Contemporary Clinical Trials Communications最新文献

{"title":"Transmural collaborative care model for cardiovascular risk management and medication review in patients using antipsychotics in primary care (TACTIC): A study protocol of an incomplete stepped wedge cluster randomized trial","authors":"Kirsti M. Jakobs ,&nbsp;Karlijn J. van den Brule-Barnhoorn ,&nbsp;Jan van Lieshout ,&nbsp;Joost G.E. Janzing ,&nbsp;Wiepke Cahn ,&nbsp;Wietske Kievit ,&nbsp;Steven Teerenstra ,&nbsp;Maria van den Muijsenbergh ,&nbsp;Marion C.J. Biermans ,&nbsp;Erik W.M.A. Bischoff","doi":"10.1016/j.conctc.2024.101418","DOIUrl":"10.1016/j.conctc.2024.101418","url":null,"abstract":"<div><h3>Background</h3><div>It is well established that patients with severe mental illness and those treated with atypical antipsychotics (AAPs) are at an increased risk of cardiovascular disease. However, primary care currently lacks adequate monitoring of AAP usage, its effects, and the associated cardiovascular risk. We have developed TACTIC, a transmural collaborative care model for patients using AAPs prescribed by the general practitioner (GP) to address the issues of potential overtreatment with AAPs and undertreatment for cardiovascular risk. TACTIC comprises three steps: an informative video for patients, a multidisciplinary meeting, and a shared decision-making consultation with the GP.</div></div><div><h3>Objectives</h3><div>To evaluate TACTIC's effectiveness on cardiovascular risk and mental health and its cost-effectiveness.</div></div><div><h3>Methods</h3><div>We will conduct an incomplete stepped wedge cluster randomized trial in the Netherlands.</div><div>40 GP-nurse clusters are randomized into four waves. Each cluster recruits adult patients (25–85 years), without prior diagnoses of dementia, delirium, or cardiovascular disease, for whom the GP prescribes AAPs. Every five months, a new wave starts with TACTIC. Measurements are taken before the intervention starts and every 5 months until the study concludes. Primary outcomes are cardiovascular risk and mental health as measured with the QRISK3 score and MHI5, respectively. The economic evaluation consists of two cost-utility analyses, one on the data collected alongside the trial and one based on a model extrapolating the trial data to a 10-year horizon. We will also evaluate the process of delivering TACTIC.</div></div><div><h3>Conclusion</h3><div>This study will assess TACTIC's (cost)effectiveness and provide insights for successful delivery in general practice.</div></div><div><h3>Clinical trials registration</h3><div>clinicaltrials.gov NCT05647980.</div></div>","PeriodicalId":37937,"journal":{"name":"Contemporary Clinical Trials Communications","volume":"44 ","pages":"Article 101418"},"PeriodicalIF":1.4,"publicationDate":"2025-01-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11787019/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143080768","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Strategies for recruitment and retention of diverse and underserved cancer survivor and caregiver dyads in clinical trials","authors":"Mary Hadeed ,&nbsp;Terry A. Badger ,&nbsp;Chris Segrin ,&nbsp;Rogelio Robles-Morales ,&nbsp;Samantha J. Werts-Pelter","doi":"10.1016/j.conctc.2024.101425","DOIUrl":"10.1016/j.conctc.2024.101425","url":null,"abstract":"<div><h3>Background</h3><div>Cancer survivor-caregiver dyads from underrepresented racial and ethnic groups and those with lower socioeconomic status are less likely to participate in clinical research. Sociocultural and socioeconomic barriers perpetuate health inequity and increase disparities in cancer care.</div></div><div><h3>Purpose</h3><div>We describe our systematic approach to recruiting and retaining diverse survivor-caregiver dyads in supportive cancer care studies.</div></div><div><h3>Methods</h3><div>Matsuda's research recruitment guidelines of evaluate, engage, reflect, and carefully match (“EERC”) were adapted and applied through a framework of six guiding principles.