Contemporary clinical trials最新文献

Introduction

Women with atypical hyperplasia (AH) or lobular carcinoma in situ (LCIS) have a significantly increased risk of breast cancer, which can be substantially reduced with antiestrogen therapy for chemoprevention. However, antiestrogen therapy for breast cancer risk reduction remains underutilized. Improving knowledge about breast cancer risk and chemoprevention among high-risk patients and their healthcare providers may enhance informed decision-making about this critical breast cancer risk reduction strategy.

Methods/design

We are conducting a cluster randomized controlled trial to evaluate the effectiveness and implementation of patient and provider decision support tools to improve informed choice about chemoprevention among women with AH or LCIS. We have cluster randomized 26 sites across the U.S. through the SWOG Cancer Research Network. A total of 415 patients and 200 healthcare providers are being recruited. They are assigned to standard educational materials alone or combined with the web-based decision support tools. Patient-reported and clinical outcomes are assessed at baseline, after a follow-up visit at 6 months, and yearly for 5 years. The primary outcome is chemoprevention informed choice after the follow-up visit. Secondary endpoints include other patient-reported outcomes, such as chemoprevention knowledge, decision conflict and regret, and self-reported chemoprevention usage. Barriers and facilitators to implementing decision support into clinic workflow are assessed through patient and provider interviews at baseline and mid-implementation.

Results/discussion

With this hybrid effectiveness/implementation study, we seek to evaluate if a multi-level intervention effectively promotes informed decision-making about chemoprevention and provide valuable insights on how the intervention is implemented in U.S. clinical settings.

Trial Registration

NCT04496739

Background

A 12-week multicomponent frailty management program - Say No To Frailty (SNTF) consisting of interactive talks and fitness exercises led by a trained program leader has shown feasibility and positive health outcomes in community-living older adults with frailty and pre-frailty in Singapore. This study aims to evaluate the clinical- and cost-effectiveness of SNTF on physical functions, self-confidence, community participation, quality of life and fall reduction in the local community setting.

Methods

This study will use the cluster-randomization method to randomly allocate 12 participating centres into three arms. Centres under two intervention arms will conduct the same SNTF program but led by a program leader with different training backgrounds (an Allied Health Professional (AHP) v.s. a non-AHP), whereas centres under the control arm will continue their usual care without an additional intervention. Eligible participants at each participating centre will be recruited via the convenience sampling method in the community setting. Primary outcome measure (frailty level) and secondary outcome measures (e.g., physical functions, self-confidence, community participation, quality of life) will be conducted by the blinded assessors at baseline, immediate, 3 months and 9 months post-intervention. Fall data will be collected during the one-year study period. Outcomes between and within groups will be compared and analysed using STATA to evaluate the clinical effectiveness. Program costs and relevant healthcare costs during the follow-up phase will be recorded for cost-effectiveness analysis.

Conclusion

This study will provide significant insights into conducting SNTF for Singapore community-living older adults with frailty and pre-frailty on clinical- and cost-effectiveness.

Australia New Zealand Clinical Trials Registry: ACTRN12621001673831.

Background

Negative affect is prevalent among adolescents with type 1 diabetes (T1D) and may impact diabetes self-management and outcomes through stress-related behaviors such as disordered eating.

Methods

We describe the development of and design for the adaptation of a mindfulness-based intervention (MBI) for adolescents with T1D and negative affect. BREATHE-T1D is an MBI designed to target negative affect that has been tailored to address the unique lived experiences of adolescents with T1D. Qualitative interviews with stakeholders and participants were used to inform iterative adaptations to the intervention and control curricula over the course of the study. The primary aim of this paper is to describe the design, development, and protocol of the present pilot feasibility trial.

Conclusions

Iterative, qualitative methodology throughout the adaptation of an intervention is important for ensuring the resulting intervention is relevant and meaningful for the target population.

