Contemporary clinical trials最新文献

{"title":"Workforce and systems change to treat adolescent substance use disorder within integrated pediatric primary care: A cluster-randomized stepped-wedge trial","authors":"Leslie A. Hulvershorn ,&nbsp;Matthew Aalsma ,&nbsp;Trey V. Dellucci ,&nbsp;Ashlyn Burns ,&nbsp;Brigid R. Marriott ,&nbsp;Bernice Pescosolido ,&nbsp;Harold D. Green Jr. ,&nbsp;Lisa Saldana ,&nbsp;Jason Chapman ,&nbsp;Patrick Monahan ,&nbsp;Sarah E. Wiehe ,&nbsp;Edward J. Miech ,&nbsp;Zachary W. Adams","doi":"10.1016/j.cct.2025.107928","DOIUrl":"10.1016/j.cct.2025.107928","url":null,"abstract":"<div><h3>Background</h3><div>While the overdose crisis has impacted all ages, overdose-related deaths among adolescents have been increasing more rapidly than any other age group, doubling between 2019 and 2020. Identifying and treating substance use disorders (SUDs) among adolescents is critical to preventing adolescent overdose deaths. While evidence-based interventions for adolescents with SUDs exist, they remain underutilized. Implementing SUD interventions in primary care settings through integrated behavioral health (IBH) is one approach for increasing access to evidence-based SUD services for adolescents.</div></div><div><h3>Methods</h3><div>This is a Hybrid Type 2, cluster-randomized, stepped-wedge trial comparing SUD IBH to standard primary care treatment. In our open cohort stepped-wedge design, primary care clinics will be randomly designated to one of three cohorts. We will use a mixed-methods approach to evaluate both implementation and effectiveness outcomes, with a focus on assessing the impact of IBH on primary care provider behaviors around SUD interventions. All cohorts will complete baseline surveys during the control condition and then every 6 months. At each time point, we will also collect and analyze patient administrative data to assess patient engagement and outcomes. In addition, we will conduct qualitative interviews at pre-, mid-, and post-implementation during sustainment of the intervention.</div></div><div><h3>Conclusion</h3><div>Addressing the overdose crisis is a national priority. IBH has the potential to reduce overdose rates by enhancing primary care provider willingness to deliver SUD services for adolescents.</div></div>","PeriodicalId":10636,"journal":{"name":"Contemporary clinical trials","volume":"154 ","pages":"Article 107928"},"PeriodicalIF":2.0,"publicationDate":"2025-04-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143892153","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":"Improving data credibility in online recruitment: Signs and strategies for detecting fraudulent participants when using ResearchMatch","authors":"Lauren M. Pageau,&nbsp;Jiying Ling","doi":"10.1016/j.cct.2025.107925","DOIUrl":"10.1016/j.cct.2025.107925","url":null,"abstract":"<div><h3>Introduction</h3><div>Online recruitment platforms are valuable tools that allow researchers to efficiently reach large pools of potential participants for clinical trials and observational studies. However, challenges associated with fraudulent respondents and bots threaten data credibility. This paper discusses signs we identified for recognizing potentially fraudulent respondents when using one online recruitment tool, ResearchMatch, to recruit study participants.</div></div><div><h3>Methods</h3><div>Participants were recruited via ResearchMatch for a study focused on stress and coping among U.S. young, low-income parents. Participants completed an online survey via Qualtrics. To screen for fraudulent respondents, we compared reports generated from Qualtrics with those from ResearchMatch.</div></div><div><h3>Results</h3><div>We identified six signs of fraudulent respondents, including 1) suspicious metadata, 2) fake addresses listed in ResearchMatch profile, 3) a common Western name for both first and last names, 4) inconsistent and duplicate data, 5) extensive list of medications and/or medical conditions, and 6) short survey completion time. We contacted 63,284 accounts through ResearchMatch and received 928 survey responses. About 46 % (<em>n</em> = 425) of responses were deemed fraudulent.</div></div><div><h3>Conclusions</h3><div>Fraudulent respondents and bots undermine the integrity of data, which may result in adverse implications for research, policy, and patient health. Given that nearly half of the survey respondents in our study were deemed fraudulent, it is evident that this is a significant issue for recruiting research participants via online recruitment platforms. To ensure reliability and accuracy of study findings, it is critical for researchers to thoroughly examine their data for signs of fraudulence.