Tag: Clinical trial

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Barriers and Facilitators in the Implementation of the Systematic Medical Appraisal, Referral, and Treatment (SMART) Mental Health Digital Intervention in Rural India: Mixed Methods Process Evaluation Study

<strong>Background:</strong> An estimated 150 million people have mental health care needs in India, but only 15% are able to access care. Depression and anxiety contribute to a large proportion of mental morbidity. The Systematic Medical Appraisal, Referral, and Treatment (SMART) Mental Health trial used a mobile-based clinical decision support system for primary care doctors and community health workers (CHWs) to identify and treat people at risk of depression, anxiety disorders, and self-harm. A community-based antistigma campaign was also delivered. The intervention led to improved remission rates for depression and anxiety and lower stigma scores. <strong>Objective:</strong> A process evaluation assessed (1) implementation fidelity, barriers, and facilitators; (2) perceptions of doctors and CHWs on the use of SMART Mental Health; and (3) the causal pathways that led to trial outcomes. <strong>Methods:</strong> A mixed methods evaluation combining backend program data and qualitative data was conducted. A total of 38 focus group discussions and 37 key informant interviews were conducted with primary doctors, CHWs, government officials, local community leaders, and research project staff. The data were coded and analyzed using a framework analysis approach based on the UK Medical Research Council guidance on process evaluations and the Reach, Effectiveness, Adoption, Implementation, and Maintenance framework. <strong>Results:</strong> The intervention had high implementation fidelity. Across clusters, the median proportion of participants with at least 1 CHW follow-up was 98% (IQR 96.6%-100%). The referral rate for a psychiatrist was low (224/1697, 13.2%), and only 23.6% (53/224) of those referred visited the psychiatrist. The median exposure to antistigma audiovisual content was 84% (IQR 65.7%-95.9%). At the community level, key implementation barriers included cultural inhibitions in seeking mental health care and the unavailability of patients due to competing demands. Proximity and tight social connections between CHWs and their communities were important facilitators in seeking medical help. Doctor and CHW training, mentoring, and feedback provided by program staff were important facilitators to support the use of the digital health components by the health workforce. <strong>Conclusions:</strong> A complex intervention that included both community-based antistigma and clinical digital health interventions achieved high implementation fidelity. Key areas to consider for maintenance of such interventions include (1) the need for sustained community-based strategies to address stigma and other cultural barriers; (2) health workforce strengthening policies, including supportive supervision for CHWs and doctors to increase capability in the use of mental health digital health tools; and (3) strategies to improve access to specialist care for those with more complex care needs. <strong>Trial Registration:</strong> Clinical Trial Registry India CTRI/2018/08/015355; https://tinyurl.com/5r63suxp
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A Sustainable Lifestyle Intervention Among Office Workers: Cluster Randomized Pilot and Feasibility Study

Background: Society faces multiple challenges, including lifestyle diseases and global climate change. Framing health education within sustainable development may enhance motivation for behavior change because proenvironmental behaviors, as well as healthy behaviors, often rely on the same behavior change principles. Combining these perspectives may therefore reinforce health behaviors and climate-friendly choices. Objective: This pilot study aims to explore changes in dietary intake, diet-related carbon footprint, and physical activity among office workers receiving sustainable plus healthy lifestyle (sustainable lifestyle arm) or healthy lifestyle education (healthy lifestyle arm) alone. It also aims to assess the feasibility of the intervention functions, including workshop attendance rate, participants’ dietary goals, social support, and facilitators and barriers to behavior change. Methods: A 2-armed participant-blinded cluster randomized study, including an experimental intervention arm (sustainable lifestyle; n=19) and a control intervention arm (healthy lifestyle; n=14), was conducted in Sweden. The study lasted 8 weeks and included 6 workplace-based workshops and was framed by the behavioral change wheel and the socioecological model. Diet, carbon footprint, and physical activity were assessed using the web-based questionnaires Meal-Q and Active-Q. Attendance rate, individual goals, social support, and facilitators and barriers were assessed using printed questionnaires. Results: The reduction of total diet-related carbon dioxide equivalents (COe) was 0.8 kg and 0.4 kg per day for the sustainable and healthy lifestyle arm, respectively. Also, there was a statistically significant interaction between time and lifestyle when the carbon footprint was expressed as a qualitative aspect of diet, that is, COe kg per 1000 kcal per day (=.05). Moreover, the intake of vitamin C, a marker for fruits and vegetables, increased to 8.0 and 12.5 mg per 1000 kcal per day for the sustainable and healthy lifestyle arms, respectively. In addition, total sedentary time decreased by 0.4 hours per day in the sustainable lifestyle arm, but not in the healthy lifestyle arm. This indicates that the educational workshops in respective arms had different impacts on health behavior over time. Minor differences were found in dietary goals, with the sustainable lifestyle arm setting more goals related to ecological and vegetarian foods. No differences were seen between arms regarding barriers or facilitators. Conclusions: This study suggests that embedding healthy lifestyle recommendations within a sustainable development context may be an efficient way to reduce carbon footprint and increase healthy behavior among office workers. Given the ongoing global epidemic of metabolic diseases, climate change, and environmental degradation, promoting a sustainable lifestyle in a workplace context has the potential to counteract these trends. Trial Registration: ClinicalTrials.gov NCT06698094; https://clinicaltrials.gov/study/NCT06698094
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STAT+: FDA to reconsider treatment for rare cancer after its surprise rejection

