A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 Dilated Cardiomyopathy

Part of paid clinical trials in San Diego, California.

Sponsor
Rocket Pharmaceuticals Inc.
Study ID
NCT07137338
Phase
PHASE1
Status
Recruiting
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Conditions

  • Dilated Cardiomyopathy (DCM)

Eligibility Criteria

Sex
ALL
Age
18 Years - 65 Years
Healthy Volunteers
Not accepted

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) — GENETIC
    One-time treatment with a single ascending dose

Study Details

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.

Key Dates

Start date
Jun 30, 2026
Status verified
May 2026
Primary completion
Jun 30, 2029
Completion
Jun 30, 2029

Study Design

Enrollment
8 participants (estimated)
Allocation
NA
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: Single ascending dose of RP-A701 in up to 2 consecutive cohorts
    Participants will receive a single intravenous dose of RP-A701 on Day 0 and will be followed for up to two years

Primary Outcome Measure

Incidence of Treatment-emergent Adverse Events (TEAE) [ Time Frame: Baseline up to End of Study (up to 24 months post-infusion) ]

Central Contacts

Locations (3)

FacilityCityStateZIPSite coordinators
University of California, San DiegoSan DiegoCalifornia92037
Clinical Research Coordinator
[email protected]
858-246-5442
Barry Greenberg, MD (PRINCIPAL_INVESTIGATOR)
Mayo ClinicRochesterMinnesota55905
Clinical Research Coordinator
[email protected]
507-538-1500
John Giudicessi, MD, PhD (PRINCIPAL_INVESTIGATOR)
Medical University of South CarolinaCharlestonSouth Carolina29425
Clinical Res
[email protected]
843-792-2300
Daniel Judge, MD (PRINCIPAL_INVESTIGATOR)

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