</div></div>","PeriodicalId":10636,"journal":{"name":"Contemporary clinical trials","volume":"154 ","pages":"Article 107925"},"PeriodicalIF":2.0,"publicationDate":"2025-04-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143886426","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":"OUTPACE: Outcomes of urinary incontinence treatment in primary care – APP co-management and electronic consult","authors":"C. Pangelina ,&nbsp;J. Bergman ,&nbsp;T. Cheng ,&nbsp;R. Fantus ,&nbsp;A. Galvez ,&nbsp;G. Gin ,&nbsp;T. Grisales ,&nbsp;J. Koola ,&nbsp;E.S. Lukacz ,&nbsp;M. Millen ,&nbsp;A. Moore ,&nbsp;K. Okamuro ,&nbsp;D. Reuben ,&nbsp;Y. Santiago-Lastra ,&nbsp;J. Singer ,&nbsp;M. Suarez ,&nbsp;M. Tai-Seale ,&nbsp;F. Vaida ,&nbsp;C. Souders ,&nbsp;J. Anger","doi":"10.1016/j.cct.2025.107922","DOIUrl":"10.1016/j.cct.2025.107922","url":null,"abstract":"<div><h3>Introduction</h3><div>Quality of care (QOC) research surrounding urinary incontinence (UI) management is limited, yet electronic (<em>E</em>-consult) and Advanced Practice Provider (APP) co-managements have proven QOC impact. We describe the design and methodology of a cluster randomized comparative effectiveness of these primary care provider (PCP) referral practices.</div></div><div><h3>Methods</h3><div>Outcomes of Urinary Incontinence Treatment in Primary Care – APP Co-management and Electronic Consult (OUTPACE) is a pragmatic clinical trial being conducted at the University of California – San Diego (UCSD), the University of California – Los Angeles (UCLA), and the University of Kansas Medical Center (KU). Approximately 300 providers within 60 PCP offices are randomized to utilize either <em>E</em>-consult or APP co-management. The first aim is to compare the relative effectiveness of these two referral mechanisms in improving UI care over a six-month period. The second and third aims include patient-reported outcomes (PROs) using validated instruments, patient knowledge, and participation in shared decision making. Secondary analyses will identify potential disparities in UI QOC related to race, ethnicity, and spoken language.</div></div><div><h3>Results</h3><div>Provider and patient enrollment began in February 2024 and is ongoing. Baseline provider QOC is assessed through retrospective chart review of 3–5 patient visits with an ICD-10 code for UI. PROs are assessed at baseline, three-month, and six-month timepoints. Both provider and patient knowledge is measured using a Pelvic Floor Awareness and Knowledge Survey at baseline and six-month timepoints.</div></div><div><h3>Conclusions</h3><div>OUTPACE will determine whether the <em>E</em>-consult or APP co-management referral mechanism has a greater impact on provider QOC, patient-reported UI outcomes, and shared decision-making scores.</div></div>","PeriodicalId":10636,"journal":{"name":"Contemporary clinical trials","volume":"153 ","pages":"Article 107922"},"PeriodicalIF":2.0,"publicationDate":"2025-04-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143869390","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":"A Flexible Individualised ExeRcise programme for cancer patients during ChEmotherapy (FIERCE): Protocol for a randomised controlled feasibility trial","authors":"Neil Kearney ,&nbsp;Deirdre Connolly ,&nbsp;Katayoun Bahramian ,&nbsp;Grainne Sheill ,&nbsp;Kelly Coghlan-Lynch ,&nbsp;Jacintha O'Sullivan ,&nbsp;Niamh Coleman ,&nbsp;Ciara O'Hanlon Brown ,&nbsp;David Gallagher ,&nbsp;Catherine O'Gorman ,&nbsp;Catherine O'Brien ,&nbsp;Antonia Tierney ,&nbsp;Kate Rankin ,&nbsp;Linda O'Neill ,&nbsp;Emer Guinan","doi":"10.1016/j.cct.2025.107923","DOIUrl":"10.1016/j.cct.2025.107923","url":null,"abstract":"<div><h3>Background</h3><div>Exercise is an important tool which has been shown to help patients manage many of the side effects of their cancer treatment, reduce toxicities, and improve prognosis. The benefits of exercise have been well documented, however, performing regular exercise during treatment remains a challenge for most patients. The Flexible Individualised ExeRcise programme for cancer patients during ChEmotherapy (FIERCE) is an exercise programme that has been co-designed by healthcare professionals and people with a personal lived experience of chemotherapy. The primary aim of this study is to examine the feasibility of delivering the FIERCE programme for cancer patients during chemotherapy.