Actively Recruiting
Phase 1 Study of Intravenous RP-A701 Gene Therapy for Adults With BAG3-Related Dilated Cardiomyopathy
Led by Rocket Pharmaceuticals Inc. · Updated on 2026-06-30
8
Participants Needed
3
Research Sites
N/A
Total Duration
AI-Summary
What this Trial Is About
Researchers are evaluating the safety, tolerability, and early effects of RP-A701, a gene therapy using a recombinant viral vector, in adults with high-risk BAG3-related dilated cardiomyopathy BAG3-DCM. This Phase 1, open-label trial aims to better understand how this treatment works in patients who have a specific genetic variant causing their heart condition. Participants will receive a single intravenous dose of RP-A701 on Day 0 and will be followed for up to two years. The study includes a dose-escalation design with up to two consecutive groups receiving increasing doses to evaluate safety and effects. During the study, participants will be monitored for treatment-related adverse events and serious side effects for up to 24 months after infusion. Researchers will assess heart function, heart failure features, protein expression of the gene therapy, and quality of life throughout the follow-up period. Participants will have regular evaluations including echocardiography or cardiac magnetic resonance imaging and clinical assessments.
CONDITIONS
Brief Title
A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 Dilated Cardiomyopathy
Research Team
C
Clinical Information
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