Gene therapy breakthrough? first human test of TN-201 for heart muscle disease begins
NCT ID NCT05836259
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tests a new gene therapy called TN-201 in 30 adults with hypertrophic cardiomyopathy caused by a specific genetic mutation (MYBPC3). The therapy is given as a one-time IV infusion and aims to correct the underlying genetic defect. The main goals are to check safety and find the right dose, while also looking at how it affects heart function and symptoms.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
TN-201 (a gene therapy given as a one-time IV infusion)
What this could lead to
If successful, this could lead to a one-time treatment that addresses the root genetic cause of hypertrophic cardiomyopathy, potentially reducing symptoms and improving heart function.
What could go wrong
This is a very early (first-in-human) trial with only 30 participants, so safety and effectiveness are not yet known. Gene therapies can have serious side effects, and it may not work for everyone.
Disclaimer
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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.
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: •••••@•••••
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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: •••••@•••••