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Phase 2 Study of RP-A501 Gene Therapy for Male Patients Aged 8 and Older With Danon Disease Evaluating Safety and Effectiveness of a Single Intravenous Dose of AAV9.LAMP2B
Led by Rocket Pharmaceuticals Inc. · Updated on 2026-05-04
14
Participants Needed
6
Research Sites
208 weeks
Total Duration
AI-Summary
What this Trial Is About
Researchers are studying the safety and effectiveness of RP-A501, a gene therapy using a modified virus to deliver the LAMP2B gene, in male patients 8 years and older with Danon Disease. This single-arm Phase 2 trial focuses on males who have a genetic variant of the LAMP2 gene and heart changes related to the disease. The goal is to understand how well this gene therapy works and how safe it is for this group of patients. Participants will receive one intravenous infusion of RP-A501, a gene therapy product made of a modified adeno-associated virus containing the human LAMP2B gene. This single dose is given during the treatment period, and patients are monitored over time to assess the therapys effects. The study does not include a comparison group and focuses solely on this treatment. During the study, participants will be evaluated at regular intervals up to 60 months after infusion. Assessments include measuring LAMP2 protein expression in heart tissue, heart size and function via left ventricular mass index, and biomarkers indicating heart injury such as high sensitivity Troponin I and NT-proBNP. The study also tracks event-free survival and safety outcomes. Patients must be able to comply with study procedures and attend follow-up visits throughout the trial.
CONDITIONS
Brief Title
A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease
Research Team
C
Clinical Information
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