</div></div><div><h3>Results</h3><div>A systematic approach to recruitment of underrepresented dyads in cancer support research includes 1) Developing a bilingual, bicultural study team with shared language and culture of the study population, 2) Ensuring team members share a passion for cancer health equity and are trained with a community-centric approach, 3) Designing accessible interventions, study materials, and shared data collection tools across similar studies with community and stakeholder input, 4) Engaging local and regional stakeholders with expertise of health disparities among the catchment area, 5) Partnering with Community Health Workers (CHWs) and gatekeepers to enhance community presence, and 6) Ensuring careful application of matching study team members and participants beyond race and ethnicity to prioritize the cultural values and social factors that impact cancer survivors and caregivers.</div></div><div><h3>Conclusion</h3><div>Applying a systematic approach to recruiting and retaining underrepresented dyads in cancer research can potentially reduce sociocultural and socioeconomic barriers to cancer health equity.</div></div>","PeriodicalId":37937,"journal":{"name":"Contemporary Clinical Trials Communications","volume":"44 ","pages":"Article 101425"},"PeriodicalIF":1.4,"publicationDate":"2025-01-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11773091/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143068433","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Comparative main effects, mediators, and moderators of cognitive behavioral therapy, acceptance and commitment therapy, and emotional awareness and expression therapy for chronic spinal pain: Randomized controlled trial rationale and protocol","authors":"John W. Burns ,&nbsp;Mark A. Lumley ,&nbsp;Kevin E. Vowles ,&nbsp;Mark P. Jensen ,&nbsp;Melissa A. Day ,&nbsp;Howard Schubiner ,&nbsp;Emma Jaszczak ,&nbsp;Britney Abro ,&nbsp;Sarah H. Addicks ,&nbsp;Michael J. Bordieri ,&nbsp;Michael M. Dow ,&nbsp;Shoshana Krohner ,&nbsp;Zyanya Mendoza ,&nbsp;Eric C. Meyer ,&nbsp;Danielle Z. Miro ,&nbsp;Hallie Tankha ,&nbsp;David S. Tubman ,&nbsp;Jolin B. Yamin ,&nbsp;Dokyoung S. You","doi":"10.1016/j.conctc.2025.101428","DOIUrl":"10.1016/j.conctc.2025.101428","url":null,"abstract":"<div><h3>Background</h3><div>Chronic spinal (back/neck) pain is common and costly. Psychosocial treatments are available but have modest effects. Knowledge of treatment mechanisms (mediators and moderators) can be used to enhance efficacy. Trials that directly compare different treatments are needed to determine which mechanisms are treatment-specific, which are shared across treatments, and which contribute the most to outcomes.</div></div><div><h3>Methods</h3><div>We will conduct a 4-arm randomized, controlled clinical trial to compare the main effects, mediators, and moderators of three pain therapies: Cognitive-Behavioral Therapy, Acceptance and Commitment Therapy, and Emotional Awareness and Expression Therapy in adults with chronic spinal pain. Following baseline assessment of outcomes variables (two primary outcomes: pain intensity and pain interference) and potential mediators and moderators, we will randomize participants (up to 460) to one of the treatments or usual care control. Treatments will be conducted individually each week for 8 weeks via telehealth. We will conduct weekly assessments of both potential mediators and outcomes, as well as post-treatment and 6-month follow-up assessments. We will test whether any of the therapies is superior to the others (Aim 1); identify mediators that are specific to treatments and those that are shared across treatments (Aim 2); and identify baseline moderators that are specific to treatments or shared across treatments, and moderated mediators of treatments (Aim 3).</div></div><div><h3>Discussion</h3><div>The findings from this project can be used to improve the effects of psychosocial chronic pain treatments by identifying the most powerful specific and shared mechanisms and revealing for whom the mediator-outcome pathways are strongest.</div></div>","PeriodicalId":37937,"journal":{"name":"Contemporary Clinical Trials Communications","volume":"44 ","pages":"Article 101428"},"PeriodicalIF":1.4,"publicationDate":"2025-01-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11773268/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143068272","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Effect of foot reflexology on pain, anxiety, and physiological indices in leukemia patients undergoing bone marrow aspiration: A randomized clinical trial study","authors":"Marjan Mehri ,&nbsp;Mohamad Golitaleb ,&nbsp;Ali Safdari ,&nbsp;Sabah Madadi ,&nbsp;Fatemeh Rafiei ,&nbsp;Hossein Shahbazi ,&nbsp;Razieh Mokhtari ,&nbsp;Nazi Nejat","doi":"10.1016/j.conctc.2025.101427","DOIUrl":"10.1016/j.conctc.2025.101427","url":null,"abstract":"<div><h3>Introduction</h3><div>Patients with leukemia undergoing bone marrow aspiration (BMA) frequently experience considerable pain and anxiety, which may negatively impact physiological outcomes. Reflexology, a complementary therapeutic approach, has demonstrated effectiveness in reducing pain and anxiety across various medical procedures. This randomized clinical trial aimed to evaluate the impact of foot reflexology on pain, anxiety, and physiological indices in leukemia patients undergoing BMA.