Can a weekly brain infusion slow sanfilippo syndrome?
NCT ID NCT07733856
First seen Jul 29, 2026 · Last updated Jul 30, 2026 · Updated 1 time
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.
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.