Could a common drug ease rare childhood epilepsy?
NCT ID NCT04937062
First seen Nov 01, 2025 · Last updated May 22, 2026 · Updated 24 times
Summary
This study tests whether a medicine called phenylbutyrate is safe and tolerable for 50 children with genetic conditions that cause seizures and developmental delays. The goal is to see if the drug can help the body's remaining proteins work better. This is an early-phase trial focused on safety, not a cure.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Locations
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Children's Hospital Colorado
Aurora, Colorado, 80045, United States
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Weill Cornell Medicine
New York, New York, 10065, United States
Conditions
Explore the condition pages connected to this study.