Experimental drug targets rare brain disease in first human test
NCT ID NCT07125066
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 2 times
Summary
This study tests a drug called ACER-001 (sodium phenylbutyrate) in a single patient with combined D,L-2-hydroxyglutaric aciduria, a rare genetic disorder that causes brain damage and seizures. The goal is to see if the drug is safe and can lower harmful acid levels in the body. Researchers will also watch for changes in seizures and breathing support needs.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- Sodium phenylbutyrate (also called Olpruva)
- What this could lead to
- If it works, this could point toward a treatment for combined D,L-2-hydroxyglutaric aciduria, a rare metabolic disorder.
- What could go wrong
- This is a very early phase 1 trial with only one patient, so results may not apply to others. The drug may cause side effects or fail to improve symptoms.
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 of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224, United States