Could a urea cycle drug help kids with rare epilepsy?
NCT ID NCT04937062
First seen Nov 01, 2025 · Last updated Jun 22, 2026 · Updated 28 times
Summary
This early-phase trial is testing whether glycerol phenylbutyrate (Ravicti), a drug already approved for a different condition, is safe and tolerable for children with genetic disorders like STXBP1 and SLC6A1 that cause seizures and developmental delays. The study will enroll 50 children and monitor for side effects and how well they take the medication. It aims to see if the drug can help the body's remaining proteins work better, but it is not yet testing if it actually improves symptoms.
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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.
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
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
Glycerol phenylbutyrate (Ravicti)
What this could lead to
If successful, this could point toward a treatment that helps children with certain genetic epilepsies and developmental delays by improving the function of their remaining proteins.
What could go wrong
This is a very early, small safety study (50 participants) with no guarantee of benefit. The drug may cause side effects or fail to improve seizures or development.
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.