Trials最新文献

{"title":"Swab Testing to Optimize Pneumonia treatment with empiric Vancomycin (STOP-Vanc): study protocol for a randomized controlled trial.","authors":"Jeffrey A Freiberg, Justin K Siemann, Edward T Qian, Benjamin J Ereshefsky, Cassandra Hennessy, Joanna L Stollings, Taylor M Rali, Frank E Harrell, Cheryl L Gatto, Todd W Rice, George E Nelson","doi":"10.1186/s13063-024-08705-6","DOIUrl":"10.1186/s13063-024-08705-6","url":null,"abstract":"<p><strong>Background: </strong>Vancomycin, an antibiotic with activity against methicillin-resistant Staphylococcus aureus (MRSA), is frequently included in empiric treatment for community-acquired pneumonia (CAP) despite the fact that MRSA is rarely implicated in CAP. Conducting polymerase chain reaction (PCR) testing on nasal swabs to identify the presence of MRSA colonization has been proposed as an antimicrobial stewardship intervention to reduce the use of vancomycin. Observational studies have shown reductions in vancomycin use after implementation of MRSA colonization testing, and this approach has been adopted by CAP guidelines. However, the ability of this intervention to safely reduce vancomycin use has yet to be tested in a randomized controlled trial.</p><p><strong>Methods: </strong>STOP-Vanc is a pragmatic, prospective, single center, non-blinded randomized trial. The objective of this study is to test whether the use of MRSA PCR testing can safely reduce inappropriate vancomycin use in an intensive care setting. Adult patients with suspicion for CAP who are receiving vancomycin and admitted to the Medical Intensive Care Unit at Vanderbilt University Medical Center will be screened for eligibility. Eligible patients will be enrolled and randomized in a 1:1 ratio to either receive MRSA nasal swab PCR testing in addition to usual care (intervention group), or usual care alone (control group). PCR testing results will be transmitted through the electronic health record to the treating clinicians. Primary providers of intervention group patients with negative swab results will also receive a page providing clinical guidance recommending discontinuation of vancomycin. The primary outcome will be vancomycin-free hours alive, defined as the expected number of hours alive and free of the use of vancomycin within the first 7 days following trial enrollment estimated using a proportional odds ratio model. Secondary outcomes include 30-day all-cause mortality and time alive off vancomycin.</p><p><strong>Discussion: </strong>STOP-Vanc will provide the first randomized controlled trial data regarding the use of MRSA nasal swab PCR testing to guide antibiotic de-escalation. This study will provide important information regarding the effect of MRSA PCR testing and antimicrobial stewardship guidance on clinical outcomes in an intensive care unit setting.</p><p><strong>Trial registration: </strong>ClinicalTrials.gov NCT06272994. Registered on February 22, 2024.</p>","PeriodicalId":23333,"journal":{"name":"Trials","volume":"25 1","pages":"854"},"PeriodicalIF":2.0,"publicationDate":"2024-12-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11682620/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142898389","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":"A randomized trial of low-dose thrombolysis, ultrasound-assisted thrombolysis, or heparin for intermediate-high risk pulmonary embolism-the STRATIFY trial: design and statistical analysis plan.","authors":"Jesper Kjærgaard, Jørn Carlsen, Emilie Sonne-Holm, Sebastian Wiberg, Lene Holmvang, Jens Flensted Lassen, Rikke Sørensen, Dan Høfsten, Peter Sommer Ulriksen, Samir Jawad, Pernille Palm, Jens Jakob Thune, Christian Hassager, Ole P Kristiansen, Kristian Eskesen, Søren Fanø, Lia E Bang","doi":"10.1186/s13063-024-08688-4","DOIUrl":"10.1186/s13063-024-08688-4","url":null,"abstract":"<p><strong>Background: </strong>Intermediate-high risk pulmonary embolism (PE) carries a significant risk of hemodynamic deterioration or death. Treatment should balance efficacy in reducing clot burden with the risk of complications, particularly bleeding. Previous studies on high-dose, short-term thrombolysis with alteplase (rtPA) showed a reduced risk of hemodynamic deterioration but no change in mortality and increased bleeding complications. Catheter-based techniques, including ultrasound-assisted thrombolysis (USAT), and low-dose thrombolysis may offer reasonable efficacy with lower risk. However, studies comparing these methods have been few. This trial aims to address this gap by randomizing patients to three treatment modalities.