1 %), with 33.7 % reporting graduate or professional education. Clinicians (<em>N</em> = 107) were enrolled from specialties including cardiology (68.2 %), internal medicine (13.1 %), and pharmacy (14.0 %). No significant associations were found between any measured patient characteristics with survey completion at baseline, 6 or 12 months.</div></div><div><h3>Conclusion</h3><div>The baseline demographics of the RED-AF trial reflect that patient participants were largely similar to prior studies investigating shared-decision making in patients with AF. The lack of association between patient demographics and attrition rates may highlight equity across the tested subgroups for survey completion for the study as a whole.</div></div>","PeriodicalId":10636,"journal":{"name":"Contemporary clinical trials","volume":"148 ","pages":"Article 107773"},"PeriodicalIF":2.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142791197","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"PLAN—Dementia literacy education and navigation for Korean elders with probable dementia and their caregivers: Rationale, methods, and design of a community-based, randomized, controlled, multi-site clinical trial","authors":"Hae-Ra Han ,&nbsp;Nancy Perrin ,&nbsp;Simona C. Kwon ,&nbsp;Jinhui Joo ,&nbsp;Ji-Young Yun ,&nbsp;Deborah Min ,&nbsp;Hochang Benjamin Lee","doi":"10.1016/j.cct.2024.107771","DOIUrl":"10.1016/j.cct.2024.107771","url":null,"abstract":"<div><h3>Background</h3><div>Linkage to medical services is key to early detection, diagnosis, and care for dementia. Yet, racial and ethnic minoritized older adults and their caregivers are particularly vulnerable to inequity in dementia care due to limited dementia literacy. Mobilizing community resources such as community health workers (CHWs) can benefit older patients and their caregivers who are challenged by linguistic barriers and low health literacy.</div></div><div><h3>Methods</h3><div>Preparing for healthy aging through dementia Literacy education And Navigation (PLAN), is a CHW-led intervention program designed to promote linkage to medical services for dementia and caregiver outcomes in a multi-site clinical trial in the Greater Washington and New York metropolitan areas. This protocol describes the rationale, design, and methods of the PLAN trial.</div></div><div><h3>Conclusion</h3><div>We recruited 288 dyads of eligible Korean American older adults with undiagnosed dementia and their caregivers, totaling 576 participants. Data collection is ongoing. Findings from this trial will contribute to knowledge around identifying community-dwelling older adults with probable dementia with limited health resources as well as navigating appropriate medical evaluation and support.</div><div><span><span>Clinicaltrials.gov</span><svg><path></path></svg></span> identifier: <span><span>NCT03909347</span><svg><path></path></svg></span></div></div>","PeriodicalId":10636,"journal":{"name":"Contemporary clinical trials","volume":"148 ","pages":"Article 107771"},"PeriodicalIF":2.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142766796","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Unsupervised clustering approach to assess heterogeneity of treatment effects across patient phenotypes in randomized clinical trials","authors":"Andrea Bellavia,&nbsp;Xinhui Ran,&nbsp;Andre Zimerman,&nbsp;Elliott M. Antman,&nbsp;Robert P. Giugliano,&nbsp;David A. Morrow,&nbsp;Sabina A. Murphy","doi":"10.1016/j.cct.2024.107778","DOIUrl":"10.1016/j.cct.2024.107778","url":null,"abstract":"<div><h3>Background</h3><div>Primary results from randomized clinical trials (RCT) only inform on the average treatment effect in the studied population, and it is critical to understand how treatment effect varies across subpopulations. In this paper we describe a clustering-based approach for the assessment of Heterogeneity of Treatment Effect (HTE) over patient phenotypes, which maintains the unsupervised nature of classical subgroup analysis while jointly accounting for relevant patient characteristics.