Contemporary clinical trials最新文献

Background: Alternatives to daily oral antiretroviral therapy (ART) are important for adolescents with HIV (AHIV) to improve adherence and outcomes. Long-Acting-injectable (LAI) cabotegravir/rilpivirine (CAB/RPV) has demonstrated excellent efficacy and safety and strong patient preference in adults.

Methods: LATA is an ongoing randomised, open-label, 96-week, non-inferiority trial evaluating the efficacy, safety and acceptability of LAI CAB/RPV vs. daily oral therapy with tenofovir (disoproxil fumarate or alafenamide)/lamivudine/dolutegravir (TLD). Participants are virologically suppressed AHIV aged 12- < 20 years in Kenya/South Africa/Uganda/Zimbabwe. Randomisation was 1:1 to LAI CAB/RPV given once every 8 weeks (after optional oral lead-in) or daily oral TLD. The primary outcome is viral rebound (two consecutive viral loads ≥50 copies/mL by 96-weeks). Viral loads are measured every 24 weeks. The trial employs the Smooth Away From the Expected (SAFE) non-inferiority frontier, where the non-inferiority margin depends on the observed event rate in the control arm. Secondary outcomes include confirmed viral load ≥200 copies/mL by 96-weeks, HIV resistance, safety, patient-reported outcomes and cost-effectiveness. LAI participants return to oral ART at confirmed viral load ≥200 copies/mL; LAI participants who become pregnant are given the choice to continue on LAI or to switch back to daily oral ART, with optional pharmacokinetic sampling during pregnancy and post-partum in both groups. Enrolment of 476 AHIV completed in April 2024. Results will be reported in 2026.

Conclusion: LATA is the first trial comparing the efficacy, safety and acceptability of LAI CAB/RPV to oral ART in AHIV, enrolled in Sub-Saharan Africa, using a programmatic approach to viral load testing.

Trial registration: This trial has been registered with ClinicalTrials.gov (NCT05154747).

Background: Patients, particularly those at the end of their lives, frequently receive goal-discordant care, and their surrogate decision-makers suffer long-term psychological injury. Contributors to these issues may include infrequent communication between clinicians and surrogates, failure to discuss prognosis, values, and treatment options that include comfort-focused care, and surrogates facing high-stakes decision-making while underprepared and overwhelmed psychologically and emotionally.

Design: This is a multicenter, patient-randomized efficacy trial of a multi-component intervention, versus usual care, for 370 incapacitated, critically ill adults at high risk of death or severe disability, and their surrogate decision-makers, from 7 hospitals across the United States.

Intervention: The intervention combines surrogate utilization of a digital Family Support Tool (FST) in real-time during their loved one's hospitalization with proactively scheduled family meetings, for which both surrogates and clinicians receive additional preparation, at set intervals during the ICU hospitalization. Those in the control arm will receive usual ICU care.

Outcomes: Our primary outcome is patient-centeredness of care, measured using the modified Patient Perceived Patient-Centeredness of Care (PPPC) scale. Secondary outcomes include surrogates' psychological symptom burden, communication and decision quality, and patients' health resource utilization and clinical outcomes.

Conclusion: This trial will provide robust evidence about the impact of combining the FST with increased and intentional communication, on patient, family, and health system outcomes for those hospitalized in the ICU.

Background: Despite advances in diabetes technology, hypoglycemia remains a major problem for people with type 1 diabetes (T1D). We developed HypoPals, a scalable digital behavioral intervention program comprised of two intervention components, (i) hypoglycemia symptom detection training ('SDT') and (ii) psychoeducation to address unhelpful hypoglycemia beliefs (Education Plus, 'ED+'), to improve hypoglycemia self-management in adults with T1D.

Methods: A 52-week pilot study was conducted to assess the feasibility of a prospective decentralized 2 × 2 factorial trial with the following characteristics: participant recruitment from a national T1D patient registry; minimization randomization; and remote outcome data collection to evaluate the effectiveness of HypoPals intervention components. This pilot study's primary outcome was the number of participants needed to achieve 20 participants completing the intervention. Intervention acceptability was also assessed.

Results: Forty participants were randomized to receive 10 weeks of either SDT only; ED+ only; both; or usual care only. All participants completed the intervention. Thus, 20 participants were recruited to achieve 20 participants completing the intervention. No significant differences in baseline characteristics were observed across randomization groups. Retention was 98% at the 26- and 52-week follow-ups, and 98% of participants provided complete outcome data. The majority (88%) found HypoPals at least somewhat helpful. No adverse events were determined to be related to the intervention.

Conclusions: This pilot trial demonstrated the feasibility of both integrating multiple advanced trial strategies and the intervention's acceptability. Results supported the conduct of a large-scale, decentralized, factorial trial to evaluate HypoPals' effectiveness in reducing hypoglycemia in adults with T1D.

Background: Myasthenia Gravis (MG) is a rare autoimmune disorder with fluctuating weakness and substantial impact on quality of life (QoL). Despite advances in pharmacological treatment, patients often experience fatigue, anxiety, and reduced daily functioning. Adapted Physical Activity (APA) has shown benefits in chronic neuromuscular disorders, while Art Therapy (AT) has demonstrated positive psychosocial effects in neurological conditions. However, no studies have investigated the potential synergy of APA combined with AT in MG.

