Could a liver drug help people with MCADD fast safely?
NCT ID NCT06069375
First seen Nov 01, 2025 · Last updated May 23, 2026 · Updated 31 times
Summary
This study tests whether sodium phenylbutyrate, a drug already approved for another condition, can help people with MCAD deficiency—a genetic disorder that makes it hard to break down fats for energy. About 24 participants aged 10 and older will take the drug for 4 weeks. Researchers will check for side effects and measure how long they can safely go without eating before low blood sugar occurs.
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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.
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Locations
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UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224, United States
Conditions
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