</p><p><strong>Methods: </strong>Multicenter, randomized trial with 1:1:1 allocation of 210 patients with acute intermediate-high risk PE, excluding those with absolute contraindications to thrombolysis. Patients are eligible for inclusion if they are > 18 years of age, have had symptoms < 14 days, and are able to give informed consent. Patients are allocated 1:1:1 into three treatment strategies: (1) unfractionated heparin (UFH)/low molecular weight heparin (LMWH), (2) UFH/LMWH + 20 mg rtPA/6 h intravenously (IV), or (3) UFH + 20 mg rtPA/6 h via USAT. Co-primary outcomes include reduction in clot burden as assessed by refined Miller score from pre-treatment to follow-up (48-96 h) computed tomography pulmonary angiogram (CTPA) comparing low-dose rtPA (± USAT) groups to UFH/LMWH group (p < 0.01, N = 210) and reduction in refined Miller score on follow-up CT angiography comparing low-dose rtPA by USAT to intravenous rtPA, p < 0.04, N = 140). Secondary outcomes comprise bleeding complications, duration of index admission, FiO₂, blood pressure, respiratory and heart rate at the time of follow-up CT angiography, mortality in the three groups, incidence of tricuspid regurgitation pressure gradient < 40 mmHg at 3 months follow-up echocardiography, 6-min walk test at 3 months comparing the three groups, and health-related quality of life at 3 months follow-up comparing the three groups.</p><p><strong>Discussion: </strong>We hypothesize that in patients with intermediate-high risk PE (1) administration of 20 mg rtPA leads to a greater reduction in clot burden compared to heparins and (2) administration of 20 mg rtPA via USAT results in a greater reduction in clot burden compared to 20 mg rtPA intravenous.</p><p><strong>Trial registration: </strong>ClinicalTrials.gov NCT04088292. Registered in September 2019 (retrospectively registered).</p>","PeriodicalId":23333,"journal":{"name":"Trials","volume":"25 1","pages":"853"},"PeriodicalIF":2.0,"publicationDate":"2024-12-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11682612/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142898198","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":"Enrolment patterns in a randomized controlled trial of probiotics in critically ill patients: a retrospective analysis of the PROSPECT trial.","authors":"Alyson Takaoka, Jennie Johnstone, François Lauzier, Diane Heels-Ansdell, Megan Davis, Nicole Zytaruk, Erick Duan, Joanna Dionne, Lois Saunders, Yaseen M Arabi, John Marshall, Lehana Thabane, France Clarke, Lori Hand, Marie-Helene Masse, Bram Rochwerg, Lauralyn McIntyre, Martin Girard, Andreas Freitag, Tim Karachi, Deborah J Cook","doi":"10.1186/s13063-024-08701-w","DOIUrl":"10.1186/s13063-024-08701-w","url":null,"abstract":"<p><strong>Background: </strong>Understanding site-related factors that influence enrolment within multicenter randomized controlled trials (RCT) may help reduce trial delays and cost over-runs and prevent early trial discontinuation. In this analysis of PROSPECT (Probiotics: Prevention of Severe Pneumonia and Endotracheal Colonization Trial), we describe patient enrolment patterns and examine factors influencing site-based monthly enrolment.</p><p><strong>Design: </strong>Retrospective analysis of a multicenter RCT.</p><p><strong>Methods: </strong>The PROSPECT multicenter RCT enrolled patients in the main trial from July 2015 to March 2019. We documented site characteristics and trial metrics including data from the methods center tracking documents, site-level data at trial initiation, screening logs submitted by research coordinators, and prospectively collected case report forms. In this retrospective analysis of trial data, we analyzed enrolment patterns across sites using negative binomial regression to explore the association between monthly enrolment rate accounting for number of ICU beds, site characteristics, and trial metrics.</p><p><strong>Results: </strong>Overall, 41 sites enrolling 2365 patients in the PROSPECT main trial were analyzed. After accounting for number of beds in each ICU, site launch early in the trial was associated with higher monthly enrolment rates, but time to first enrolment and research coordinator experience was not. We observed considerable variability in the number of active screening months and enrolment rates across sites.</p><p><strong>Conclusion: </strong>These findings highlight the complexity of recruitment dynamics in critical care RCTs and emphasize the need for tailored approaches to trial planning and execution.</p><p><strong>Trial registration: </strong>PROSPECT (Probiotics: Prevention of Severe Pneumonia and Endotracheal Colonization Trial): NCT02462590 (registered June 2, 2015).