Could a common drug ease rare childhood epilepsy?
NCT ID NCT04937062
First seen Nov 01, 2025 · Last updated May 11, 2026 · Updated 23 times
Summary
This study tests whether a medicine called phenylbutyrate is safe and tolerable for children with certain genetic conditions that cause seizures and developmental delays, such as STXBP1 and SLC6A1 disorders. The drug aims to help the body's remaining proteins work better. Researchers will monitor side effects and how well children take the medication over time.
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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.