RecruitingDilated Cardiomyopathy (DCM)
A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 Dilated Cardiomyopathy
Eligible age
18–65 yrs
Accepts
All genders
Locations
3 states
Healthy volunteers
No
See if you qualify for this study
Answer a few quick questions about your location and health. Takes about a minute.
About this study
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.
Sponsor: Rocket Pharmaceuticals Inc.
You may qualify if…
- ✓ 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
You may not qualify if…
- ✕ 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
Where it's recruiting
Source: ClinicalTrials.gov · NCT07137338 · last updated 2026-06-30