Could a urea cycle drug help kids with rare epilepsy?
NCT ID NCT04937062
First seen Jun 27, 2026 · Last updated Jun 27, 2026
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.
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.
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
-
Children's Hospital Colorado
Aurora, Colorado, 80045, United States
-
Weill Cornell Medicine
New York, New York, 10065, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- New drug LP352 aims to control seizures in severe epilepsy over the long term
- New epilepsy drug shows promise in long-term safety trial
- New hope for kids with rare epilepsy: drug trial targets seizure control
- Gene therapy trial for rare childhood epilepsy halted after just one patient
- New hope for Hard-to-Treat seizures: phase 3 trial launches