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

PHASE1RecruitingINTERVENTIONAL
Enrollment

9

Participants

Timeline

Start Date

August 29, 2023

Primary Completion Date

September 30, 2025

Study Completion Date

September 30, 2026

Conditions
PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM)
Interventions
GENETIC

RP-A601

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)

Trial Locations (3)

19104

RECRUITING

Children's Hospital of Philadelphia, Philadelphia

27710

RECRUITING

Duke University, Durham

92037

RECRUITING

University of California, San Diego, La Jolla

All Listed Sponsors
lead

Rocket Pharmaceuticals Inc.

INDUSTRY