Clinical Trial Registration Number: NCT05268393

Clinical trials evaluate the safety and efficacy of treatments for specific diseases. Ensuring these studies are well-powered is crucial for identifying superior treatments. With the rise of personalized medicine, treatment efficacy may vary based on biomarker profiles. However, researchers often lack prior knowledge about which biomarkers are linked to varied treatment effects. Fixed or response-adaptive designs may not sufficiently account for heterogeneous patient characteristics, such as genetic diversity, potentially reducing the chance of selecting the optimal treatment for individuals. Recent advances in Bayesian nonparametric modeling pave the way for innovative trial designs that not only maintain robust power but also offer the flexibility to identify subgroups deriving greater benefits from specific treatments. Building on this inspiration, we introduce a Bayesian adaptive design for multi-arm trials focusing on time-to-event endpoints. We introduce a covariate-adjusted response adaptive randomization, updating treatment allocation probabilities grounded on causal effect estimates using a random intercept accelerated failure time BART model. After the trial concludes, we suggest employing a multi-response decision tree to pinpoint subgroups with varying treatment impacts. The performance of our design is then assessed via comprehensive simulations.

Introduction

The National Health Service (NHS) in England is currently piloting a weight loss programme for remission of newly diagnosed type 2 diabetes (T2D), where participants replace all food with low-energy nutritionally complete formula products for 12 weeks (total diet replacement, TDR) and receive behavioural support. In a clinical trial, this programme led to remission in nearly half the participants. However, this weight loss programme might also worsen disordered eating and prompt eating disorders in susceptible people. We aim to investigate if the TDR programme is non-inferior to standard care in terms of disordered eating in susceptible individuals.

Methods

Fifty six people with newly diagnosed T2D, BMI ≥ 27 kg/m², and medium to high scores of disordered eating based on the Eating Disorders Examination questionnaire (EDE-Q) will be randomised 1:1 to TDR receiving remote weekly/bi-weekly dietetic support or standard care. Participants will be re-assessed remotely at 1, 3, 4, 6, and 12 months. The primary outcome will be the between-group difference in the score of the EDE-Q. If the sample size can be expanded to 150, we will reduce the non-inferiority boundary. Weight, glycated haemoglobin (HbA1c), impairment from disordered eating, and distress will be secondary outcomes. Using the recorded consultations, we will evaluate the process in observed changes in eating behaviour and disordered eating.

Conclusions

If TDR for T2D remission is deemed non-inferior to standard care, more people may enrol and benefit from T2D remission. If TDR exacerbates disordered eating, screening may reduce unintended harm.

Trial Registration: NCT05744232 (ClinicalTrials.gov, prospectively registered).

Background

Although patients undergoing hematopoietic stem cell transplantation (HSCT) must cope with psychological distress and isolation during an extended transplant hospitalization, psychosocial interventions to address these unmet needs are lacking. Virtual reality offers an innovative modality to deliver a patient-centered psychosocial intervention to address psychosocial needs of patients undergoing HSCT. However, there are currently no supportive care interventions leveraging virtual reality in patients undergoing HSCT.

Objective

To describe the methods of a randomized clinical trial (RCT) to assess the feasibility and preliminary efficacy of a self-administered, virtual reality-delivered psychosocial intervention (BMT-VR) to improve psychological distress and quality of life (QOL) for patients hospitalized for HSCT.

Methods

This study entails a single-center RCT of BMT-VR compared to usual transplant care in 80 patients hospitalized for HSCT. Adult patients with hematologic malignancies hospitalized for autologous or allogeneic HSCT are eligible. BMT-VR includes psychoeducation about the HSCT process, psychosocial skill building to promote effective coping and acceptance, and self-care and positive psychology skills to promote post-HSCT recovery. The primary aim is to assess the feasibility defined a priori as ≥60% of eligible patients enrolling in the study, and of those enrolled and randomized to the BMT-VR, ≥ 60% completing 4/6 BMT-VR modules. Secondary objectives include assessing the preliminary effects on psychological distress and QOL.

Discussion

This is the first RCT of a virtual reality-delivered psychosocial intervention for the HSCT population. If deemed feasible, a future larger multi-site clinical trial can evaluate the efficacy of BMT-VR on outcomes for patients hospitalized for HSCT.

Background

The majority of adults suffering from alcohol use disorders (AUD) do not receive treatment. To address this gap in care, we must develop new models to increase identification, engagement and delivery of accessible and effective treatment. This paper describes the protocol for a randomized controlled trial (RCT) testing a novel telehealth treatment model for primary care patients with untreated AUD.

