Gene therapy breakthrough offers hope for rare heart disease
NCT ID NCT06092034
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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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Boston Children's Hospital
RECRUITINGBoston, Massachusetts, 02115, United States
Contact Email: •••••@•••••
Contact
Contact Email: •••••@•••••
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Children's Hospital of Philadelphia
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
Contact Email: •••••@•••••
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German Heart Center Munich
RECRUITINGMunich, Germany
Contact Email: •••••@•••••
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Great Ormund Street Hospital & UCL Institute of Cardiovascular Science
RECRUITINGLondon, United Kingdom
Contact Email: •••••@•••••
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Meyer Childrens Hospital
RECRUITINGFlorence, Italy
Contact Email: •••••@•••••
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University of California, San Diego
RECRUITINGLa Jolla, California, 92037, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••