A Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM)
Condition(s) studied
Investigational drug(s) / intervention(s)
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)
Study 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
Primary outcome measure(s)
- Evaluation of safety associated with RP-A601 — 12 months post-infusion
Incidence of treatment emergent adverse events (TEAEs), incidence of Serious Adverse Events (SAEs), and identification of Dose Limiting Toxicities (DLTs)
Trial sites (3)
| Facility | City | Region | Status |
|---|---|---|---|
| University of California, San Diego | La Jolla | California | Recruiting |
| Duke University | Durham | North Carolina | Recruiting |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | Withdrawn |
More Rocket Pharmaceuticals Inc. trials in the USA
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT05885412 on ClinicalTrials.gov ↗ ← All trials in the USA