Gene therapy offers hope for kids with rare brain disease
NCT ID NCT02716246
First seen Nov 06, 2025 · Last updated May 16, 2026 · Updated 28 times
Summary
This study tests a one-time gene therapy (UX111) for children with Sanfilippo syndrome type A, a rare genetic disorder that damages the brain. The treatment aims to reduce harmful substances in the brain and slow down the disease. About 36 children will take part in this advanced-stage trial.
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This is a summary of
the original study
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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: •••••@•••••
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Contact
Phone: •••-•••-•••• 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
Contact Email: •••••@•••••
Contact Email: •••••@•••••
Contact
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Nationwide Children's Hospital
COMPLETEDColumbus, Ohio, 43205, United States
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Vall d'Hebron Barcelona Hospital Campus
RECRUITINGBarcelona, 08035, Spain
Contact Email: •••••@•••••
Contact
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Women's and Children's Hospital
COMPLETEDNorth Adelaide, South Australia, 5006, Australia
Conditions
Explore the condition pages connected to this study.