A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in
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.
| Condition(s) | Dilated Cardiomyopathy (DCM) |
|---|---|
| Status | Recruiting |
| Phase | Phase 1 |
| Study type | Interventional |
| 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. |
| Who can participate | 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 cardiomyopath |
| Ages | 18 Years to 65 Years |
| Sex | All |
| Lead sponsor | Rocket Pharmaceuticals Inc. |
| Locations | San Diego, California, United States; Rochester, Minnesota, United States; Charleston, South Carolina, United States |
| Start date | 2026-06 |
| NCT ID | NCT07137338 |
| Official listing | https://clinicaltrials.gov/study/NCT07137338 |