Status: Recruiting
A Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM)
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.
Participants needed: 9
Trial details
Phase: Phase 1Age: 18+Biological sex: AllType: InterventionalSponsor: Rocket Pharmaceuticals Inc.Updated: Apr 16, 2026Locations: 3
Eligibility criteria
Male or female ≥18 years at the time of signing the informed consent [+6]
Anti-AAVrh.74 capsid neutralizing antibody titer of >1:40 [+4]