RecruitingPhase 1NCT07137338

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

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)

Sponsor

Rocket Pharmaceuticals Inc.

Enrollment

8 participants

Start Date

Jun 1, 2026

Study Type

INTERVENTIONAL

Conditions

Dilated Cardiomyopathy (DCM)

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.

Eligibility

Min Age: 18 YearsMax Age: 65 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying 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) for people with dilated cardiomyopathy (dcm). The study is currently recruiting participants at 3 locations. People eligible for this study include aged 18 Years to 65 Years.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

GENETICRP-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