Clinical Trial: A Multi-Center, Open Label Gene Therapy Study of RP-A501 in Male Patients With Danon Disease
Study Status: Completed
Recruit Status: RECRUITING
Condition: Danon Disease
Study Type: INTERVENTIONAL
Official Title: Gene Therapy for Danon Disease: A Phase 2 Study Evaluating the Efficacy and Safety of Intravenously Administered Adeno-Associated Virus Serotype 9 (rAAV9) Vector Containing the Human LAMP2 Isoform B T
Brief 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.
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