Methods

We aim to recruit 300 adults across 2 healthcare systems for this two-arm RCT. Participants, initially identified for recruitment based on AUD-related indicators in their electronic health record (EHR), are RCT-eligible if they meet AUD criteria (mild, moderate, severe), report ≥ 3 drinking days/week in past 30 days, and have not received AUD psychotherapy in the past 90 days. Participants are randomized to an intervention or enhanced usual care control (EUC) condition, both individually-delivered. The intervention includes a telephone-delivered motivational interviewing (MI) engagement session and 8 sessions of MI-Cognitive Behavioral Therapy (MI-CBT). EUC involves AUD psychoeducation, advice to reduce drinking and seek treatment, and provision of community resources. Outcomes will be measured at 3-, 6-, and 12-months; primary outcomes include: AUD psychotherapy initiation and engagement (within the study and external community) and alcohol consumption (percent drinking days and heavy drinking days).

Conclusions

This study addresses whether proactive patient identification and engagement and delivery of patient-centered telehealth psychotherapy to patients with untreated AUD is effective in increasing treatment use and improving alcohol outcomes. If effective, this could be a highly scalable model for reducing the public health impact of AUD.

Trial Registration

ClinicalTrials.gov # NCT05410561. University of Michigan HUM00204315. Ann Arbor VA IRB #1655886.

Background

Pulmonary hypertension is a progressive disease for which early treatment interventions are essential. Traditionally, patients undergo periodic clinical assessments. However, recent advances in wearable technology could improve the quality and efficiency of follow-up monitoring in patients with pulmonary hypertension.

Trial design

To our knowledge, this is the first study describing direct data transmission from a smartwatch to patients' electronic health records. It implements a novel update and customised program to continuously and automatically transmit data from a smartwatch to the patient's electronic healthcare records. It will evaluate continuous monitoring in patients with pulmonary hypertension and monitor their physical activity time, heart rate variability, and heart rate at rest and during physical activity via a smartwatch. It will also evaluate the data transmission method, and its data will be assessed by the treating physicians supplemental to clinical practice.

Smartwatch integration promises numerous advantages: comprehensive cardiovascular monitoring and improved patient experience. Our continuous smartwatch monitoring approach offers a solution for earlier detection of clinical worsening and could be included as a combined endpoint in future clinical trials. It could improve patient empowerment, enhance precision medicine, and reduce hospitalisations. The user-friendly smartwatch is designed to minimise disruption in daily life.

Conclusion

The ability to transfer real-time data from wearable devices to electronic health records could help to transform the treatment of patients with pulmonary hypertension and their follow-up monitoring outside a clinical setting, enhancing the efficiency of healthcare delivery.

Background

Given the increasing number of young adult cancer survivors and the impacts of cancer on various life domains, interventions addressing the psychosocial needs of young adult survivors are crucial. However, such intervention research is limited, and the existing literature has often: 1) overlooked young adult survivors' psychosocial needs; 2) targeted depression, anxiety, or fear of recurrence – rather than positive outcomes like well-being; and 3) failed to consider scalable approaches, like digital health.

Methods

This paper documents the development and refinement of an 8-week digital, coach-assisted intervention targeting hope among young adult cancer survivors (ages 18–39, within 3 years of treatment completion) and presents the protocol of the 2-arm RCT (comparing intervention vs. attention control). The intervention builds upon a 2017–2018 pilot trial (n = 56); intervention refinements were based on subsequent semi-structured interviews among young adult survivors (n = 23).

Results

The pending trial design involves an increased sample size (n = 150) to increase power and diversified recruitment efforts (i.e., clinic-based, social media, community-based organizations, etc.) to facilitate intervention reach, accessibility, and scalability. The intervention was enhanced by integrating highly relevant theoretical and therapeutic frameworks, specifically the concept of hope and Acceptance and Commitment Therapy, as well as updating intervention delivery technology. Intervention outcomes include feasibility and acceptability at end-of-treatment and preliminary efficacy on hope (primary outcome) and quality of life measures (secondary outcomes) at end-of-treatment and 16-week follow-up.

Conclusions

This paper may facilitate discussion regarding approaches for addressing the significant psychosocial challenges faced by young adult survivors and catalyze dissemination of trial results.

Trial Registration: NCT05905250