Trials最新文献

{"title":"Factors associated with male recruitment in a multi-site randomized behavioral clinical trial targeting the metabolic syndrome: analysis of screening and recruitment data from the ELM trial.","authors":"Chen Yeh, Melissa M Crane, Bryce Daniels, Barbara Lohse, Kelly Karavolos, Tami Olinger, Jacinda Nicklas, Lynda H Powell, Sumihiro Suzuki","doi":"10.1186/s13063-024-08703-8","DOIUrl":"10.1186/s13063-024-08703-8","url":null,"abstract":"<p><strong>Background: </strong>Males are underrepresented in behavioral clinical trials of lifestyle. The aim of this exploratory study was to investigate factors associated with trial interest in males at different stages of recruitment and overall, into a multi-site behavioral trial targeting lifestyle change and remission of the metabolic syndrome. Similar analyses were performed for female participation to investigate the uniqueness or consistency with the findings for males.</p><p><strong>Methods: </strong>Data collected at various stages of recruitment in an ongoing multi-site behavioral clinical trial were used. A series of logistic regressions compared respondents who moved forward to the next step of the screening process versus those who did not. These analyses were stratified by sex. A chi-squared test was used to directly compare proportions of men and women who chose to advance to the next step.</p><p><strong>Results: </strong>Males who showed interest in the trial were more likely to be self-aware of their current health risk. Comparison of males and females showed that men tended to lose interest earlier in the recruitment process (58.3% of men vs. 66.5% of women attended an in-person information session, p < 0.001), but the proportion that moved forward among those who demonstrated initial interest was similar in men and women.</p><p><strong>Conclusion: </strong>Efforts to increase male enrollment in behavioral clinical trials will benefit from a focus on early stages of recruitment, aiming to increase potential participants' initial levels of interest and awareness of their health risk. As men and women differ in the reasons they choose to participate in a behavioral trial, recruitment should be tailored to sex to maximize trial participation.</p><p><strong>Trial registration: </strong>ClinicalTrials.gov NCT04036006. Registered on July 29, 2019. https://clinicaltrials.gov/study/NCT04036006.</p>","PeriodicalId":23333,"journal":{"name":"Trials","volume":"25 1","pages":"844"},"PeriodicalIF":2.0,"publicationDate":"2024-12-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11662765/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142872862","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"International workshop: what is needed to ensure outcome measures for Rett syndrome are fit-for-purpose for clinical trials? June 7, 2023, Nashville, USA.","authors":"Jenny Downs, Dominique C Pichard, Walter E Kaufmann, Joseph P Horrigan, Melissa Raspa, Gillian Townend, Eric D Marsh, Helen Leonard, Kathleen Motil, Andrew C Dietz, Nupur Garg, Amitha Ananth, Breanne Byiers, Sarika Peters, Christopher Beatty, Frank Symons, Aleksandra Jacobs, James Youakim, Bernhard Suter, Paramola Santosh, Jeffrey L Neul, Tim A Benke","doi":"10.1186/s13063-024-08678-6","DOIUrl":"10.1186/s13063-024-08678-6","url":null,"abstract":"<p><strong>Introduction: </strong>The clinical, research and advocacy communities for Rett syndrome are striving to achieve clinical trial readiness, including having fit-for-purpose clinical outcome assessments. This study aimed to (1) describe psychometric properties of clinical outcome assessment for Rett syndrome and (2) identify what is needed to ensure that fit-for-purpose clinical outcome assessments are available for clinical trials.</p><p><strong>Methods: </strong>Clinical outcome assessments for the top 10 priority domains identified in the Voice of the Patient Report for Rett syndrome were compiled and available psychometric data were extracted. The clinical outcome assessments measured clinical severity, functional abilities, comorbidities and quality of life, and electrophysiological biomarkers. An international and multidisciplinary panel of 29 experts with clinical, research, psychometric, biostatistical, industry and lived experience was identified through International Rett Syndrome Foundation networks, to discuss validation of the clinical outcome assessments, gaps and next steps, during a workshop and in a follow-up questionnaire. The identified gaps and limitations were coded using inductive content analysis.