RecruitingDanon Disease
A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease
Eligible age
8+ yrs
Accepts
Men
Locations
3 states
Healthy volunteers
No
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About this study
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.
Sponsor: Rocket Pharmaceuticals Inc.
Where it's recruiting
Source: ClinicalTrials.gov · NCT06092034 · last updated 2026-05-04