</div></div><div><h3>Methods</h3><div>The FIERCE study is a randomised controlled feasibility trial which will include 50 participants who are scheduled to receive chemotherapy for the treatment of breast, colorectal, or ovarian cancer. Participants will be randomly allocated to Group 1: FIERCE programme, or Group 2: Self-managed pedometer programme in a 2:1 ratio. Participants will be enrolled in the study for the duration of their chemotherapy treatment. The primary outcome of feasibility will be measured using a mixed-methods approach. Secondary outcomes of cardiorespiratory fitness, muscular strength, skeletal muscle mass, physical function, fatigue, and quality of life will be measured at baseline (T0) and post-intervention (T1).</div></div><div><h3>Discussion</h3><div>The FIERCE feasibility study aims to explore if a flexible, individualised exercise programme will support individuals to be active during chemotherapy treatment. If proven to be feasible, a large-scale randomised controlled trial will be undertaken focusing on the efficacy of the FIERCE programme on different health outcomes.</div></div><div><h3>Trial Registration</h3><div>The study is registered with <span><span>ClinicalTrials.gov</span><svg><path></path></svg></span>, registration number: <span><span>NCT06280885</span><svg><path></path></svg></span>.</div></div>","PeriodicalId":10636,"journal":{"name":"Contemporary clinical trials","volume":"153 ","pages":"Article 107923"},"PeriodicalIF":2.0,"publicationDate":"2025-04-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143882865","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":"The 4 youth by youth (4YBY) crowdsourced HIV prevention intervention: A stepped-wedge longitudinal trial on HIV self-testing uptake among adolescents and young people in Nigeria","authors":"Juliet Iwelunmor ,&nbsp;Chisom Obiezu-Umeh ,&nbsp;Titilola Gbaja-Biamila ,&nbsp;David Oladele ,&nbsp;Ucheoma Nwaozuru ,&nbsp;Adesola Z. Musa ,&nbsp;Olunike R. Abodunrin ,&nbsp;Folahanmi T. Akinsolu ,&nbsp;Temitope Ojo ,&nbsp;Olufunto Olusanya ,&nbsp;Tajudeen Bamidele ,&nbsp;Nkiru Ezeama ,&nbsp;Chinyere Okeke ,&nbsp;Ifiok Johnny ,&nbsp;Moses Ekene ,&nbsp;Nurudeen Rahman ,&nbsp;Tomilola Musari-Martins ,&nbsp;Sola Ajibaye ,&nbsp;Akeem Lateef ,&nbsp;Victor Ojo ,&nbsp;Oliver Ezechi","doi":"10.1016/j.cct.2025.107919","DOIUrl":"10.1016/j.cct.2025.107919","url":null,"abstract":"<div><div>Adolescents and young adults (AYAs) participatory approaches for HIV control have increased across LMICs, but there are few trials to evaluate effectiveness. We assessed a crowdsourced HIV self-testing (HIVST) intervention among a cohort of AYA in Nigeria.</div></div><div><h3>Methods</h3><div>We conducted a pragmatic stepped-wedge cluster randomized control trial recruiting participants (aged 14–24 years) from 32 local government areas across four geo-political zones in Nigeria. Eligible AYA were HIV negative or unknown HIV status, residing in study sites, spoke English, and consented. Areas were randomly assigned to one of four steps and AYA were followed for 24 months. AYA research facilitators implemented a 4YBY crowdsourced HIV prevention bundle. The primary outcome was self-reported HIVST uptake. We compared the probability of HIVST between the control and intervention periods using a generalized linear mixed model. We examined the fixed cost and per capita cost of the intervention. The protocol was registered with Clinical <span><span>Trials.gov</span><svg><path></path></svg></span> on January 15, 2021, under registration <span><span>NCT04710784</span><svg><path></path></svg></span>.</div></div><div><h3>Results</h3><div>2652 AYA were screened, and 1500 were enrolled in the study (March 10, 2021- August 31, 2023). 1333/1500 (89 %) were followed up at 24 months. The mean age of AYA was 20 ± 2.65 years old, most were students (1155/1500, 77 %), and unemployed (915/1500, 61 %). The intervention led to a 9.96-fold increase in HIV self-testing uptake compared to the control period (95 % CI: 8.36–11.85, <em>p</em> &lt; 0.0001). The annual fixed cost of the intervention was estimated at US$42,237, with a per capita testing cost of US$14.8. No significant adverse events were reported.</div></div><div><h3>Conclusion</h3><div>A crowdsourced HIV prevention intervention increased HIVST uptake among Nigerian AYA. Greater participation of AYA in the design and implementation of clinical trials is needed to achieve UNAIDS targets.