One-Time gene therapy aims to halt fatal brain disease in children
NCT ID NCT02716246
First seen Jun 25, 2026 · Last updated Jul 17, 2026 · Updated 2 times
Summary
This trial tests a one-time gene therapy called UX111 for children with Sanfilippo A, a rare genetic disorder that causes severe brain damage. The therapy delivers a working copy of the missing gene to cells. Researchers will measure whether it reduces harmful substances in the brain and improves development. The study is currently recruiting 36 participants.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- UX111 (a gene therapy that delivers a working copy of the SGSH gene into cells)
- What this could lead to
- If it works, this could slow or stop the progression of Sanfilippo A, a severe genetic brain disease, potentially allowing children to live longer with better function.
- What could go wrong
- This is an early-to-mid-stage trial with only 36 participants. Gene therapy carries risks like immune reactions or the treatment not reaching enough cells. Lifelong monitoring is needed.
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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
Email: •••••@•••••
Locations
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Children's Hospital of Pittsburgh
COMPLETEDPittsburgh, Pennsylvania, 15224, United States
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Hospital Clínico Universitario de Santiago
RECRUITINGSantiago de Compostela, 15706, Spain
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Nationwide Children's Hospital
COMPLETEDColumbus, Ohio, 43205, United States
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Vall d'Hebron Barcelona Hospital Campus
RECRUITINGBarcelona, Barcelona, 08035, Spain
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Women's and Children's Hospital
COMPLETEDNorth Adelaide, South Australia, 5006, Australia
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