Could a urea cycle drug help MCAD patients fast longer?
NCT ID NCT06069375
First seen Jun 27, 2026 · Last updated Jul 07, 2026 · Updated 2 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 affects fat breakdown). About 24 participants aged 10 and older will take the drug for 4 weeks. Researchers will check for side effects and measure how long participants can safely fast without developing low blood sugar.
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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
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UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224, United States
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