Condition: Danon Disease · Sponsor: Rocket Pharmaceuticals Inc.
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.
This description comes directly from the study's public registry record.
Clinical Information · 646-627-0033 · clinicaltrials@rocketpharma.com
Always discuss trial participation with your own doctor first.
| University of California, San Diego | La Jolla, California, United States | Recruiting |
| Boston Children's Hospital | Boston, Massachusetts, United States | Recruiting |
| Children's Hospital of Philadelphia | Philadelphia, Pennsylvania, United States | Recruiting |
| German Heart Center Munich | Munich, Germany | Recruiting |
| Meyer Childrens Hospital | Florence, Italy | Recruiting |
| Great Ormund Street Hospital & UCL Institute of Cardiovascular Science | London, United Kingdom | Recruiting |
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Source record: clinicaltrials.gov/study/NCT06092034