A Phase 1 Dose Escalation Trial Evaluating an Intravenously Administered Recombinant Adeno-Associated Virus Serotype rh.74 (AAVrh.74) Vector Containing the Human Plakophilin-2a (PKP2a) Coding Sequence (RP-A601; AAVrh.74-PKP2a) in Subjects With Arrhythmogenic Cardiomyopathy Arising From Pathogenic PKP2 Variants (PKP2-ACM)
Status: Recruiting
Location: See all (3) locations...
Intervention Type: Genetic
Study Type: Interventional
Study Phase: Phase 1
SUMMARY
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.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Healthy Volunteers: f
View:
• Male or female ≥18 years at the time of signing the informed consent
• Capable and willing to provide signed informed consent
• Clinical diagnosis of ACM as defined by the 2010 revised Task Force Criteria (TFC)
• Documentation of a pathogenic or likely pathogenic truncating variant in PKP2
• History of Implantable Cardioverter-Defibrillator (ICD) implantation ≥6 months prior to enrollment
• PVC frequency ≥500 per 24 hours by ambulatory rhythm monitoring
• Left ventricular ejection fraction by echocardiogram or CMR ≥50%
Locations
United States
California
University of California, San Diego
RECRUITING
La Jolla
North Carolina
Duke University
RECRUITING
Durham
Pennsylvania
Children's Hospital of Philadelphia
RECRUITING
Philadelphia
Contact Information
Primary
Clinical Information
clinicaltrials@rocketpharma.com
646-627-0033
Time Frame
Start Date: 2023-08-29
Estimated Completion Date: 2026-09
Participants
Target number of participants: 9
Treatments
Experimental: RP-A601
Single ascending dose of RP-A601 in 2 consecutive cohorts
Related Therapeutic Areas
Sponsors
Leads: Rocket Pharmaceuticals Inc.