A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease
Recruiting now · Phase 2
Conditions studied: Danon Disease
In brief
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.
Key facts
- Study ID
- NCT06092034
- Run by
- Rocket Pharmaceuticals Inc.
- People needed
- 14
- Starts
- 2023-09-05
- Expected to finish
- 2032-04-01
- Last updated by the study team
- 2026-05-04
Who can join
Age: 8 and older. Sex: male. Healthy volunteers: not accepted.
Where it is running
- University of California, San Diego — La Jolla, California, United States (enrolling)
- Boston Children's Hospital — Boston, Massachusetts, United States (enrolling)
- Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States (enrolling)
- German Heart Center Munich — Munich, Germany (enrolling)
- Meyer Childrens Hospital — Florence, Italy (enrolling)
- Great Ormund Street Hospital & UCL Institute of Cardiovascular Science — London, United Kingdom (enrolling)
Full record on ClinicalTrials.gov
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