Can a weekly brain infusion slow sanfilippo syndrome?
NCT ID NCT07733856
First seen Jul 29, 2026 · Last updated Sep 21, 2026 · Updated 2 times
Summary
This early-access program tests whether a weekly infusion of tralesinidase alfa directly into the brain's fluid spaces is safe and tolerable for children with Sanfilippo syndrome type B, a rare genetic disorder that causes severe neurological decline. The study enrolls about 10 children, including those as young as 12 months, and treats them for up to a year. The goal is to provide early access to a potential therapy while gathering safety data.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- tralesinidase alfa given directly into the brain's fluid spaces
- What this could lead to
- If it works, this could provide a treatment option for children with Sanfilippo syndrome type B, a rare and devastating genetic disease that currently has no approved therapy.
- What could go wrong
- This is a small, early-access program, not a controlled trial, so results may be limited. The treatment requires weekly infusions into the brain, which carries risks like infection or bleeding.
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Study facts
What this study's own registry entry says, in plain language.
- 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
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12 months and older
- Sex
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Anyone
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: Has a diagnosis of MPS IIIB confirmed by deficient NAGLU enzyme activity analyzed at the laboratory during Screening. Is ≥12 and ≤60 months of age with a BSID-III-C raw score ≥70. or Is \>60 months of age regardless of cognitive level. Has provided written informed consent from a parent or legal guardian Has been determined by the investigator, after consultation with the participant (if applicable) and their family/caregiver, that the known risks of TA ICV infusion are outweighed by its potential benefits. If female and of childbearing potential, agrees to follow contraception guidelines from screening until 30 days after the last dose of TA. Has the ability to comply with program requirements, in the opinion of the investigator. Exclusion Criteria: Has contraindications for neurosurgery (e.g., congenital heart disease, severe respiratory impairment, or clotting abnormalities). Has contraindications for MRI scans, if the investigator deems an MRI and not a CT scan is required for ICV access device placement or follow-up post-placement. Has a history of poorly controlled seizure disorder. Is prone to complications from ICV infusion, including participants with hydrocephalus or ventricular shunts. Has received any investigational medication within 30 days prior to the Baseline Visit or is scheduled to receive any investigational drug during the course of the program. Has a medical condition or extenuating circumstance that, in the opinion of the investigator, might compromise the participant's ability to comply with program requirements, the participant's well-being or safety, or the interpretability of the participant's clinical data.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
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