Brain and blood gene therapy tested for rare neurological disease
NCT ID NCT07180355
Summary
This is the first human study testing SGT-212, an experimental gene therapy for Friedreich's ataxia, a rare inherited neurological condition. The trial will enroll 10 participants to determine safe dosing levels and monitor side effects when the therapy is delivered both into a specific brain region and through an IV. Participants will be followed for approximately 5 years to assess long-term safety.
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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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