Two companies developing a therapy for a rare blood cancer have reached an agreement with the Food and Drug Administration that walked back the agency’s main reason for rejecting the drug in January. 

Pierre Fabre Pharmaceuticals and Atara Biotherapeutics, makers of the drug called Ebvallo, said Thursday that a meeting held in late April with FDA officials ended with the agency agreeing that their already completed, single-arm clinical trial was sufficient to support a review and potential approval. 

When the FDA rejected Ebvallo, the agency said the same study was flawed and the data produced from it was “insufficient” to support the drug’s approval. The drug’s review was conducted by the FDA’s Center for Biologics Evaluation and Research, led at the time by Vinay Prasad. He departed the agency at the end of April. 

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STAT+: Cytokinetics drug Myqorzo meets twin efficacy goals in study of genetic heart disease

Cytokinetics said Tuesday that its drug Myqorzo significantly improved heart failure symptoms and cardiovascular fitness in patients with non-obstructive hypertrophic cardiomyopathy, an inherited heart disorder. 

The results, reported in a company press release, achieved the dual efficacy goals of a Phase 3 clinical trial, called ACACIA, with statistical significance.

Cytokinetics is in the early days of Myqorzo’s commercial launch as a treatment for the more severe “obstructive” form of hypertrophic cardiomyopathy, or HCM. The successful outcome of the ACACIA study, if also cleared by regulators, could greatly expand the number of HCM patients eligible for treatment — and boost the drug’s peak sales to $5 billion annually, according to analyst forecasts. 

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Usability, Acceptability, and Feasibility of a Personalized Adaptive Mirror Therapy for Upper-Limb Poststroke Rehabilitation Using Immersive Virtual Reality and Myoelectric Control: Single-Arm Pre-Post Study

<strong>Background:</strong> Stroke remains a primary cause of long-term disability worldwide, with upper-limb deficits affecting up to 80% of survivors acutely and 40% chronically. These deficits lead to considerable effects on their independence and overall quality of life. Conventional rehabilitation therapies are most effective when initiated shortly after a stroke, yet many patients face barriers to ongoing therapy post discharge. Recent advancements in low-cost rehabilitation systems, particularly those using virtual reality (VR) technologies, offer promising alternatives for enhancing upper-limb recovery. <strong>Objective:</strong> Given the burden on health care systems and the limitations in access to high-intensity postdischarge rehabilitation, this study aimed to evaluate the feasibility, acceptability, and usability of an upper-limb adaptive mirror therapy using VR and myoelectric control for the rehabilitation of patients with chronic stroke developed through a user-centered design approach. <strong>Methods:</strong> In this study, a total of 12 community-dwelling survivors of chronic stroke (mean age 52.9, SD 16.0 years; 4 female) with moderate to severe upper-limb impairments were enrolled. Participants were stratified by age (young: 18-55 years; older: 56-80 years) and impairment level (Fugl-Meyer Assessment-Upper Extremity score: 18-36=severe; 37-54=moderate). Acceptability was assessed for each session by patient self-evaluation of satisfaction and motivation through a visual analog scale, while the therapist assessed the patient’s participation in therapy using the Pittsburgh Participation Rehabilitation Scale. Usability was measured with the User Satisfaction Evaluation Questionnaire scale and feasibility through the NASA (National Aeronautics and Space Administration) Task Load Index cognitive workload indices. <strong>Results:</strong> Patients reported a significant increase in satisfaction from the intermediate to the final assessment (T1: 72% vs T2: 85%; <i>P</i>=.01) and stable high motivation levels. Differences in participation and motivation were observed based on impairment levels, with no effect of age. Usability ratings remained high (&gt;80%) throughout the intervention, with no significant differences between baseline and end line (<i>P</i>=.56). Cognitive workload assessments showed a significant reduction over time, in perceived cognitive (<i>P</i>=.04), performance (<i>P</i>=.007), and effort demands (<i>P</i>&lt;.001). Impairment level significantly influenced perceived workload, with participants with more severe impairment reporting higher cognitive, physical, temporal, and effort demands (all <i>P</i>&lt;.001), while age did not contribute to variability in acceptability, usability, or workload measures. <strong>Conclusions:</strong> VR therapy was found to be feasible, under adaptive task conditions, ensuring stable performance across patients. The protocol was usable and acceptable among patients with chronic stroke, especially those with moderate impairment, supporting its potential as a user-centered digital rehabilitation tool, warranting further investigation in controlled and home-based settings. <strong>Trial Registration:</strong> ClinicalTrials.gov NCT07103122; https://clinicaltrials.gov/study/NCT07103122
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STAT+: Biotech raises $42 million to run Huntington’s disease trial

