Could a urea cycle drug help MCAD patients fast longer?

NCT ID NCT06069375

First seen Jun 27, 2026 ยท Last updated Jun 27, 2026

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.

medium chain acyl-CoA dehydrogenase deficiency

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • UPMC Children's Hospital of Pittsburgh

    Pittsburgh, Pennsylvania, 15224, United States