</div></div><div><h3>Methods</h3><div>This randomized clinical trial was conducted at Ayatollah Khansari Hospital in Arak between August 2021 and January 2023. Patients were randomly assigned to either a reflexology intervention group or a control group receiving routine care. The intervention group underwent a 30-min foot reflexology session. Pain was assessed using the Numeric Rating Scale (NRS), and anxiety levels were measured via the Spielberger State-Trait Anxiety Inventory (STAI). Physiological indices, including systolic and diastolic blood pressure, heart rate, and arterial oxygen saturation, were measured before and after the intervention.</div></div><div><h3>Results</h3><div>Of the 72 patients, 68 completed the study (intervention: n = 35, control: n = 33). Post-intervention, the intervention group showed a significant reduction in systolic (p = 0.017) and diastolic blood pressure (p = 0.028), while the control group experienced no significant changes. Heart rate significantly increased in the control group (p = 0.030), but no significant change was observed in the intervention group (p = 0.977). Anxiety levels significantly decreased in the intervention group (p &lt; 0.001), with a notable between-group difference (p = 0.006). Pain intensity was also significantly lower in the intervention group compared to the control group (p = 0.034).</div></div><div><h3>Conclusion</h3><div>Foot reflexology is an effective intervention for reducing pain and anxiety in leukemia patients undergoing BMA. Reflexology may be considered a valuable, non-invasive supportive therapy for managing procedural pain and anxiety in oncology settings.</div></div>","PeriodicalId":37937,"journal":{"name":"Contemporary Clinical Trials Communications","volume":"44 ","pages":"Article 101427"},"PeriodicalIF":1.4,"publicationDate":"2025-01-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11773030/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143068412","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Utilization of a structured research site mentorship model to facilitate site performance in a clinical research network","authors":"Marcus R. Johnson ,&nbsp;Danielle Beck ,&nbsp;Melyssa Sueiro ,&nbsp;Makaila Decker ,&nbsp;Jeff Newcomb ,&nbsp;Margaret Tiktin ,&nbsp;Amelia Kiliveros ,&nbsp;Aliya Asghar","doi":"10.1016/j.conctc.2024.101423","DOIUrl":"10.1016/j.conctc.2024.101423","url":null,"abstract":"<div><h3>Background</h3><div>Research site mentorship has a positive impact on study enrollment. The VA Cooperative Studies Program's (CSP) Network of Dedicated Enrollment Sites (NODES) utilized an existing site mentorship model to onboard 13 new expansion sites. We describe the successes, challenges, and lessons learned during the development and implementation of this model in this paper.</div></div><div><h3>Methods</h3><div>NODES established a “Site Mentorship/Expansion Workgroup (SWG)” in October 2020 to plan and guide the consortium on providing mentorship and services to other clinical research networks, non-Node CSP study sites, and NODES expansion sites. In 2021, the SWG developed a 12-month implementation plan to onboard 13 new sites by pairing original Node (mentor) sites with expansion Node (mentee) sites. Mentors offered prompt guidance and solutions to mentees on site-level challenges by working with them closely. Implementation of the plan occurred from February 2022 through September 2023.</div></div><div><h3>Results</h3><div>Data from the implementation of this mentorship plan demonstrated a 32.7 % increase (from 54.8 % in 2022 to 87.5 % in 2023) in the expansion sites’ achievement of their Objectives &amp; Key Results (OKRs). From October 2020–September 2021, prior to mentorship assignments, the original sites (mentors) achieved an average of 88 % of their OKRs and attained an average of 86.7 % and 80.9 % of those OKRs in October–September of 2022 and 2023 respectively during the mentorship implementation phase.