</p><p><strong>Results: </strong>Variable validation profiles across 26 clinical outcome assessments of clinical severity, functional abilities, and comorbidities were discussed. Reliability, validity, and responsiveness profiles were mostly incomplete; there were limited content validation data, particularly parent-informed relevance, comprehensiveness and comprehensibility of items; and no data on meaningful change or cross-cultural validity. The panel identified needs for standardised administration protocols and systematic validation programmes.</p><p><strong>Conclusion: </strong>A pipeline of collaborative clinical outcome assessment development and validation research in Rett syndrome can now be designed, aiming to have fit-for-purpose measures that can evaluate meaningful change, to serve future clinical trials and clinical practice.</p>","PeriodicalId":23333,"journal":{"name":"Trials","volume":"25 1","pages":"845"},"PeriodicalIF":2.0,"publicationDate":"2024-12-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11663341/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142872903","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Time to look beyond (and before) journal reporting requirements: a review of ethnicity reporting in UK clinical trial results publications in three high-impact journals.","authors":"Mahwar Khanum, Shoba Dawson, Jhulia Dos Santos, Huda Hajinur, Carmel Conefrey, Sangeetha Paramasivan","doi":"10.1186/s13063-024-08596-7","DOIUrl":"10.1186/s13063-024-08596-7","url":null,"abstract":"","PeriodicalId":23333,"journal":{"name":"Trials","volume":"25 1","pages":"843"},"PeriodicalIF":2.0,"publicationDate":"2024-12-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11660696/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142872914","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Irregular assessment times in pragmatic randomized clinical trials.","authors":"Andrea Apter, Frances K Barg, Sanjib Basu, Alex Federman, Winifred J Hamilton, Jerry A Krishnan, Tianjing Li, Russell Localio, Christine Pindle, Daniel O Scharfstein, Justin D Smith, Kaharu Sumino","doi":"10.1186/s13063-024-08676-8","DOIUrl":"10.1186/s13063-024-08676-8","url":null,"abstract":"<p><p>Deviation from protocolized assessment times is commonplace in pragmatic randomized clinical trials. Working with a stakeholder advisory board for a Patient-Centered Outcomes Research Institute®-funded project on statistical methods for handling potential biases introduced by irregular assessment times, we identified reasons for off-schedule or missed assessments. We used the Consolidated Framework for Implementation Research 2.0 to organize our findings. We conjectured that timely completion of outcome assessments is a function of multiple determinants, only some related to participants' health status. We identified potential determinants that can be modified during the protocol design stage and can be reassessed and mitigated during trial implementation stage. Research to more formally evaluate our findings is warranted as well as studies to evaluate multi-level strategies that reduce off-schedule or missed assessments.</p>","PeriodicalId":23333,"journal":{"name":"Trials","volume":"25 1","pages":"841"},"PeriodicalIF":2.0,"publicationDate":"2024-12-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11660989/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142865593","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Perioperative methadone compared to placebo in elderly hip fracture patients: a study protocol for a randomized controlled trial (MetaHip trial).","authors":"Kevin Heebøll Nygaard, Thomas Strøm, Kirsten Specht, Sofie Ronja Petersen, Jesper Ougaard Schønnemann","doi":"10.1186/s13063-024-08694-6","DOIUrl":"10.1186/s13063-024-08694-6","url":null,"abstract":"<p><strong>Background: </strong>Hip fractures are a source of severe pain among the elderly population and pose challenges due to limited analgesic tolerance. Perioperative methadone has shown promise in our pilot study suggesting a safe dose of 0.10 mg/kg, prompting further investigation into its benefits for elderly hip fracture patients.</p><p><strong>Methods: </strong>This study employs a double-blinded randomized controlled trial to assess the analgesic effects of a single dose of methadone during hip fracture surgery. Patients aged ≥ 60 years are consecutively enrolled and randomized to receive either perioperative methadone (treatment group) or a saline solution (placebo group). A sample size of 130 patients is required for 88% statistical power. The medication is administered intravenously at anesthesia induction and monitored until discharge. A follow-up observation is conducted 3 months post-surgery.