Experimental drug targets rare brain acid disorder in first human test
NCT ID NCT07125066
First seen Jun 24, 2026 · Last updated Jun 24, 2026
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 harmful acid buildup in the body. The goal is to see if the drug is safe and can reduce acid levels, seizures, and the need for breathing support. Because this is a very early, one-person trial, the results will be very limited.
Disclaimer
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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 of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224, 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
Sodium phenylbutyrate (also known as Olpruva)
What this could lead to
If it works, this could point toward a treatment for combined D,L-2-hydroxyglutaric aciduria by lowering harmful acid levels and reducing seizures.
What could go wrong
This is a very early, single-patient study, so results may not apply to others. The drug may cause side effects or not improve symptoms.
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.