</div></div><div><h3>Conclusions</h3><div>The results demonstrate that developing and implementing a research site mentorship model to facilitate onboarding and performance of research sites into an established network was feasible and contributed to the success of those sites.</div></div>","PeriodicalId":37937,"journal":{"name":"Contemporary Clinical Trials Communications","volume":"44 ","pages":"Article 101423"},"PeriodicalIF":1.4,"publicationDate":"2024-12-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11782873/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143080805","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Challenges and lessons learned in recruiting participants for school-based disease prevention programs during COVID-19","authors":"Yelena P. Wu ,&nbsp;Elise K. Brunsgaard ,&nbsp;Nic Siniscalchi ,&nbsp;Tammy Stump ,&nbsp;Heather Smith ,&nbsp;Douglas Grossman ,&nbsp;Jakob Jensen ,&nbsp;David B. Buller ,&nbsp;Jennifer L. Hay ,&nbsp;Jincheng Shen ,&nbsp;Benjamin A. Haaland ,&nbsp;Kenneth P. Tercyak","doi":"10.1016/j.conctc.2024.101399","DOIUrl":"10.1016/j.conctc.2024.101399","url":null,"abstract":"<div><div>Schools provide an ideal setting for delivery of disease prevention programs due to the ability to deliver health education and counseling, including health behavior interventions, to large numbers of students. However, the remote and hybrid learning models that arose during the coronavirus (COVID-19) pandemic created obstacles to these efforts. In this article, we provide insights on collaborating with schools to deliver disease prevention programming during the height of the COVID-19 pandemic, and in subsequent years. We illustrate these strategies by drawing upon our firsthand research experiences engaging high schools in a school-based cancer prevention trial focused on sun safety. Delivery of a cluster-randomized trial of a school-based skin cancer prevention program was initiated in the spring of 2020 at the onset of the COVID-19 pandemic in the U.S. We present multilevel evaluation data on strategies used to reach schools remotely and share lessons learned that may inform similar approaches moving forward during times of crises. Although the COVID-19 pandemic interrupted school-based recruitment for this trial, enrollment improved one year later and did not appear to differ between rural and urban schools. Recruitment strategies and trial-related procedures were modified to address new challenges brought about by the pandemic. Despite the COVID-19 crisis altering US classrooms, disease prevention programming can continue to be offered within schools, given close community partnerships and new adaptations to the ways in which such programming and research are conducted.</div></div>","PeriodicalId":37937,"journal":{"name":"Contemporary Clinical Trials Communications","volume":"42 ","pages":"Article 101399"},"PeriodicalIF":1.4,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11652739/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142855944","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Using community engagement with FRAME: Framework for reporting adaptations and modifications to evidence-based interventions","authors":"Jordana L. Clayton ,&nbsp;Rebecca L. Utz ,&nbsp;Nancy Aruscavage ,&nbsp;Sara G. Bybee ,&nbsp;Sharon E. Bigger ,&nbsp;Eli Iacob ,&nbsp;Kara B. Dassel","doi":"10.1016/j.conctc.2024.101398","DOIUrl":"10.1016/j.conctc.2024.101398","url":null,"abstract":"<div><div>Community engagement is increasingly considered a key component of intervention development, as it can leverage community members’ knowledge, experiences, and insights to create a nuanced intervention which meets the needs, preferences, and realities of the population of interest. Community engagement exists along a spectrum from outreach to the community to partnership with community members and organizations, and all levels of community engagement can benefit from systematic documentation of community feedback and decision-making processes. This paper demonstrates how we utilized the “Framework for Reporting Adaptations and Modifications to Evidence-based Interventions” (FRAME; Wiltsey Stirman et al., 2019) model to track and report adaptations to our dementia end-of-life care planning intervention based on community engagement via a project-specific Community Advisory Board (CAB). Using FRAME, we generated a comprehensive report of the iterative changes made to our pilot intervention, including whether the change was planned, who made the decision to modify the intervention, the nature of the change, its relationship to intervention fidelity, and the reason for the change. This process ensured that we effectively integrated feedback and assistance from our CAB, increased the appropriateness of our intervention for our population of interest, established criteria to monitor intervention fidelity, and prepared our team to run a rigorous clinical trial of the revised intervention. Clinical Trial Registration Number: NCT05909189.