A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 Dilated Cardiomyopathy
Condition(s) studied
Investigational drug(s) / intervention(s)
RP-A701 is a recombinant viral vector composed of an AAV serotype rh.74 (AAVrh.74) capsid encapsulating the transgene, BCL2-associated Athanogene 3 (BAG3): One-time treatment with a single ascending dose
Study 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.
Eligibility
Primary outcome measure(s)
- Incidence of Treatment-emergent Adverse Events (TEAE) — Baseline up to End of Study (up to 24 months post-infusion)
Number of participants with Adverse Events following a single IV dose of RP-A701 - Incidence of Treatment-emergent Serious Adverse Events (SAE). — Baseline up to End of Study (up to 24 months post-infusion)
Number of participants with Serious Adverse Events (SAE) following a single IV dose of RP-A701 - Incidence of Dose Limiting Toxicities (DLT). — Baseline up to End of Study (up to 24 months post-infusion)
Number of participants with Dose Limiting Toxicities (DLT) following a single IV dose of RP-A701
Trial sites (3)
| Facility | City | Region | Status |
|---|---|---|---|
| University of California, San Diego | San Diego | California | Recruiting |
| Mayo Clinic | Rochester | Minnesota | Recruiting |
| Medical University of South Carolina | Charleston | South Carolina | Recruiting |
More Rocket Pharmaceuticals Inc. trials in the USA
Other trials for the same condition
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT07137338 on ClinicalTrials.gov ↗ ← All trials in the USA