New study tracks rare brain disease in children to pave way for future treatments
NCT ID NCT05825131
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study follows 30 children and young adults with Sanfilippo syndrome type C, a rare genetic disorder that causes severe brain damage. Researchers will measure changes in development and thinking skills over time using standard tests. The goal is to better understand how the disease progresses, which can help design future clinical trials for potential treatments.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- What this could lead to
- If successful, this study will provide crucial data on how Sanfilippo syndrome type C progresses, which could help design future treatments.
- What could go wrong
- This is an observational study, not a treatment trial. It will not directly improve symptoms or slow the disease. Results may take years to impact care.
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
Locations
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Hospices Civils De Lyon
RECRUITINGBron, 690007539, France
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The University of Texas Southwestern Medical Center
RECRUITINGDallas, Texas, 75390, United States
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