A Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM)

Part of paid clinical trials in La Jolla, California.

Sponsor
Rocket Pharmaceuticals Inc.
Study ID
NCT05885412
Phase
PHASE1
Status
Recruiting
Apply to this trial

Conditions

  • PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM)

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • RP-A601 — GENETIC
    RP-A601 is a recombinant viral vector composed of an AAV serotype rh.74 (AAVrh.74) capsid encapsulating the transgene, human plakophilin 2 (PKP2), transcript variant 2a (PKP2a)

Study Details

This Phase 1 dose escalation trial will assess the safety and preliminary efficacy of a single dose intravenous infusion of RP-A601 in high-risk adult patients with PKP2-ACM.

Key Dates

Start date
Aug 29, 2023
Status verified
Apr 2026
Primary completion
Sep 30, 2028
Completion
Sep 30, 2029

Study Design

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

Arms

  • Experimental: RP-A601
    Single ascending dose of RP-A601 in 2 consecutive cohorts

Primary Outcome Measure

Evaluation of safety associated with RP-A601 [ Time Frame: 12 months post-infusion ]

Central Contacts

Locations (3)

FacilityCityStateZIPSite coordinators
University of California, San DiegoLa JollaCalifornia92037
Barry Greenberg, MD
[email protected]
858-246-2987
Duke UniversityDurhamNorth Carolina27710
Meghan Pergola, MPH
[email protected]
James Daubert, MD (PRINCIPAL_INVESTIGATOR)
Children's Hospital of PhiladelphiaPhiladelphiaPennsylvania19104-

Find similar trials in La Jolla, CA

By research site
University of California, San Diego· La Jolla, CADuke University· Durham, NCChildren's Hospital of Philadelphia· Philadelphia, PA