Gene therapy trial hopes to slow rare neurological disease
NCT ID NCT07180355
First seen Nov 01, 2025 · Last updated May 24, 2026 · Updated 30 times
Summary
This early-stage trial tests a new gene therapy called SGT-212 in 10 people with Friedreich's ataxia, a rare genetic disorder that affects movement and coordination. The treatment is given both as an injection into the brain and through an IV. The main goal is to see if it is safe and tolerable over about 5 years.
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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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The Children's Hospital of Philadelphia (CHOP)
NOT_YET_RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
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The Ohio State University
RECRUITINGColumbus, Ohio, 43210, United States
Contact Email: •••••@•••••
Contact
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The University of California, Los Angeles (UCLA)
NOT_YET_RECRUITINGLos Angeles, California, 90095, United States
Contact Email: •••••@•••••
Contact
Conditions
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