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NCT07137338Phase 1RECRUITING

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
C60/100
Study Details
Enrollment8 participants
Primary Completion—
Lead SponsorRocket Pharmaceuticals Inc.
Data Retrieved2026-04-11 04:25:37.504915+00:00
What This Trial Means for Biotech Investors
Trial Phase Context

Phase 1 trials primarily test safety and dosing in a small number of participants (typically 20–100). Results do not yet speak to efficacy. FDA approval is typically many years away from this stage.

Status & Expected Data

Currently recruiting: the trial is enrolling patients. A data readout is not expected until after enrollment closes and the follow-up period is complete.

Enrolled: 8 participants
Sponsor Catalyst Profile

Rocket Pharmaceuticals (RCKT) is the sponsoring company for this trial. BiotechSign currently grades this company C (60/100) based on composite catalyst signals across its full pipeline. This trial is one data point in that overall catalyst picture.

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