</p><p><strong>Discussion: </strong>Primary outcome: Daily consumption of opioids within the first 3 days after surgery. Secondary outcomes include pain, mobility, nausea, vomiting, time to discharge, need for antidote, delirium, and constipation. The 3-month follow-up includes opioid use, pain, EQ-5D-5L scores, mobility, and persistent side effects. If statistically significant advantages are found in the treatment group, perioperative methadone could be considered as standard care for hip fracture patients, potentially enhancing their pain management. The study's outcomes will provide insights into the feasibility and effectiveness of incorporating methadone into routine clinical practices for this patient group.</p><p><strong>Trial registration: </strong>ClinicalTrials.gov ID: NCT06086171, submitted 4. October 2023.</p><p><strong>Eu-ct: </strong>2023-506252-24-00, UTN: U1111-1294-6125.</p>","PeriodicalId":23333,"journal":{"name":"Trials","volume":"25 1","pages":"842"},"PeriodicalIF":2.0,"publicationDate":"2024-12-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11660571/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142872911","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Process-based therapy vs. routine-CBT for difficult-to-treat mood and anxiety disorders: study protocol for a randomized controlled trial.","authors":"Ulrich Stangier, Viktoria Kohl, Nora Görg, Lucie Sendig, Bettina Hufschmidt, Desiree Bonarius, Arwin Nemani, Mareike Ebert, Stefan G Hofmann","doi":"10.1186/s13063-024-08689-3","DOIUrl":"10.1186/s13063-024-08689-3","url":null,"abstract":"<p><strong>Background: </strong>Process-based therapy (PBT) is a new framework to intervention planning, based on the use of ecological momentary assessment (EMA) data and dynamic and idiographic network analyses. Support for its applicability has been reported from a single-case studies. Here, we examine the feasibility and effectiveness of PBT in a larger clinical sample. We have translated a training manual of PBT and modified for delivery of CBT in mental health service. The aim of this study is to test the relative efficacy of PBT compared to traditional CBT delivered in routine practice (r-CBT) for difficult-to-treat mood and anxiety disorders.</p><p><strong>Methods: </strong>The study is a randomized controlled trial (RCT) of PBT vs r-CBT for difficult-to-treat unipolar depression and anxiety disorders. In total, 80 patients are recruited at an outpatient clinic and included in two intervention arms. Primary outcome is emotional distress; secondary outcomes include psychological well-being and quality of life, adaptive behavior, psychological flexibility, and reflective functioning. Assessments of outcome variables are conducted before and after therapy and at 6 months follow-up. Weekly patient-rated outcomes are collected for every session to investigate process of change. Outcome assessors, blind to treatment allocation, will perform the observer-based symptom ratings, and adherence with manual will be monitored using self-report.</p><p><strong>Discussion: </strong>The current study will be the first RCT of PBT in a health care setting. The planned moderator and mediator analyses will clarify the mechanisms of change in psychotherapy and the association between personalized assessment based on dynamic network analysis and treatment effect.</p><p><strong>Trial registration: </strong>ClinicalTrials.gov NCT06517589. Registered 24 July 2024.</p>","PeriodicalId":23333,"journal":{"name":"Trials","volume":"25 1","pages":"838"},"PeriodicalIF":2.0,"publicationDate":"2024-12-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11657819/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142865594","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Effect of transcutaneous electro-stimulation in postoperative rehabilitation pain treatment in thoracic surgery: a randomized clinical trial.","authors":"Daniel David Álamo-Arce, Daniel López-Fernández, Raquel Medina-Ramírez, Martín Vílchez-Barrera, Pilar Etopa-Bitata, Maria Del Pino Quintana-Montesdeoca, Aníbal Báez-Suárez, Jorge L Freixinet","doi":"10.1186/s13063-024-08613-9","DOIUrl":"10.1186/s13063-024-08613-9","url":null,"abstract":"<p><strong>Background: </strong>Chest pain is one of the most difficult problems to solve after thoracic surgery. Its correct control is often quite difficult, which can cause complications due to an ineffective cough and superficial respiratory movements.</p><p><strong>Methods: </strong>This study has been designed with the purpose of studying the value of transcutaneous electrical stimulation (TENS) in the postoperative pain rehabilitation of thoracotomy. A prospective and randomized study has been developed. The patients (n = 109) have been treated after hospital discharge with physiotherapy for 3 weeks. Three groups have been established: experimental (n = 37), control (n = 35), and placebo (n = 37), experimental and placebo including the application of TENS during the physiotherapy protocol. Postoperative pain (McGill test) and spirometry have been studied before and after treatment.