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A Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM)
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)
Who can join
Ages 18 and older · All sexes
Full eligibility criteria
Key Inclusion Criteria: 1. Male or female ≥18 years at the time of signing the informed consent 2. Capable and willing to provide signed informed consent 3. Clinical diagnosis of ACM as defined by the 2010 revised Task Force Criteria (TFC) 4. Documentation of a pathogenic or likely pathogenic truncating variant in PKP2 5. History of Implantable Cardioverter-Defibrillator (ICD) implantation ≥6 months prior to enrollment 6. PVC frequency ≥500 per 24 hours by ambulatory rhythm monitoring 7. Left ventricular ejection fraction by echocardiogram or CMR ≥50% Key Exclusion Criteria: 1. Anti-AAVrh.74 capsid neutralizing antibody titer of \>1:40 2. Cardiomyopathy related to a genetic etiology other than PKP2 truncating variant 3. Previous participation in a study of gene transfer or gene editing 4. Severe Right Ventricular (RV) dysfunction 5. New York Heart Association (NYHA) Class IV heart failure.
About the study
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.
What is being tested
- RP-A601 (genetic)
Sponsor: Rocket Pharmaceuticals Inc. · Participants: 9 · Started: Aug 29, 2023
Contact the study team
- Clinical Information · Phone: 646-627-0033
Official record on ClinicalTrials.gov — NCT05885412
Locations in the U.S.
| California | University of California, San Diego, La Jolla |
| North Carolina | Duke University, Durham |
Conditions
From ClinicalTrials.gov, data retrieved Oct 2, 2026. Each study sets its own eligibility; the study team decides who can join.