Gene therapy startup Latus Bio has raised another $42 million to start its first clinical trials, where it will try to sidestep issues that have set back a more advanced competitor. 

Latus is moving two treatments through clinical trials this year. The first is for a form of Batten disease called CLN2 disease, a fatal genetic condition that causes seizures, vision loss, and cognitive problems. The company anticipates having initial clinical data by the end of the year. 

Now, Latus — founded by Beverly Davidson, chief scientific strategy officer at the Children’s Hospital of Philadelphia — is turning its attention to a second drug candidate, a gene therapy for Huntington’s disease.

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A Bilingual AI-Based Chatbot for Nutrition Education in a Food Is Medicine Intervention for High-Risk Pregnant Women: Design and Development Study

Background: Conversational agents (artificial intelligence [AI]–based chatbots) offer a novel approach to health interventions by providing personalized, adaptive interactions that improve over time based on user engagement. In nutrition education, given the wide variation in knowledge, skills, and abilities across participants, AI-based chatbots have the potential to enhance accessibility, engagement, and behavior change. Food is Medicine (FIM) interventions, which aim to improve food security and diet quality among multicultural, at-risk populations, often face challenges related to sustained engagement and use. Objective: This paper describes the design, development, and iterative refinement of a bilingual AI-driven nutrition chatbot integrated into an FIM intervention for high-risk pregnant women receiving care at obstetric clinics in Houston, Texas. Methods: The chatbot was developed using an iterative process informed by behavioral theory, human-centered design (HCD), and plan-do-study-act (PDSA) quality improvement cycles. The conversational agent was embedded within an ongoing 2-arm randomized controlled trial (N=200) comparing standard FIM nutrition education to FIM plus AI-driven nutrition chatbot support. HCD activities took place prior to deployment and involved community advisory group members and implementation stakeholders. Postdeployment refinements were guided by 2 PDSA cycles and informal question-and-answer sessions conducted with intervention arm participants. Qualitative feedback was collected using structured scripts to identify facilitators of and barriers to chatbot engagement. Results: The chatbot was developed using the GPT-3.5 Turbo application programming interface. An initial prototype built in Python using Gradio enabled rapid testing but lacked flexibility for modifications. To improve scalability and logging capabilities, the system was rebuilt using PHP, HTML, JavaScript, and SQL. To further understand usage patterns, participants who interacted with the chatbot at least once or not at all (classified as low users; n=32) were engaged in question-and-answer sessions. Of these participants, all were female (32/32, 100%), 88% (28/32) identified as Hispanic or Latino, and 90% (29/32) preferred Spanish. Two PDSA cycles guided iterative refinements. Cycle 1 identified low initial engagement, whereas cycle 2 focused on improving content clarity and cultural relevance through physical reminder prompts. Qualitative findings identified key barriers to engagement, including high cooking self-efficacy with perceived lack of need for support, low technology self-efficacy, and low urgency due to competing priorities. Conclusions: Embedding a bilingual AI-driven nutrition chatbot within an FIM intervention was feasible and featured critical design and implementation considerations for engaging high-risk pregnant populations. Findings show the importance of HCD and iterative refinement to address engagement barriers. This work provides actionable guidance for integrating conversational agents into FIM programs, with implications for future evaluation of clinical outcomes, long-term engagement, and scalability. Trial Registration: ClinicalTrials.gov NCT07165990; https://clinicaltrials.gov/study/NCT07165990
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