</p><p><strong>Results: </strong>The largest between-group discrepancy occurred between the experimental and control groups, 16.77 points (p < 0.001). Spirometry has shown an improvement in FVC (27.11%) and FEV1 (28.68%) (p < 0.001) in the experimental group, which was statistically significant compared to the other groups.</p><p><strong>Conclusion: </strong>The use of TENS, as an adjunctive treatment to physiotherapy, leads to an improvement in pain control and spirometry values in patients after thoracic surgery, without producing side effects with the technique. These findings provide physiological evidence for the use of TENS in post-pulmonary surgery and may form the basis for the development of pain managed-based programs in clinics and hospitals.</p><p><strong>Trial registration: </strong>NCT04964973 (ClinicalTrials.gov). First registration: July 16, 2021.</p><p><strong>Protocol: </strong>https://clinicaltrials.gov/study/NCT04964973 .</p>","PeriodicalId":23333,"journal":{"name":"Trials","volume":"25 1","pages":"839"},"PeriodicalIF":2.0,"publicationDate":"2024-12-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11660560/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142865592","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Factors that influence recruitment to COVID-19 vaccine trials: a qualitative evidence synthesis.","authors":"Linda Biesty, Sarah Sheehan, Pauline Meskell, Maura Dowling, Claire Glenton, Sasha Shepperd, Xin Hui S Chan, Rebecca Cox, Declan Devane, Andrew Booth, Catherine Houghton","doi":"10.1186/s13063-024-08670-0","DOIUrl":"10.1186/s13063-024-08670-0","url":null,"abstract":"<p><strong>Background: </strong>The COVID-19 pandemic marked a unique period characterised by an extraordinary global virus spread. The collective effort to halt the transmission of the virus led to various public health initiatives, including a variety of COVID-19 vaccine trials. Many of these trials used adaptive methods to address the pandemic's challenges, such as the need for rapid recruitment. These adaptive methods allow for modifications to the trial procedures without undermining the trial's integrity, making the research process more flexible and efficient. However, recruiting participants for vaccine trials remains a considerable challenge. The aim of this qualitative evidence synthesis (QES) is to explore the factors that influence a person's decision to participate in a COVID-19 vaccine trial. Lessons learned from this could help shape future trials' design and conduct, particularly those conducted within a pandemic.</p><p><strong>Methods: </strong>We conducted a systematic search for qualitative studies and mixed methods studies with a qualitative component in the WHO COVID-19 Research Database, MEDLINE, CINAHL, PsycINFO, Epistemomikos, Online Resource for Research in Clinical Trials (ORCCA), and the Cochrane COVID-19 Study Register. We used the best-fit framework synthesis approach and the Social Ecological Model as an a priori framework. We used the GRADE-CERQual approach to assess our confidence in the review findings.</p><p><strong>Results: </strong>Five studies involving 539 participants were included. One of these studies included participants in a COVID-19 vaccine trial. In three of the studies, participants were asked hypothetically about their attitudes. Another study included people who had either not responded to or declined an invitation to participate in a COVID-19 vaccine trial. We developed six themes outlining the factors that influence a person's decision to participate in a COVID-19 vaccine trial: (1) personal gains, (2) perceived risk, (3) influence of family and community, (4) contributing for others, (5) institutional trust and mistrust, and (6) accessibility of the trial.</p><p><strong>Conclusion: </strong>This review sheds light on how people perceive the potential personal, family, and community advantages of trial participation and how these perceptions may be weighed against concerns about vaccine safety. The findings also point toward specific aspects of trial methodology to consider when designing COVID-19 vaccine trials.