Status:
NOT_YET_RECRUITING
A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 Dilated Cardiomyopathy
Lead Sponsor:
Rocket Pharmaceuticals Inc.
Conditions:
Dilated Cardiomyopathy (DCM)
Eligibility:
All Genders
18-65 years
Phase:
PHASE1
Brief 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...
Eligibility Criteria
Inclusion
- Subjects are eligible for inclusion into the study only if all the following criteria apply:
- Male or female between 18 and 65 years of age at the time of signing the informed consent
- Capable of and willing to provide signed informed consent
- Clinical diagnosis of DCM defined as and requiring each of the following:
- Mild to moderate systolic dysfunction (LVEF ≥ 25% and ≤ 45%) by echocardiography or CMR performed within 3 months of enrollment.
- Absence of severe coronary artery disease (\>70% stenosis) or active myocardial ischemia as the etiology of LV systolic dysfunction
- Absence of uncontrolled hypertension, significant cardiac valve disease (i.e., greater than moderate in severity), infiltrative disorder, or systemic disease known to cause cardiomyopathy.
- Documentation of a pathogenic or likely pathogenic variant in BAG3
- History of ICD implantation ≥ 3 months prior to enrollment
- NYHA Class II or III HF symptoms with stable HF therapeutic guideline-directed medical regimen for ≥ 30 days enrollment
Exclusion
- CV disease that may be related to a genetic etiology other than a BAG3 pathogenic or likely pathogenic variant.
- Previous participation in a study of gene transfer or gene editing.
- I.V. inotropic, vasodilator, or diuretic therapy ≤ 30 days prior to enrollment.
- History of intracardiac thrombosis or arterial thromboembolic events
- Severe RV dysfunction assessed by echocardiogram or CMR ≤ 12 months prior to screening
- LVEF \< 25% by echocardiogram or CMR at ≤ 3 months prior to screening
- NYHA Class I or IV HF
Key Trial Info
Start Date :
June 1 2026
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
June 1 2029
Estimated Enrollment :
8 Patients enrolled
Trial Details
Trial ID
NCT07137338
Start Date
June 1 2026
End Date
June 1 2029
Last Update
August 22 2025
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