Gene therapy hope for rare nerve disease enters first human tests
NCT ID NCT07180355
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early-phase trial is testing a gene therapy called SGT-212 for Friedreich's ataxia, a rare genetic disease that damages the nervous system. Ten adults with the condition will receive the therapy through an injection into the brain and a vein. The main goal is to see if it is safe and tolerable over about 5 years.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- SGT-212 (a gene therapy using a harmless virus to deliver a working copy of the frataxin gene)
- What this could lead to
- If it works, this could point toward a treatment that slows or stops Friedreich's ataxia progression.
- What could go wrong
- This is a very early, first-in-human trial with only 10 people. It is designed mainly to check safety, not effectiveness. Gene therapies can have unexpected side effects, and it may not work at all.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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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: •••••@•••••
Locations
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The Children's Hospital of Philadelphia (CHOP)
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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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