Gene therapy breakthrough offers hope for rare heart disease

NCT ID NCT06092034

Recruiting now Disease control Sponsor: Rocket Pharmaceuticals Inc. Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests a gene therapy called RP-A501 in 14 males with Danon disease, a rare genetic condition that causes heart problems. The treatment uses a harmless virus to deliver a working copy of the LAMP2 gene, aiming to improve heart muscle function and reduce thickening. Participants will be monitored for safety and effectiveness over time.

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Conditions

The condition(s) this trial relates to.

cardiomyopathy Danon disease hypertrophic cardiomyopathy Hypertrophy, Left Ventricular lysosomal storage disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

DANON DISEASE

Contacts and locations

Locations

Boston Children's Hospital
RECRUITING

Boston, Massachusetts, 02115, United States
Children's Hospital of Philadelphia
RECRUITING

Philadelphia, Pennsylvania, 19104, United States
German Heart Center Munich
RECRUITING

Munich, Germany
Great Ormund Street Hospital & UCL Institute of Cardiovascular Science
RECRUITING

London, United Kingdom
Meyer Childrens Hospital
RECRUITING

Florence, Italy
University of California, San Diego
RECRUITING

La Jolla, California, 92037, United States

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