</div></div>","PeriodicalId":10636,"journal":{"name":"Contemporary clinical trials","volume":"154 ","pages":"Article 107919"},"PeriodicalIF":2.0,"publicationDate":"2025-04-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143886427","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":"Recruitment and retention in a real-world comparative effectiveness trial to improve diabetes self-care behaviors","authors":"Satya Surbhi ,&nbsp;Mace Coday ,&nbsp;Cardella Leak ,&nbsp;Ming Chen ,&nbsp;James E. Bailey","doi":"10.1016/j.cct.2025.107914","DOIUrl":"10.1016/j.cct.2025.107914","url":null,"abstract":"<div><h3>Background</h3><div>Effective strategies are needed to recruit and retain participants in trials, especially for minority patients. This paper describes recruitment phases of the Management of Diabetes in Everyday Life (MODEL) study, with an emphasis on examining factors that predicted study enrollment and retention.</div></div><div><h3>Methods</h3><div>The MODEL study was a pragmatic clinical trial designed to compare the effectiveness of Text Messaging versus Health Coaching with Educational Materials on improving diabetes self-care among patients of African-American race with uncontrolled diabetes. Descriptive statistics including counts and proportions were used to describe the recruitment process. Multivariable logistic regression was used to examine factors associated with study enrollment and retention.</div></div><div><h3>Results</h3><div>Of 4310 patients contacted, 2300 (53.4 %) were prescreened, of those 1221 (28.3 %) were screened, and 666 (15.4 %) eligible screenees were randomized. Of those who got excluded after signing consent, about 48 % were excluded because they did not respond to text messages and/or voice messages and 26.8 % did not have a recent A1c test. Of those screened, the majority were contacted using a diabetes registry (70.5 %) followed by physician referral (15.7 %). Patients randomized had higher education levels than those who were excluded, and patients in the Health Coaching were significantly less likely to complete the study.</div></div><div><h3>Conclusions</h3><div>The study showed that technology barriers, not having regular follow-up, and being unaware of their chronic conditions, are recruitment barriers for minority populations. Practice participation in a registry can facilitate recruitment of underserved minority populations. Furthermore, patients participating in the time-intensive health coaching intervention experienced lowest study retention.</div></div>","PeriodicalId":10636,"journal":{"name":"Contemporary clinical trials","volume":"154 ","pages":"Article 107914"},"PeriodicalIF":2.0,"publicationDate":"2025-04-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143892114","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":"Protocol for a wait list randomised controlled trial: Using social media for health promotion, communication and advocacy – A massive open online course","authors":"Kahlia McCausland ,&nbsp;Katharina Wolf ,&nbsp;Becky Freeman ,&nbsp;Justine E. Leavy ,&nbsp;Tama Leaver ,&nbsp;HuiJun Chih ,&nbsp;Barbara A. Mullan ,&nbsp;Sonya Girdler ,&nbsp;Gwyneth Peaty ,&nbsp;Mark Chenery ,&nbsp;Jonine Jancey","doi":"10.1016/j.cct.2025.107920","DOIUrl":"10.1016/j.cct.2025.107920","url":null,"abstract":"<div><h3>Introduction</h3><div>Social media can be a powerful tool for raising awareness, engaging and mobilising communities, collecting data, fostering behaviour change and advancing advocacy efforts. However, many public health professionals hesitate to incorporate social media into their work. In responding to this need, we developed a 6-module massive open online course (MOOC) designed to improve knowledge and understanding of how to effectively frame health promotion messages and increase confidence in using social media for health promotion, communication and advocacy. This paper outlines the protocol for a mixed-methods evaluation of this MOOC.</div></div><div><h3>Methods</h3><div>A wait list randomised controlled trial, guided by elements of the RE-AIM and Kirkpatrick models, will collect qualitative and quantitative data from eligible participants at three time points: baseline (T0), interim post-test (T1) and final post-test (T2). The primary outcome measure will be participants' social media context awareness. Secondary outcomes will be social media competency and the impact of the MOOC on professional practice. Additionally, a process evaluation will examine implementation, participant engagement and satisfaction with the MOOC.