A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 Dilated Cardiomyopathy

Study 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.

Conditions

Dilated Cardiomyopathy (DCM)

Interventions / Drugs

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)

View on ClinicalTrials.gov ↗

Sponsor

RCKT

Rocket Pharmaceuticals

Gene Therapy

C51/100

Study Details

Enrollment8 participants

Primary Completion—

Lead SponsorRocket Pharmaceuticals Inc.

Data Retrieved2026-04-11 04:25:37.504915+00:00

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