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RECRUITING
NCT07137338
PHASE1

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.

View on ClinicalTrials.gov

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

Interventions

GENETIC

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