</p>","PeriodicalId":23333,"journal":{"name":"Trials","volume":"25 1","pages":"851"},"PeriodicalIF":2.0,"publicationDate":"2024-12-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11673600/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142898367","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 individualized versus conventional perioperative blood pressure management on postoperative major complications in high-risk patients undergoing noncardiac surgery: study protocol for the SPROUT-4 multicenter randomized controlled trial.","authors":"Jaeyeon Chung, Chang-Hoon Koo, Jungchan Park, Hye-Bin Kim, Jinyoung Bae, Jae-Woo Ju, Soowon Lee, Ah Ran Oh, Hyo Sung Kim, Soo Jung Park, Yunseok Jeon, Karam Nam","doi":"10.1186/s13063-024-08707-4","DOIUrl":"10.1186/s13063-024-08707-4","url":null,"abstract":"<p><strong>Background: </strong>Intraoperative hypotension is very common during surgery and is linked to major organ dysfunction and mortality. Current perioperative blood pressure management is largely based on universal blood pressure thresholds ranging from a mean arterial pressure of 60-70 mmHg. However, the effectiveness of this conventional management remains unproven in prospective randomized trials. Therefore, we will conduct this study to test if individualized perioperative blood pressure management decreases the incidence of postoperative major adverse outcomes.</p><p><strong>Methods: </strong>This multicenter, randomized controlled superiority trial will enroll 1896 high-risk patients undergoing major noncardiac surgery from five tertiary university hospitals in South Korea. In the control group, mean arterial pressure will be maintained at ≥ 65 mmHg and systolic blood pressure ≥ 90 mmHg during surgery. In the intervention group, mean arterial pressure and systolic blood pressure will be maintained at no less than 20% of their baseline values. The baseline values are calculated as the average of all values measured from the day before surgery until the morning of surgery. These targets will be maintained until the patient is discharged from the post-anesthesia care unit or, for those who are transferred to the intensive care unit after surgery, until the end of the surgery. No specific restrictions, except for these blood pressure targets, will be applied to perioperative management. The primary composite outcome consists of all-cause death, stroke, myocardial infarction, new or worsening congestive heart failure, unplanned coronary revascularization, and acute kidney injury within 7 days after noncardiac surgery or until hospital discharge, whichever occurs first.</p><p><strong>Discussion: </strong>This study will reveal if individualized perioperative blood pressure management decreases the risk of major adverse outcomes in patients at high-risk undergoing noncardiac surgery.</p><p><strong>Trial registration: </strong>ClinicalTrials.gov NCT06225453. Registered on January 26, 2024.</p>","PeriodicalId":23333,"journal":{"name":"Trials","volume":"25 1","pages":"850"},"PeriodicalIF":2.0,"publicationDate":"2024-12-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11673592/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142898212","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":"A multi-center, open-label, randomized clinical trial evaluating the preventive effect of perampanel on craniotomy-induced epileptogenesis in seizure-naive patients with supratentorial brain tumors: study protocol for a GRAMPAS trial.","authors":"Junya Yamaguchi, Fumiharu Ohka, Kazuya Motomura, Tomotaka Ishizaki, Norimoto Nakahara, Shigeru Fujitani, Tetsuya Nagatani, Masasuke Ohno, Masahiko Ando, Yachiyo Kuwatsuka, Kazuki Nishida, Ryuta Saito","doi":"10.1186/s13063-024-08693-7","DOIUrl":"10.1186/s13063-024-08693-7","url":null,"abstract":"<p><strong>Background: </strong>Early seizures after craniotomy are significant perioperative complications that can adversely impact patient outcomes. Despite current guidelines advising against the routine use of antiseizure drugs for seizure after craniotomy prevention due to limited efficacy data, many clinicians continue prescribing them. This discrepancy highlights the need for robust evidence to guide clinical practice. This multi-center, randomized clinical trial was designed to investigate the efficacy of perampanel in preventing early seizures after craniotomy.