Methods: We designed a monocentric, prospective, randomized crossover pilot trial to evaluate the feasibility and preliminary efficacy of an integrated APA + AT program compared to APA alone. A total of 102 adult patients with MG will be recruited from Lille University Hospital and partner centers. Each participant will undergo two 6-week phases (APA only vs APA + AT), separated by a 46-week washout. Primary outcome is change in QoL (MG-QOL15) at 3 months. Secondary outcomes include fatigue (FSS), anxiety and depression (HADS), activities of daily living (MG-ADL), physical function tests, physical activity (IPAQ, accelerometry), and psychosocial outcomes assessed through qualitative methods (interviews, ethnography, creative elicitation). Quantitative data will be analyzed with repeated-measures models; qualitative data will undergo thematic analysis.

Discussion: This trial will be the first to explore a combined APA + AT intervention in MG. By addressing both physical and psychosocial dimensions, the study may provide evidence for integrated, patient-centered care strategies in rare neuromuscular disorders. If feasible and effective, results could inform future multicenter trials and clinical practice.

Trial registration: ClinicalTrials.gov Identifier: NCT07125105.

Background: Cardiovascular disease (CVD) is the leading cause of mortality in China, with hypertension as the most common modifiable risk factor. Despite comprehensive primary care based on chronic disease management, patients' awareness of CVD risk and motivation for self-management remain suboptimal.

Methods: This study is a parallel-group, individually randomized controlled trial conducted within routine chronic disease management at a community health center in Kunshan, China. Approximately 400 adults aged 60 years and above with hypertension will be randomly allocated (1:1) to receive either a personalized Heart Age report with brief physician explanation or usual care. The primary outcome is CVD risk awareness at 3 months. Secondary outcomes include changes in self-reported physical activity, diet, and medication adherence. An exploratory analysis of clinical indicators (blood pressure, glucose, lipids, BMI) will be conducted at 12 months. Implementation outcomes (feasibility, acceptability, fidelity) will also be assessed.

Discussion: This pragmatic trial integrates a personalized Heart Age risk communication model into routine chronic disease management. By embedding brief, physician-delivered risk feedback within existing primary care workflows, the intervention seeks to improve patient awareness and encourage self-management behaviors without increasing resource burden. Findings will inform the feasibility and scalability of personalized risk communication strategies in low-resource primary care settings.

Trial registration numbers: NCT07045363.

Background: Despite advances in treatments, children with type 1 diabetes struggle to achieve glycemic targets. Though school-based programs may help support families to achieve targets, challenges remain with coordinating care between health systems and schools, where students spend much of their day. Monthly, virtual meetings between a parent, school nurse, and a diabetes specialist may bridge this gap by using a shared treatment plan to meet goals.

Methods: This is a pilot cluster-randomized trial to test a school-partnered collaborative care model (SPACE for T1D) for children aged 5-12 years with type 1 diabetes for ≥6 months. School districts were randomized in a 2:1 allocation ratio to the intervention (SPACE for T1D) or enhanced usual care. Participants complete the study over four consecutive months. Parents and school nurses evaluate the feasibility, acceptability, appropriateness, and usability of SPACE using valid questionnaires. The study team monitors retention, fidelity, and practicality. Exploratory outcomes assess change in glycemia, care utilization, type 1 diabetes-related quality of life, self-efficacy skills, and missed class time due to diabetes interventions.

Results: Twelve school districts were randomized. One dropped out due to no eligible students, leaving 11 participating districts. We approached 46 students; 30 provided consent for a recruitment rate of 65%.

Conclusions: This study will evaluate the feasibility and acceptability of the SPACE for T1D intervention in varied school districts. Findings will facilitate a fully-powered trial to evaluate the impact on health and academic outcomes, adding to the evidence base of school-based interventions to support youth with chronic disease. NCT: NCT06420661.

Background: The current paper describes the protocol for the Hybrid type 1 effectiveness-implementation trial titled Multilevel Action Toward Colorectal Cancer and Hepatitis C Education and Screening (MATCHES). The aims of the MATCHES intervention trial are to promote both colorectal cancer (CRC) and hepatitis C virus (HCV) screening among patients aged 45-75 years old receiving primary care at a federally qualified health center (FQHC) who are not currently up-to-date with both screenings and to examine implementation outcomes.

Methods: Ten health center sites in the FQHC will be randomized in two waves using a stepped wedge design. Intervention strategies target the system, clinician, and patient levels. System-level strategies include a customized electronic health record best practice alert that pairs CRC and HCV screening and standard operating procedures. Clinician-level strategies include a training session and feedback on screening rates. Patient-level strategies include a theory-informed educational booklet about CRC and HCV screening in English or Spanish and access to stool DNA testing and HCV screening among 350 participants. The primary outcome is both CRC and HCV screening uptake at 12 months post-intervention among 350 participants. System-wide CRC and HCV screening rates among patients 45-75 years old also will be monitored. Implementation barriers and facilitators will be examined.

Results: The project is ongoing; effectiveness and implementation outcomes will be determined following study completion.

Conclusions: Results may inform potential subsequent scale-up and offer insights for promoting multiple screening behaviors in a single intervention.

Clinical trials identifier: NCT06745895 (date registered: 2024-12-20); https://clinicaltrials.gov/study/NCT06745895?cond=CRC%20AND%20HCV&rank=.