Gene therapy breakthrough targets inherited heart disease
NCT ID NCT05836259
First seen Mar 24, 2026 · Last updated May 13, 2026 · Updated 4 times
Summary
This study tests a new gene therapy called TN-201 for people with a genetic form of hypertrophic cardiomyopathy (HCM), a condition where the heart muscle thickens and makes it harder to pump blood. The therapy uses a harmless virus to deliver a working copy of the MYBPC3 gene directly to heart cells. About 30 adults with symptoms will receive a single dose to see if it is safe and well-tolerated, with follow-up for one year.
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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: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Brigham and Women's Hospital
WITHDRAWNBoston, Massachusetts, 02115, United States
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Cleveland Clinic
RECRUITINGCleveland, Ohio, 44195, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Emory University
WITHDRAWNAtlanta, Georgia, 30322, United States
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Hospital of the University of Pennsylvania
WITHDRAWNPhiladelphia, Pennsylvania, 19104, United States
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Houston Methodist Hospital
RECRUITINGHouston, Texas, 77030, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Mayo Clinic
RECRUITINGRochester, Minnesota, 55905, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Oregon Health & Science University
WITHDRAWNPortland, Oregon, 97239, United States
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The Christ Hospital Physicians - The Ohio Heart and Vascular Center
WITHDRAWNCincinnati, Ohio, 45219, United States
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UC San Diego Altman Clinical and Translational Research Institute - Center for Clinical Research
RECRUITINGLa Jolla, California, 92093, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of California San Francisco
RECRUITINGSan Francisco, California, 94117, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
Explore the condition pages connected to this study.