Actively Recruiting
A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 Dilated Cardiomyopathy
Led by Rocket Pharmaceuticals Inc. · Updated on 2026-05-07
8
Participants Needed
3
Research Sites
156 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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
Official Title
A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 Dilated Cardiomyopathy
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male or female between 18 and 65 years of age at the time of signing the informed consent
- Able and willing to provide signed informed consent
- Clinical diagnosis of dilated cardiomyopathy with mild to moderate systolic dysfunction (LVEF ≥ 25% and ≤ 45%) confirmed by echocardiography or cardiac MRI within 3 months
- No severe coronary artery disease (>70% stenosis) or active myocardial ischemia causing left ventricular dysfunction
- No uncontrolled hypertension, significant heart valve disease, infiltrative disorder, or systemic disease causing cardiomyopathy
- Documented pathogenic or likely pathogenic variant in the BAG3 gene
- History of implantable cardioverter defibrillator (ICD) implantation at least 3 months prior to enrollment
- NYHA Class II or III heart failure symptoms with stable heart failure treatment for at least 30 days before enrollment
You will not qualify if you...
- Cardiovascular disease potentially caused by a genetic variant other than BAG3
- Previous participation in gene transfer or gene editing studies
- Use of intravenous inotropic, vasodilator, or diuretic therapy within 30 days before enrollment
- History of intracardiac thrombosis or arterial thromboembolic events
- Severe right ventricular dysfunction assessed by echocardiogram or cardiac MRI within 12 months before screening
- Left ventricular ejection fraction (LVEF) less than 25% by echocardiogram or MRI within 3 months before screening
- NYHA Class I or IV heart failure
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 3 locations
1
University of California, San Diego
San Diego, California, United States, 92037
Actively Recruiting
2
Mayo Clinic
Rochester, Minnesota, United States, 55905
Actively Recruiting
3
Medical University of South Carolina
Charleston, South Carolina, United States, 29425
Actively Recruiting
Research Team
C
Clinical Information
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
1
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