Clinical Trial

A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease

Recruiting Phase 2
View on ClinicalTrials.gov →
Summary
This is a single arm Phase 2 trial to evaluate the efficacy and safety of RP-A501, a recombinant adeno-associated virus serotype 9 (AAV9) containing the human lysosome-associated membrane protein 2 isoform B (LAMP2B) transgene, in male patients with Danon Disease.
Protocol Amendment History 4 amendments
This ClinicalTrials.gov record has been amended 4 times since 2023-10-17; most recent amendment 2026-04-28.
Status change: Active, Not Recruiting → Recruiting 2025-10-01
Status change: Recruiting → Active, Not Recruiting 2024-09-23
Trial Details
NCT Number NCT06092034
Lead Sponsor Rocket Pharmaceuticals Inc.
Conditions Danon Disease
Enrollment 14 participants
Start Date 2023-09-05
Primary Completion 2028-04 (estimated)
Study Completion 2032-04 (estimated)
Updated on ClinicalTrials.gov 2026-05-04