</div></div><div><h3>Methods</h3><div>We applied phenotype-based stratification in the ENGAGE AF-TIMI 48 trial, a non-inferiority trial comparing the effects of higher-dose edoxaban regimen (direct anticoagulant) versus warfarin (vitamin K antagonist) on a composite endpoint of stroke and systemic embolism in 14,062 patients with atrial fibrillation.</div></div><div><h3>Results</h3><div>We identified three distinct phenotypes: non-white participants, mostly from Asia (A); white participants without previous use of vitamin-K antagonists (B); and white participants with previous use of vitamin-K antagonist (C). The effect of the higher-dose edoxaban regimen vs warfarin significantly varied over phenotypes (p for interaction = 0.03) with the strongest benefit in cluster A (HR = 0.72, 95 % CI: 0.52–1.00), moderate effect in cluster B (HR = 0.80, 95 % CI: 0.61, 1.06) and no observed effect in cluster C (HR = 1.01, 95 % CI: 0.80, 1.27).</div></div><div><h3>Conclusions</h3><div>Assessing HTE over patients' phenotypes might represent a relevant complement to other stratification approaches to elucidate results from subgroups analyses, especially in those settings where an overwhelming superiority overall effect was not observed. Cluster analysis allows a clear discrimination of patients with direct interpretability of who are the patients that would most benefit from the investigated strategy or treatment.</div></div>","PeriodicalId":10636,"journal":{"name":"Contemporary clinical trials","volume":"148 ","pages":"Article 107778"},"PeriodicalIF":2.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142827622","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Factors associated with HIV self-testing and PrEP use among Nigerian youth: Baseline outcomes of a pragmatic, stepped-wedge, cluster-randomized controlled trial","authors":"Juliet Iwelunmor ,&nbsp;Ebenezer Adeoti ,&nbsp;Titilola Gbaja-Biamila ,&nbsp;Ucheoma Nwaozuru ,&nbsp;Chisom Obiezu-Umeh ,&nbsp;Adesola Z. Musa ,&nbsp;Hong Xian ,&nbsp;Weiming Tang ,&nbsp;David Oladele ,&nbsp;Collins O. Airhihenbuwa ,&nbsp;Nora Rosenberg ,&nbsp;Donaldson F. Conserve ,&nbsp;Franklin Yates ,&nbsp;Temitope Ojo ,&nbsp;Oliver Ezechi ,&nbsp;Joseph D. Tucker","doi":"10.1016/j.cct.2024.107733","DOIUrl":"10.1016/j.cct.2024.107733","url":null,"abstract":"<div><h3>Introduction</h3><div>Adolescents and young adults (AYA, 14–24 years) bear a disproportionate burden of new HIV infections in Nigeria and are more likely to have worse HIV outcomes compared to other age groups. However, little is known about their access to recommended sexual health care services, including HIV self-testing (HIVST), sexually transmitted infections (STI) testing, sexual behavior patterns, awareness and or access to pre-exposure prophylaxis (PrEP), and overall risk for HIV.</div></div><div><h3>Methods</h3><div>We present a baseline analysis of the 4 Youth by Youth randomized controlled trial aimed to evaluate the uptake and sustainability of crowdsourced HIVST strategies led by and for young people across 14 states in Nigeria. None of the participants had received intervention at the time of completing this self-reported behavioral survey. We conducted a descriptive analysis to summarize participants' characteristics, sexual behavior, HIV testing, STI testing, and knowledge of PrEP and use across the study sample of the AYAs. We conducted a chi-square test, and the level of significance was set at ≤0.05.</div></div><div><h3>Results</h3><div>A total of 1551 participants completed the baseline survey comprising males (777, 50.1 %) and females (774, 49.9 %). The majority (77 %) of the participants were students at enrollment. Very few of the participants had ever tested for syphilis, 47 (3.1 %), gonorrhea, 49 (3.2 %), chlamydia, 31 (2.0 %), and hepatitis B, 106 (6.9 %). 678 (43.8 %) of the participants reported to be sexually active at the time of enrolment into the study, of which about 38 % of them engaged in condomless sex. Paying for sex, alcohol use, and drug use are all significant sexual behaviors (<em>p</em> &lt; 0.01). Only 14 (1 %) have ever used PrEP. 481 (31.4 %) have ever tested for HIV, and 104 (6.8 %) have ever used an HIV self-testing kit at baseline. 