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NCT07137338PHASE1Recruiting

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

Rocket Pharmaceuticals Inc.

Start Date

6/1/2026

Completion Date

6/1/2029

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 Criteria

Age Range: 18 years to 65 years

Inclusion Criteria: Subjects are eligible for inclusion into the study only if all the following criteria apply: 1. Male or female between 18 and 65 years of age at the time of signing the informed consent 2. Capable of and willing to provide signed informed consent 3. Clinical diagnosis of DCM defined as and requiring each of the following: 1. Mild to moderate systolic dysfunction (LVEF ≥ 25% and ≤ 45%) by echocardiography or CMR performed within 3 months of enrollment. 2. Absence of severe coronary artery disease (\>70% stenosis) or active myocardial ischemia as the etiology of LV systolic dysfunction 3. Absence of uncontrolled hypertension, significant cardiac valve disease (i.e., greater than moderate in severity), infiltrative disorder, or systemic disease known to cause cardiomyopathy. 4. Documentation of a pathogenic or likely pathogenic variant in BAG3 5. History of ICD implantation ≥ 3 months prior to enrollment 6. NYHA Class II or III HF symptoms with stable HF therapeutic guideline-directed medical regimen for 30 days prior to enrollment Exclusion Criteria: 1. CV disease that may be related to a genetic etiology other than a BAG3 pathogenic or likely pathogenic variant. 2. Previous participation in a study of gene transfer or gene editing. 3. I.V. inotropic, vasodilator, or diuretic therapy ≤ 30 days prior to enrollment. 4. History of intracardiac thrombosis or arterial thromboembolic events 5. Severe RV dysfunction assessed by echocardiogram or CMR ≤ 12 months prior to screening 6. LVEF \< 25% by echocardiogram or CMR at ≤ 3 months prior to screening 7. NYHA Class I or IV HF

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)

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Conditions

Dilated Cardiomyopathy (DCM)

Locations

University of California, San Diego

San Diego, California 92037

United States

Mayo Clinic

Rochester, Minnesota 55905

United States

Medical University of South Carolina

Charleston, South Carolina 29425

United States