</div></div><div><h3>Conclusion</h3><div>This research will assess the effectiveness of a MOOC in enhancing health promotion knowledge, framing health messages, and confidence in using social media for health promotion, communication and advocacy among public health professionals. The findings will inform the design and evaluation of future online programs in this field, with results disseminated upon completion of the study.</div><div>Australian New Zealand Clinical Trials Registry (ANZCTR): ACTRN12624001486516.</div></div>","PeriodicalId":10636,"journal":{"name":"Contemporary clinical trials","volume":"153 ","pages":"Article 107920"},"PeriodicalIF":2.0,"publicationDate":"2025-04-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143869389","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":"Using a human-centered design framework and behavioral economic interventions to increase fruit and vegetable purchases in an online grocery store: Study design and methodologies","authors":"Sophia V. Hua ,&nbsp;Tamar Klaiman ,&nbsp;Samantha Coratti ,&nbsp;Jenna White ,&nbsp;Aileen John ,&nbsp;Mary E. Putt ,&nbsp;Erica Dixon ,&nbsp;Hannah Posner ,&nbsp;Kevin G. Volpp","doi":"10.1016/j.cct.2025.107921","DOIUrl":"10.1016/j.cct.2025.107921","url":null,"abstract":"<div><h3>Background</h3><div>Diet-related chronic conditions are leading causes of death in the U.S. Healthier diets with fruits and vegetables can reduce the risk of developing these diseases. This study tests interventions to increase redemption of produce subsidies and purchases of fruits and vegetables using an online grocery delivery platform among people who have obesity and type 2 diabetes.</div></div><div><h3>Design</h3><div>Participants drawn from Penn Medicine primary care practices are given access to online grocery shopping and delivery. The intervention tests ways of increasing subsidy use for fruits and vegetables through a salience manipulation with loss-framed text messages, choice architecture on the grocery platform, or a combination of the two. A 10-person pre-pilot was trialed for two weeks. Learnings from qualitative interviews with the pre-pilot participants were used to refine the design prior to the launch of the 3-month randomized controlled trial (RCT) (projected <em>n</em> = 180).</div></div><div><h3>Primary outcome</h3><div>Total dollar expenditures of eligible fruits and vegetables across the 3 months of the study.</div></div><div><h3>Secondary outcomes</h3><div>1) mean percentage of the monthly subsidy used across 3 months; 2) cumulative percentage of expenditures spent on eligible fruits and vegetables (i.e., total spent on fruits and vegetables/total spent); 3) change in hemoglobin A1c from baseline; and 4) qualitative results including enrollment experience, experience using the study store, communication with the study team, and dietary effects of the study.</div></div><div><h3>Discussion</h3><div>The rationale, design, and protocol of this study are described. This study can provide insights on how food is medicine programs can be structured to improve engagement.</div><div><span><span>ClinicalTrials.gov</span><svg><path></path></svg></span> Registration Number: <span><span>NCT06283394</span><svg><path></path></svg></span></div></div>","PeriodicalId":10636,"journal":{"name":"Contemporary clinical trials","volume":"153 ","pages":"Article 107921"},"PeriodicalIF":2.0,"publicationDate":"2025-04-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143860691","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":"Using Bayesian pre-trial simulations to optimize the design of adaptive clinical trials in childhood nephrotic syndrome","authors":"Cal H. Robinson ,&nbsp;Rulan S. Parekh ,&nbsp;Brian Cuthbertson ,&nbsp;Eddy Fan ,&nbsp;Yongdong Ouyang ,&nbsp;Anna Heath","doi":"10.1016/j.cct.2025.107918","DOIUrl":"10.1016/j.cct.2025.107918","url":null,"abstract":"<div><h3>Background</h3><div>Randomized controlled trials (RCTs) are often infeasible in rare pediatric diseases. Adaptive trials can increase trial efficiency while maintaining scientific validity. Our aim was to determine the optimal design of a Bayesian adaptive RCT in childhood nephrotic syndrome using simulation.