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First received: October 11, 2023

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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