Phase 1Recruiting
A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 Dilated Cardiomyopathy
A Phase 1 Dose Escalation Trial Evaluating an Intravenously Administered Recombinant Adeno-associated Virus Serotype rh.74 (AAVrh.74) Vector Containing the Human BCL2-associated Athanogene 3 (BAG3) Gene Coding Sequence (RP-A701) in Subjects With Dilated Cardiomyopathy Arising From Pathogenic BAG3 Variants (BAG3-DCM)
- Sponsor
- Rocket Pharmaceuticals Inc. (Industry)
- Enrolment
- 8 planned
- Conditions
- Dilated Cardiomyopathy (DCM)
- Interventions
- 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)
- Ages
- adults, older adults
- Registry
- NCT07137338
Full record on ClinicalTrials.gov
From the registry
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.
Primary outcomes
- Incidence of Treatment-emergent Adverse Events (TEAE)
- Incidence of Treatment-emergent Serious Adverse Events (SAE).
- Incidence of Dose Limiting Toxicities (DLT).
History
Primary completion expected
Last registry update
Started
Registered