Could a fatty acid drug tame a rare metabolic disease?
NCT ID NCT06340685
First seen Feb 16, 2026 · Last updated May 02, 2026 · Updated 7 times
Summary
This early-stage study tests triheptanoin, a drug already approved for other conditions, in 6 children with pyruvate dehydrogenase complex (PDC) deficiency. The goal is to see if it can improve metabolic markers, reduce seizures, and lower hospital visits. The study focuses on safety and how well the drug works, with no expectation of a cure.
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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.
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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Phone: •••-•••-•••• Email: •••••@•••••
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Locations
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UPMC Children's Hospital of Pittsburgh
RECRUITINGPittsburgh, Pennsylvania, 15224, United States
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Conditions
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