NCT06092034

A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease

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

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 14 (estimated)
• Sponsor: Rocket Pharmaceuticals Inc. · Industry
• Sex: Male
• Age: 8 Years
• Healthy volunteers: Not accepted

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.

Detailed description

The study is a single arm Phase 2 clinical trial to characterize the safety and efficacy of RP-A501, a recombinant adeno-associated serotype 9 (rAAV9 capsid containing the human lysosome-associated membrane protein 2 isoform B (LAMP2B) transgene) in male patients with Danon Disease. Male subjects ≥8 years of age will receive a single intravenous infusion of RP-A501.

Conditions

• Danon Disease

Interventions

Type	Name	Description
GENETIC	RP-A501	RP-A501 is a gene therapy product consisting of a rAAV9 capsid containing the human LAMP2B transgene which will be administered as a single IV infusion.

Timeline

• Start date: 2023-09-05
• Primary completion: 2028-04-01
• Completion: 2032-04-01
• First posted: 2023-10-23
• Last updated: 2025-10-07

Locations

6 sites across 4 countries: United States, Germany, Italy, United Kingdom

Regulatory

• FDA-regulated drug study