Actively Recruiting
Phase 1 Trial of Intravenous RP-A701 Gene Therapy in Adults with BAG3 Dilated Cardiomyopathy
Led by Rocket Pharmaceuticals Inc. · Updated on 2026-05-07
8
Participants Needed
3
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating RP-A701, a gene therapy delivered through a single intravenous dose, in adults with BAG3-related Dilated Cardiomyopathy (BAG3-DCM). This Phase 1 trial aims to understand the safety, tolerability, and initial effects of this treatment in high-risk patients aged 18 to 65 years who have a specific gene variant causing their heart condition. Participants will receive one dose of RP-A701 on Day 0 and be followed closely for up to two years. The study involves a dose-escalation design with up to two groups receiving increasing amounts of the gene therapy vector. Researchers will monitor how the gene therapy is processed in the body and its impact on heart function and heart failure symptoms. During the study, participants will have regular medical visits to check heart function, quality of life, and any side effects. Tests may include heart imaging like echocardiograms or cardiac magnetic resonance, along with other health assessments done from baseline up to 24 months after treatment. The main focus is to track any adverse events and measure changes in cardiovascular health and quality of life over the study period.
CONDITIONS
Brief 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 aged between 18 and 65 years at consent
- Able and willing to provide signed informed consent
- Diagnosed with Dilated Cardiomyopathy showing mild to moderate systolic dysfunction (LVEF 25% to 45%) confirmed by echocardiography or cardiac MRI within 3 months
- No severe coronary artery disease (>70% stenosis) or active myocardial ischemia causing heart dysfunction
- No uncontrolled hypertension, significant valve disease, infiltrative disorder, or systemic disease causing cardiomyopathy
- Documented pathogenic or likely pathogenic BAG3 gene variant
- History of implantable cardioverter-defibrillator (ICD) placement at least 3 months before enrollment
- NYHA Class II or III heart failure symptoms with stable therapy for at least 30 days prior to enrollment
You will not qualify if you...
- Cardiovascular disease related to genetic causes other than BAG3 variant
- Previous participation in gene transfer or gene editing studies
- Receiving intravenous inotropic, vasodilator, or diuretic therapy within 30 days before enrollment
- History of intracardiac thrombosis or arterial thromboembolic events
- Severe right ventricular dysfunction by echocardiogram or cardiac MRI within 12 months before screening
- Left ventricular ejection fraction below 25% by echocardiogram or cardiac MRI within 3 months before screening
- NYHA Class I or IV heart failure
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Single day
Participants receive a single intravenous dose of RP-A701 gene therapy on Day 0.
1 treatment visit (in-person)
Duration - Up to 24 months post-infusion
Participants are followed for up to 24 months to monitor safety, cardiovascular function, heart failure features, and quality of life after treatment.
Regular visits over 24 months (exact schedule varies)
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
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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