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)
Rocket Pharmaceuticals Inc.
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
- Age range
- 18+ years
- Sex
- All
- Healthy volunteers
- No
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.7…
Interventions
- GeneticRP-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)
Locations (3)
- University of California, San DiegoLa Jolla, California
- Duke UniversityDurham, North Carolina
- Children's Hospital of PhiladelphiaPhiladelphia, Pennsylvania