</div></div>","PeriodicalId":37937,"journal":{"name":"Contemporary Clinical Trials Communications","volume":"42 ","pages":"Article 101398"},"PeriodicalIF":1.4,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11664155/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142883212","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A simple blinding index for randomized controlled trials","authors":"David Petroff ,&nbsp;Miroslav Bacak ,&nbsp;Nikolaos Dagres ,&nbsp;Patrick Dilk ,&nbsp;Rolf Wachter","doi":"10.1016/j.conctc.2024.101393","DOIUrl":"10.1016/j.conctc.2024.101393","url":null,"abstract":"<div><div>Blinding is an essential part of many randomized controlled trials. However, its quality is usually not checked, and when it is, common measures are the James index and/or the Bang index. In the present paper we discuss these two indices, providing examples demonstrating their considerable weaknesses and limitations, and propose an alternative method for measuring blinding. We argue that this new approach has a number of advantages. We also provide an R-package for computing our blinding index.</div></div>","PeriodicalId":37937,"journal":{"name":"Contemporary Clinical Trials Communications","volume":"42 ","pages":"Article 101393"},"PeriodicalIF":1.4,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142744574","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Examining recollections of Black women with breast cancer who participated in clinical trials: A grounded practical theory study of patient-provider communication","authors":"Katherine E. Ridley-Merriweather ,&nbsp;Oseme Precious Okoruwa ,&nbsp;Katherine Vogel","doi":"10.1016/j.conctc.2024.101403","DOIUrl":"10.1016/j.conctc.2024.101403","url":null,"abstract":"<div><div>The presence of strong barriers to research participation for Black women is indisputable. However, existing evidence supports the possibility of equal levels of participation among members of minoritized populations in past breast cancer (BC) clinical trials (CTs), demonstrating that while these participation barriers undoubtedly exist but are not insurmountable. This work aims to investigate patient-provider conversations to try to illuminate how providers can better engage Black women in communication that will positively influence their perceptions of breast cancer clinical trial participation. Fourteen women (N = 14) who self-identified as Black, Black American, or African American and who had previously or were currently involved in a BC CT participated in the grounded theory-guided study. These women were recruited through emails and social media platforms and interviewed regarding their communication with their medical providers and their pathway to CT participation. Findings revealed three primary categories: 1) participants reported the following provider types as most effective communicators: attentive, matter-of-fact, warm, or above-and beyond; 2) participants frequently received no information about CTs from their providers; and 3) cultural constructs including faith, word of mouth, and storytelling are important to the recruitment of these Black women to BC CTs. Our findings demonstrate the importance of healthcare providers adjusting their communication to meet one of the preferred provider archetypes of communication styles, understanding and incorporating cultural constructs in their communication, and providing information about BC CTs to Black women. Through improved patient-provider communication, healthcare providers may positively influence Black women's perceptions of and participation in BC CTs.</div></div>","PeriodicalId":37937,"journal":{"name":"Contemporary Clinical Trials Communications","volume":"42 ","pages":"Article 101403"},"PeriodicalIF":1.4,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142744573","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

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