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A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 Dilated Cardiomyopathy
Sponsor: Rocket Pharmaceuticals Inc.
Summary
This is a Phase 1, open-label, dose-escalation trial to characterize the safety, tolerability, and preliminary efficacy of RP-A701 following a single IV administration in high-risk adult patients with BAG3-DCM.
Official title: A Phase 1 Dose Escalation Trial Evaluating an Intravenously Administered Recombinant Adeno-associated Virus Serotype rh.74 (AAVrh.74) Vector Containing the Human BCL2-associated Athanogene 3 (BAG3) Gene Coding Sequence (RP-A701) in Subjects With Dilated Cardiomyopathy Arising From Pathogenic BAG3 Variants (BAG3-DCM)
Key Details
Gender
All
Age Range
18 Years - 65 Years
Study Type
INTERVENTIONAL
Enrollment
8
Start Date
2026-06
Completion Date
2029-06
Last Updated
2026-04-02
Healthy Volunteers
No
Conditions
Interventions
RP-A701 is a recombinant viral vector composed of an AAV serotype rh.74 (AAVrh.74) capsid encapsulating the transgene, BCL2-associated Athanogene 3 (BAG3)
One-time treatment with a single ascending dose
Locations (3)
University of California, San Diego
San Diego, California, United States
Mayo Clinic
Rochester, Minnesota, United States
Medical University of South Carolina
Charleston, South Carolina, United States