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 Transgene (RP-A501; AAV9.LAMP2B) in Male Patients With Danon Disease
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.
• Documentation of a pathogenic or likely pathogenic variant of the LAMP2 gene.
• Male.
• Age ≥8 years.
• Evidence of left ventricular hypertrophy with preserved systolic function phenotype as defined by each of the following:
‣ Abnormal thickening of Left ventricular wall,
⁃ Left ventricular ejection fraction (LVEF) ≥ 50%.
• New York Heart Association (NYHA) Class II to III.
• High sensitivity Troponin I (hsTnI) ≥20% above the upper limit of normal (ULN)
• Ability to comply with study procedures including investigational therapy and follow-up evaluations.