Experimental gene therapy offers hope for rare heart disease
NCT ID NCT06092034
First seen Oct 31, 2025 · Last updated May 15, 2026 · Updated 23 times
Summary
This study tests a gene therapy called RP-A501 in 14 males aged 8 and older with Danon disease, a rare genetic condition that weakens the heart. The therapy 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 heart changes 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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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: •••••@•••••
Conditions
Explore the condition pages connected to this study.