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Gene Therapy for Male Patients With Danon Disease (DD) Using RP-A501; AAV9.LAMP2B

NCT03882437 · Status: UNKNOWN · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 7

Last updated 2023-03-03

No results posted yet for this study

Summary

This is a non-randomized open-label Phase 1 study to evaluate the safety and toxicity of gene therapy using a recombinant adeno-associated virus serotype 9 (AAV9) containing the human lysosome-associated membrane protein 2 isoform B (LAMP2B) transgene (investigational product (IP), RP-A501) in male patients with Danon Disease (DD).

Conditions

  • Danon Disease

Interventions

BIOLOGICAL

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

  • Matthew Taylor, MD, PhD · University of Colorado, Anschutz Medical Ctr

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

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
8 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-04-17
Primary Completion
2025-03-24
Completion
2025-03-24
FDA Drug
Yes

Countries

  • United States

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 NCT03882437 on ClinicalTrials.gov