New hope for kids with rare brain disease: drug targets toxic buildup
NCT ID NCT06181136
First seen Jun 25, 2026 · Last updated Jul 14, 2026 · Updated 3 times
Summary
This study tests a drug called DNL126 in 20 children with Sanfilippo syndrome type A, a rare genetic disorder that causes brain damage. The drug is given through a vein and aims to reduce harmful substances in the brain and body. The trial lasts about 6 months, with options to continue treatment for up to 4 years.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- DNL126 (a drug given intravenously)
- What this could lead to
- If successful, this could point toward a treatment that slows or stops the progression of Sanfilippo syndrome type A in children.
- What could go wrong
- This is an early-phase trial with only 20 participants, so results may not apply to all patients. The drug may not reduce symptoms or could cause side effects.
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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Baylor College of Medicine and Texas Children's Hospital
Houston, Texas, 77030, United States
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UCSF Benioff Children's Hospital Oakland
Oakland, California, 94609, United States
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University of Iowa Stead Family Children's Hospital
Iowa City, Iowa, 52242, United States
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University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27514, United States