</div></div><div><h3>Methods</h3><div>We used simulation to evaluate candidate Bayesian adaptive clinical trial designs for a planned non-inferiority RCT comparing low-dose vs. standard-dose steroids for childhood nephrotic syndrome relapses. Each design had a unique combination of adaptive settings (stopping thresholds, futility margin, initial recruitment, and interim analysis frequency). We simulated 10,000 RCTs for each design to estimate operating characteristics (power, type 1 error rate, and mean sample size). The best designs were tested under plausible RCT conditions (varying treatment effect, recruitment rate, and prior distributions).</div></div><div><h3>Results</h3><div>We simulated 10,000 trials for each of 540 adaptive RCT designs with unique combinations of adaptation settings (5.4 million simulated trials). For the top three designs, we simulated another 10,000 trials under 40 different RCT conditions (1.2 million simulated trials). The optimal trial design was associated with the lowest mean sample size, smallest probability of an inconclusive trial, and a type 1 error rate &lt; 5 %. Compared to a frequentist RCT, using this Bayesian adaptive design with an informative prior decreased sample size by 71 % (<em>n</em> = 198 vs. <em>n</em> = 682).</div></div><div><h3>Conclusions</h3><div>Bayesian trial simulation was used to optimize the design of an adaptive RCT in childhood nephrotic syndrome, lowering estimated sample size. Adaptive designs can reduce barriers to conducting RCTs in rare pediatric diseases.</div></div>","PeriodicalId":10636,"journal":{"name":"Contemporary clinical trials","volume":"153 ","pages":"Article 107918"},"PeriodicalIF":2.0,"publicationDate":"2025-04-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143843627","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":"Produce prescription to improve health among adults with type 2 diabetes in Australia: Protocol for a randomised controlled trial","authors":"Kimberly Glover ,&nbsp;Megan Gow ,&nbsp;Kathy Trieu ,&nbsp;Liping Huang ,&nbsp;Kristy K. Law ,&nbsp;Bruce Neal ,&nbsp;Jencia Wong ,&nbsp;Ted Wu ,&nbsp;Stephen Twigg ,&nbsp;Amanda Gauld ,&nbsp;Maria Constantino ,&nbsp;Margaret McGill ,&nbsp;Stephanie Noonan ,&nbsp;David Simmons ,&nbsp;Ian D. Caterson ,&nbsp;Dariush Mozaffarian ,&nbsp;Claudia Nau ,&nbsp;Jing Li ,&nbsp;Gian Luca Di Tanna ,&nbsp;Vincent Wong ,&nbsp;Jason H.Y. Wu","doi":"10.1016/j.cct.2025.107915","DOIUrl":"10.1016/j.cct.2025.107915","url":null,"abstract":"<div><h3>Background</h3><div>‘Food is medicine’ programs such as Produce Prescription (PRx) aim to integrate food-based nutrition programs into healthcare, for the prevention, management and treatment of diet-related diseases, typically for those experiencing food insecurity. However, the impact of PRx on health indicators in Australia has never been tested in a randomised trial.</div></div><div><h3>Objectives</h3><div>To determine the effect of PRx on blood glucose control and other health indicators in adults with type 2 diabetes experiencing hyperglycaemia and food insecurity and/or financial hardship in Australia.</div></div><div><h3>Methods</h3><div>Using a parallel design randomised controlled trial, <em>n</em> = 224 participants will be randomised (1:1) to PRx or usual care. Over 26 weeks, the intervention group will receive a weekly delivery of fruits, vegetables, wholegrains and nuts, and up to 3 sessions with an accredited dietitian. Controls will receive usual care. The primary outcome is change in mean HbA1c over 26 weeks, comparing the intervention and control group. Secondary outcomes include between-group differences at 26 weeks in change in blood pressure, body weight, blood lipids, food and nutrition insecurity, person-reported outcome measures, medication use, and diet quality. Implementation outcomes assessed will include feasibility, acceptability, scalability and cost effectiveness.</div></div><div><h3>Discussion</h3><div>This Australia-first PRx trial will provide novel and rigorous data for an intervention that may be feasible to improve health and health equity as part of the Australian healthcare system. We anticipate PRx will lead to a clinically meaningful reduction in HbA1c, contribute to improved health equity and long-term health benefits for adults with type 2 diabetes and food insecurity.</div></div>","PeriodicalId":10636,"journal":{"name":"Contemporary clinical trials","volume":"153 ","pages":"Article 107915"},"PeriodicalIF":2.0,"publicationDate":"2025-04-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143848216","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}