Gene therapy for sanfilippo a: does it last?
NCT ID NCT04360265
First seen Jun 26, 2026 · Last updated Jul 17, 2026 · Updated 2 times
Summary
This study follows 41 children with Sanfilippo A (MPS IIIA) who previously received UX111 gene therapy in earlier trials. Researchers will monitor safety and how well the therapy controls the disease over time, using tests like the Bayley cognitive scale. No new gene therapy is given in this follow-up; some children may receive immune-suppressing drugs if needed.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- UX111 (gene therapy)
- What this could lead to
- If successful, this could show that a single gene therapy treatment safely controls Sanfilippo A symptoms for years, offering a long-term management option.
- What could go wrong
- This is a follow-up study with no new treatment given, so it only measures past effects. The number of participants is small (41), and results may not apply to all patients. Some children may need additional immune-suppressing drugs.
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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Locations
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Hospital Clínico Universitario de Santiago
Santiago de Compostela, Spain
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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
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Vall d'Hebron Barcelona Campus
Barcelona, 08035, Spain
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Women's and Children's Hospital
North Adelaide, South Australia, Australia
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