Trial tests if existing drug can help rare metabolic disorder
NCT ID NCT06069375
Summary
This study is testing if a medication called sodium phenylbutyrate (ACER-001), already approved for a different condition, can help people aged 10 and older with a specific form of MCAD deficiency. Researchers want to see if taking the drug for 4 weeks allows patients to fast longer without developing dangerously low blood sugar. The trial involves short hospital stays for fasting tests and is currently suspended.
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Contacts and locations
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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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