</p><p><strong>Method: </strong>This multi-center, open-label, randomized clinical trial will be conducted across five hospitals in Nagoya, Japan, from February 2024 to December 2026. A total of 142 seizure-naive patients with supratentorial brain tumors will be recruited and randomized (1:1) into the treatment and control groups. The treatment group will receive 2 mg of perampanel starting 2 days preoperatively and continuing for 28 days postoperatively, while the control group will receive no antiseizure drugs. The primary outcome is the incidence of seizures within 28 days after craniotomy. Secondary outcomes are length of hospital and intensive care unit stays and postoperative complications.</p><p><strong>Discussion: </strong>This study addresses the critical need for evidence-based recommendations regarding antiseizure drug use for preventing early seizures after craniotomy. As the first multi-center, randomized trial evaluating perampanel's efficacy in this setting, the findings may significantly influence clinical guidelines and perioperative practices.</p><p><strong>Trial registration: </strong>This trial was registered with the Japan Registry of Clinical Trials (approval number: jRCTs041230117) on December 18, 2023, a member of the Primary Registry Network of the World Health Organization's International Clinical Trials Registry Platform.</p>","PeriodicalId":23333,"journal":{"name":"Trials","volume":"25 1","pages":"849"},"PeriodicalIF":2.0,"publicationDate":"2024-12-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11667933/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142883040","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":"The effect of Sarcomeal® oral supplementation plus vitamin D3 on muscle parameters and metabolic factors in diabetic sarcopenia patients: study protocol of a randomized controlled clinical trial.","authors":"Ramin Abdi Dezfouli, Narges Zargar Balajam, Sara Shirazi, Ramin Heshmat, Gita Shafiee","doi":"10.1186/s13063-024-08700-x","DOIUrl":"10.1186/s13063-024-08700-x","url":null,"abstract":"<p><strong>Background: </strong>Diabetes is a significant risk factor for sarcopenia, a muscle dystrophy affecting older individuals. Sarcopenia management typically involves resistance exercise and oral supplements. Given the limitations of resistance training for many elderly individuals, oral supplements play a crucial role in treatment. This study is a protocol for evaluating the efficacy of the Sarcomeal® supplement, a mixture of whey protein, creatine, branch-chained amino acids (BCAAs), glutamine, and hydroxyl-methyl-butyrate (HMB) in diabetic people who also have sarcopenia. METHODS AND ANALYSIS: This study is a randomized clinical trial, in which sixty diabetic sarcopenia patients who meet the inclusion criteria will be randomly assigned to the control or the intervention group with a 1:1 allocation. The intervention group will receive one Sarcomeal® supplement sachet plus 1000 IU of vitamin D daily and both groups will be recommended to consume protein-rich food, be educated about the disease, and perform light exercises for 12 weeks. Anthropometric measurements, body composition analysis, muscle strength assessments, and blood tests will be conducted at the trial's start and end.</p><p><strong>Discussion: </strong>It is hypothesized that the Sarcomeal® supplement plus vitamin D may be beneficial for the management of diabetic sarcopenia by reducing inflammation, oxidative stress, and glucose metabolism. The outcome of this trial will provide a basis for prescribing sarcomeal to patients with diabetic sarcopenia.</p><p><strong>Ethics and dissemination: </strong>This protocol is registered at the Iranian Registry of Clinical Trials (IRCT20230831059311N1) and also is approved by the ethics committee of Tehran University of Medical Sciences (September 2023, IR.TUMS.EMRI.REC.1402.071).</p><p><strong>Trial registration: </strong>Iranian Registry of Clinical Trials (ID: IRCT20230831059311N1).</p>","PeriodicalId":23333,"journal":{"name":"Trials","volume":"25 1","pages":"848"},"PeriodicalIF":2.0,"publicationDate":"2024-12-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11667863/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142883058","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":"Application of indocyanine green-human serum albumin complex in fluorescence image-guided laparoscopic anatomical liver resection: study protocol for a randomized controlled trial.","authors":"Qingyun Xie, Fengwei Gao, Xiaoyun Ran, Xin Zhao, Manyu Yang, Kangyi Jiang, Tianyang Mao, Jiayin Yang, Kun Li, Hong Wu","doi":"10.1186/s13063-024-08695-5","DOIUrl":"10.1186/s13063-024-08695-5","url":null,"abstract":"<p><strong>Background: </strong>Indocyanine green (ICG) is a near-infrared fluorescent dye widely used for intraoperative navigation during liver surgeries because of its non-radioactive nature, high safety, and minimal impact on liver function. However, variability in its dosage and concentration and its low imaging success rates have limited its widespread application. To address these issues, we developed a novel ICG-human serum albumin (ICG-HSA) complex to enhance fluorescence visualization during laparoscopic anatomical liver resection.