Tag: Clinical trial

Posted on

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. 

Continue to STAT+ to read the full story…

Posted on

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
Posted on

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.

Continue to STAT+ to read the full story…

Posted on

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
<img src="https://jmir-production.s3.us-east-2.amazonaws.com/thumbs/a63127d3ad7741a792dfe16a916ed09f" />

Posted on

OTX-202 Smartphone App to Reduce Suicidal Ideation Among High-Risk Transition-Age Youth: Open-Label, Single-Arm, Phase 1 Clinical Trial

<strong>Background:</strong> The transition from adolescence to adulthood (18 to 25 years) is associated with an increased risk of suicidal ideation and behaviors. Suicide-focused cognitive behavioral therapies (CBTs) have been shown to significantly reduce suicidal ideation and behaviors but are not widely available to high-risk individuals. Digital therapeutics could improve access to these treatments. <strong>Objective:</strong> This study aimed to evaluate the acceptability, safety, and potential efficacy of OTX-202 among transition-age youth (18 to 25 years) receiving mental health care outside an inpatient hospital setting. <strong>Methods:</strong> In this phase 1 single-arm clinical trial, 59 transition-age youth with recent suicidal ideation or suicide attempts used OTX-202, a smartphone app designed to deliver suicide-focused CBT, concurrently with usual outpatient mental health care. After baseline, eligible patients completed 12 weekly assessments of suicidal ideation, depression, and anxiety. <strong>Results:</strong> From baseline to week 12, participants reported statistically significant, large reductions in suicidal ideation (mean difference –5.1, 95% CI –6.5 to –3.7; <i>d</i>=0.95). In total, 3 (5.1%; 95% CI 0%-11.2%) participants reported suicide attempts. Reductions in suicidal ideation and suicide attempt rates were consistent with results from previously published randomized clinical trials of suicide-focused CBTs. Participants rated OTX-202 in the 97th percentile of usability and completed a mean of 9.0 (SD 3.5) of 12 app modules, supporting the app’s acceptability. There were no patient deaths, device-related events, or severe adverse events, supporting the app’s safety. <strong>Conclusions:</strong> Results support the safety, acceptability, and potential efficacy of OTX-202 for reducing suicide risk among transition-age youth. <strong>Trial Registration:</strong> ClinicalTrials.gov NCT06008132; https://clinicaltrials.gov/study/NCT06008132
Posted on

Effects of bifrontal-transcranial direct current stimulation combined with music listening on sleep quality, cortical activation and functional connectivity in patients with insomnia: a randomised controlled trial by fNIRS

BackgroundAlthough music listening and transcranial direct current stimulation (tDCS) alone have certain effects in the treatment of insomnia, the sleep regulatory effects and neural mechanisms of the combined treatment in patients with insomnia disorder (ID) are unclear. This study aimed to investigate the efficacy of combined bifrontal-tDCS (F3: anode, F4: cathode) with music listening in patients with ID using functional near-infrared spectroscopy (fNIRS).Methods76 ID patients were randomly divided into an intervention group (n=38) and a control group (n=38), and received 4 weeks of a total of 20 sessions of music + tDCS therapy and music + sham tDCS therapy (30-second stimulation with fade-in/fade-out to mimic somatic sensations), respectively. The Pittsburgh Sleep Quality Index Scale (PSQI), Self-rating Depression Scale (SDS), Self-rating Anxiety Scale (SAS), and Perceived Stress Scale (PSS-14) were compared between the two groups before and after treatment. Oxy-haemoglobin (HbO2) concentration and functional connectivity (FC) were assessed during the verbal fluency task using fNIRS.ResultsCompared with the control group, the PSQI total score (mean difference: -2.57 points, 95% CI: -4.43 to -0.71, p = 0.001), PSQI sub-scores except “sleep disturbance and daytime dysfunction”, SDS and SAS scores of the intervention group improved significantly after treatment. It was observed by fNIRS that the HbO2 concentration in the medial prefrontal cortex (mPFC), left dorsolateral prefrontal cortex (DLPFC), right ventrolateral prefrontal cortex, and right superior frontal cortex (SFC) increased significantly after treatment in the intervention group but was not superior to the control group. In addition, the FC enhancement of left SFC-left DLPFC and left SFC-mPFC after treatment was significantly better in the intervention group than in the control group, and the PSQI improvement was positively correlated with the FC enhancement of channel-averaged and left SFC-right DLPFC.ConclusionsCombining bifrontal-tDCS with music listening is more helpful in improving sleep quality and prefrontal functional connectivity in ID patients compared with music listening alone. For ID patients, music electrical stimulation headphones may be a safe, effective, and convenient new treatment strategy.Clinical trial registrationhttps://www.chictr.org.cn/, identifier ChiCTR2400086233.
Posted on

Low-dose oral nicotinamide mononucleotide for immune thrombocytopenia: a phase 1/2 trial

Nature Medicine, Published online: 29 April 2026; doi:10.1038/s41591-026-04366-x

Preclinical and phase 1/2 trial data show that anti-CD38 monoclonal antibody treatment restores platelet counts in patients with immune thrombocytopenia by increasing nicotinamide adenine dinucleotide (NAD+) levels, and low-dose oral treatment with the NAD+ precursor nicotinamide mononucleotide can similarly increase platelet counts without serious adverse effects.

Posted on

Opinion: STAT+: Did Kennedy just stack the deck on FDA oversight of peptides?

I’ve been waiting for health secretary Robert F. Kennedy Jr. to do something big on oversight of what I call pop peptides, like BPC-157 and GHK-Cu. He had long signaled that he was going to free such peptides from what he saw as a past, misguided FDA that had banned them in 2023.

It’s finally happened — and the way it went down shook me up a bit.

For a few years, a loophole in compounding rules had allowed specialty pharmacies to make and market these peptides. It effectively meant that substances nominated for compounding — even unproven drugs — could be made and marketed by qualified pharmacies while the FDA pondered the nominations. But in 2023, the Food and Drug Administration rightly moved peptides to a no-compounding-allowed status called Category 2 due to concerns about safety and lack of clinical trial data. Now Kennedy is working to undo that with major risks to the public.

Continue to STAT+ to read the full story…

Posted on

STAT+: FDA launches effort to speed up clinical trials, using AI

WASHINGTON — The Food and Drug Administration on Tuesday announced efforts to make clinical trials more efficient, starting by reviewing data in real time from trials conducted by AstraZeneca and Amgen.  

The agency also asked the public to weigh in on a potential pilot program to work with companies that use AI to enhance safety monitoring and medication dose selections, identify safety signals, and improve patient recruitment in clinical trials. 

AstraZeneca is conducting a Phase 2 trial of its combination therapy for patients with an aggressive form of lymphoma. The trial will take place at the University of Texas MD Anderson Cancer Center and the University of Pennsylvania. Amgen is conducting a Phase 1b trial of its treatment for small cell lung carcinoma. The trials will rely on a real-time data platform built by Paradigm Health. 

Continue to STAT+ to read the full story…