Experimental drug aims to help rare metabolic disorder patients handle fasting
NCT ID NCT06069375
Summary
This study is testing if a medication called sodium phenylbutyrate can help people with a rare genetic disorder called MCAD deficiency. The main goal is to see if the drug allows patients to safely fast for longer periods without developing low blood sugar. The trial involves 24 participants aged 10 and older and includes monitored fasting tests before and after taking the medication for four weeks.
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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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