One-Time infusion aims to fix faulty heart gene
NCT ID NCT06092034
Summary
This study is testing a one-time gene therapy infusion for males aged 8 and older with Danon Disease, a rare genetic condition that damages the heart. The goal is to see if delivering a corrected gene can improve heart health and slow disease progression. Researchers will measure changes in heart structure and function, as well as track patient survival and hospitalizations.
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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.