Can a direct brain infusion slow sanfilippo syndrome?
NCT ID NCT06567769
First seen Sep 08, 2026 · Last updated Sep 09, 2026 · Updated 1 time
Summary
This trial tests an experimental enzyme replacement called GC1130A in children with Sanfilippo syndrome type A, a rare genetic disease that damages the brain. The enzyme is given through a small device placed under the scalp that delivers it directly into the fluid around the brain. Researchers want to see if the treatment is safe and whether it can reduce the buildup of harmful substances in the brain. The study includes children aged 1 to 18 years with a confirmed diagnosis.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- GC1130A, a recombinant human heparan N-sulfatase enzyme, delivered directly into the brain via an implanted device
- What this could lead to
- If it works, this could slow or halt the nerve damage caused by Sanfilippo syndrome type A, potentially preserving skills and extending life.
- What could go wrong
- This is an early, small trial focused on safety. The enzyme may not reach all brain areas or may cause side effects from the surgery or infusion.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
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About 9 people
The number the study aims to enrol. It can still change while the study runs.
- Started
-
Nov 2024
- Expected to finish
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Jun 2027
An estimate. End dates often move.
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
-
12 months to 18 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Participants with documented MPS IIIA diagnosis * Participants aged ≥ 12 months and ≤ 18 years Exclusion Criteria: * Participants with significant non-MPS IIIA related central nervous system impairment * Participants with previous complication from intraventricular drug administration * Participants with contraindications for MRI scans and for neurosurgery * Participants that received treatment with any investigational drug or a device intended as a treatment for MPS IIIA within 30 days or 5 half-lives prior to the study * Participants that received a hematopoietic stem cell or bone marrow transplant or received gene therapy
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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
Locations
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Ajou University Medical Center
Suwon, Gyeongi-do, South Korea
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Samsung Medical Center
Seoul, South Korea
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UCSF Benioff Children's Hospital
Oakland, California, 94609, United States
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University of Minnesota
Minneapolis, Minnesota, 55455, United States