</p>","PeriodicalId":23333,"journal":{"name":"Trials","volume":"25 1","pages":"837"},"PeriodicalIF":2.0,"publicationDate":"2024-12-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11657826/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142855435","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Rationale and design of a large trial of perioperative ketamine for prevention of chronic post-surgical pain.","authors":"Philip J Peyton, Sabine Braat, Anurika De Silva, David Story, Lisbeth Evered, Paul S Myles, Matthew Chan, Stephan Schug, Malcolm Hogg, Alex Holmes, Sofia Sidiropoulos, Kate Leslie","doi":"10.1186/s13063-024-08672-y","DOIUrl":"10.1186/s13063-024-08672-y","url":null,"abstract":"<p><strong>Background: </strong>Chronic post-surgical pain (CPSP) is recognised as one of the most common and debilitating complications of major surgery. Progression from acute to chronic pain after surgery involves sensitisation of central nervous system pathways with the N-methyl-D-aspartate (NMDA) receptor having a central role. Ketamine is a potent, non-selective NMDA antagonist commonly used for management of acute postoperative pain. Inconsistent but largely supportive evidence from small trials of a preventative effect of perioperative ketamine on CPSP risk suggests that a confirmative large trial is needed.</p><p><strong>Methods: </strong>The ROCKet (Reduction Of Chronic Post-surgical Pain with Ketamine) Trial is a multicentre, double-blind, placebo-controlled, individually randomised superiority trial conducted in 36 hospitals across Australia, New Zealand, and Hong Kong. The trial aims to recruit 4884 patients undergoing abdominal, thoracic, or major orthopaedic surgery. Eligible participants are randomised equally to perioperative intravenous ketamine or placebo for up to 72 h. Incidence of pain in the area of the index surgery is measured by structured telephone interview at 3 months (primary trial endpoint) and 12 months. Pain severity, nature, and associated psychological and quality of life indices are measured using the modified Brief Pain Inventory short form, Neuropathic Pain Questionnaire, Kessler K-10 Psychological Distress Scale, Pain Catastrophising Scale, EQ-5D-3L, and measures of healthcare utilisation and costs. The trial is being conducted by the Department of Critical Care, University of Melbourne, and the Australian and New Zealand College of Anaesthetists Clinical Trials Network. The trial is funded by the Australian National Health and Medical Research Council.</p><p><strong>Discussion: </strong>The ROCKet trial will clarify the effectiveness of ketamine in primary prevention of CPSP. In addition, it will provide high-quality, prospective data on the epidemiology of CPSP which will better inform further research into prevention and management of CPSP.</p><p><strong>Trial registration: </strong>Australian New Zealand Clinical Trials Registry (ACTRN12617001619336) on the date of 12/11/2017.</p>","PeriodicalId":23333,"journal":{"name":"Trials","volume":"25 1","pages":"840"},"PeriodicalIF":2.0,"publicationDate":"2024-12-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11660817/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142865600","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Re-analysis of data from cluster randomised trials to explore the impact of model choice on estimates of odds ratios: study protocol.","authors":"Karla Hemming, Jacqueline Y Thompson, Monica Taljaard, Samuel I Watson, Jessica Kasza, Jennifer A Thompson, Brennan C Kahan, Andrew J Copas","doi":"10.1186/s13063-024-08653-1","DOIUrl":"10.1186/s13063-024-08653-1","url":null,"abstract":"<p><strong>Background: </strong>There are numerous approaches available to analyse data from cluster randomised trials. These include cluster-level summary methods and individual-level methods accounting for clustering, such as generalised estimating equations and generalised linear mixed models. There has been much methodological work showing that estimates of treatment effects can vary depending on the choice of approach, particularly when estimating odds ratios, essentially because the different approaches target different estimands.</p><p><strong>Methods: </strong>In this manuscript, we describe the protocol for a planned re-analysis of data from a large number of cluster randomised trials. Our main objective is to examine empirically whether and how odds ratios estimated using different approaches (for both primary and secondary binary outcomes) vary in cluster randomised trials. We describe the methods that will be used to identify the datasets for inclusion and how they will be analysed and reported.</p><p><strong>Discussion: </strong>There have been a number of small comparisons of empirical differences between the different approaches to analysis for CRTs. The systematic approach outlined in this protocol will allow a much deeper understanding of when there are important choices around the model approach and in which settings. This will be of importance given the heightened awareness of the importance of estimands and the specification of statistical analysis plans.</p>","PeriodicalId":23333,"journal":{"name":"Trials","volume":"25 1","pages":"818"},"PeriodicalIF":2.0,"publicationDate":"2024-12-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11653799/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142855495","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}