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

Study 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.

Conditions

Danon Disease

Interventions / Drugs

RP-A501

View on ClinicalTrials.gov ↗

Sponsor

RCKT

Rocket Pharmaceuticals

Gene Therapy

C51/100

Study Details

Enrollment14 participants

Primary Completion—

Lead SponsorRocket Pharmaceuticals Inc.

Data Retrieved2026-04-11 04:25:37.504947+00:00

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