</p><p><strong>Methods: </strong>This prospective, double-blind, single-center, randomized controlled trial will compare the fluorescence navigation effects of the novel ICG-HSA complex with the guideline-recommended ICG administration scheme. The study will involve patients aged 18 to 75 years with malignant liver tumors. The participants will undergo evaluations at specified time points, and data will be collected using an internet-based electronic data capture system. The primary outcome will be the effectiveness of intraoperative fluorescence imaging, assessed by three independent experts. The secondary outcomes will be conversion to open surgery, the total operative time, intraoperative blood loss, and long-term survival rates.</p><p><strong>Discussion: </strong>The aim of using this novel ICG-HSA complex will be to improve the success rate of fluorescence navigation in liver resection by ensuring better stability and a longer liver retention time compared with free ICG. This study seeks to validate the clinical value of ICG-HSA in enhancing surgical precision and outcomes, ultimately promoting its broader clinical application. The results are expected to provide high-level evidence supporting the safety and efficacy of this new fluorescence imaging agent.</p><p><strong>Trial registration: </strong>ClinicalTrial.gov NCT06219096. Registered on 1 December 2024.</p>","PeriodicalId":23333,"journal":{"name":"Trials","volume":"25 1","pages":"847"},"PeriodicalIF":2.0,"publicationDate":"2024-12-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11664937/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142877867","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":"Implementation of a parent training intervention (SPARCK) to prevent childhood mental health problems: study protocol for a pragmatic implementation trial in Norwegian municipalities.","authors":"Anette Arnesen Grønlie, Agathe Backer-Grøndahl, Ragnhild Bang Nes, Maria Begoña Gomez, Truls Tømmerås","doi":"10.1186/s13063-024-08704-7","DOIUrl":"10.1186/s13063-024-08704-7","url":null,"abstract":"<p><strong>Background: </strong>Effective evidence-based interventions (EBI) are necessary to prevent and avoid negative life trajectories for children with mental health problems. Even though many EBIs prove effective when tested, few are successfully implemented and used in real-world clinical practice. As a result, many children and families do not receive the best care in due time or at all. To reduce this research-practice gap, a combined RCT and implementation study of Supportive Parents-Coping Kids (SPARCK), a parent training intervention to prevent childhood mental health problems, will be performed. This study protocol concerns the implementation part of the larger effectiveness-implementation project.</p><p><strong>Methods: </strong>The study is a correlational multi-site implementation study of SPARCK performed alongside a two-armed RCT, in 24 Norwegian municipalities. A quantitative three-wave longitudinal web-based data collection will be conducted among SPARCK practitioners and leaders in relevant services. We will investigate the relations between theory-driven and empirical implementation determinants and implementation outcomes, measured by fidelity, acceptability, appropriateness, and feasibility. In addition, we will examine how these implementation determinants and outcomes are associated with the clinical outcomes of SPARCK.</p><p><strong>Discussion: </strong>The current study will investigate implementation determinants and their relation to indicators of implementation success, while simultaneously investigating effectiveness of an intervention optimized to the needs of both the target group and relevant stakeholders. Together, this may improve clinical effect, contextual fit, implementation success, and reduce the time lag between research findings and application in real-world settings.</p><p><strong>Trial registration: </strong>ClinicalTrials.gov NCT05800522. Registered on 2023.03.23.</p>","PeriodicalId":23333,"journal":{"name":"Trials","volume":"25 1","pages":"846"},"PeriodicalIF":2.0,"publicationDate":"2024-12-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11663316/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142872867","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 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}