457 (38.6 %) were eligible for PrEP.</div></div><div><h3>Conclusions</h3><div>HIVST and STI uptake were low at baseline among the AYA in this study. Most AYAs also do not receive recommended sexual health care services, including STI testing services. This underlines the need for interventions to increase the uptake of HIV/STI prevention services among Nigerian AYA.</div></div>","PeriodicalId":10636,"journal":{"name":"Contemporary clinical trials","volume":"148 ","pages":"Article 107733"},"PeriodicalIF":2.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142638676","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Corrigendum to ‘Integrative data analysis of clinical trials network studies to examine the impact of psychosocial treatments for Black people who use cocaine: Study protocol’ [Contemporary Clinical Trials 133 (2023) 107329].","authors":"Angela M. Haeny ,&nbsp;Caravella McCuistian ,&nbsp;A. Kathleen Burlew ,&nbsp;Lesia M. Ruglass ,&nbsp;Adriana Espinosa ,&nbsp;Ayana Jordan ,&nbsp;Christopher Roundtree ,&nbsp;Joel Lopez ,&nbsp;Antonio A. Morgan-Lopez","doi":"10.1016/j.cct.2024.107740","DOIUrl":"10.1016/j.cct.2024.107740","url":null,"abstract":"","PeriodicalId":10636,"journal":{"name":"Contemporary clinical trials","volume":"147 ","pages":"Article 107740"},"PeriodicalIF":2.0,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142692806","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Practical considerations of promising zone design for interim sample size Re-estimation: An application to GRAPHITE for graft vs host disease","authors":"Jingjing Chen ,&nbsp;Yohei Takanami ,&nbsp;Johan Jansson ,&nbsp;Guillermo Rossiter","doi":"10.1016/j.cct.2024.107765","DOIUrl":"10.1016/j.cct.2024.107765","url":null,"abstract":"<div><h3>Background</h3><div>Sample size calculation and power estimate are an integral part of clinical trials. With accelerated development to address the unmet medical needs, the fast-paced development may lead to uncertainties in initial planning and assumptions of clinical trials. Promising zone design presents sponsors an opportunity to re-estimate the sample size based on the interim data to mitigate risks, reduce uncertainties, and increase probability of trial success.</div></div><div><h3>Methods</h3><div>This paper aims to use the GRAPHITE trial (<span><span>NCT03657160</span><svg><path></path></svg></span>) as a real data application to showcase the practical considerations in implementation of promising zone design for interim sample size re-estimation (SSR), in light of sample size adaptation rules, maximum sample size allowed, multiplicity adjustment, and sponsor access to interim results. GRAPHITE is a phase 3 trial with vedolizumab for prophylaxis of acute graft vs host disease (aGvHD) after allogeneic hematopoietic stem cell transplant (allo-HSCT). The primary efficacy endpoint is lower intestinal aGVHD-free survival by Day +180 after allo-HSCT. A simulation study was conducted to demonstrate the evaluation of operating characteristics by various true underlying treatment effects at the design stage.</div></div><div><h3>Conclusion</h3><div>The application of promising zone design for interim SSR is novel and has successfully helped the sponsor achieve the balance between minimizing the risks and maintaining scientific integrity. This work aims to highlight the necessity of empirical guidance to gain better insights for clinical researchers in practice and is expected to facilitate the understanding and implementation of promising zone design for interim SSR in phase 3 trials.</div></div>","PeriodicalId":10636,"journal":{"name":"Contemporary clinical trials","volume":"148 ","pages":"Article 107765"},"PeriodicalIF":2.0,"publicationDate":"2024-11-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142738686","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}