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

NCT06092034 · Status: RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 14

Last updated 2026-05-04

No results posted yet for this study

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.

Conditions

  • Danon Disease

Interventions

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.

Sponsors & Collaborators

  • Rocket Pharmaceuticals Inc.

    lead INDUSTRY

Principal Investigators

  • Joseph Rossano, MD · Children's Hospital of Philadelphia

  • Barry Greenberg, MD · University of California, San Diego

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
8 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-09-05
Primary Completion
2028-04-30
Completion
2032-04-30
FDA Drug
Yes

Countries

  • United States
  • Germany
  • Italy
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06092034 on ClinicalTrials.gov