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First received: August 10, 2023

Clinical Trial: Study of Triheptanoin for the Prevention of Hypoglycemia in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)

Study Status: Completed

Recruit Status: RECRUITING

Condition: Medium-chain Acyl-CoA Dehydrogenase Deficiency

Study Type: INTERVENTIONAL


Official Title: A Phase II, Escalating Dose, Open Label Study to Evaluate the Safety of Triheptanoin for the Prevention of Hypoglycemia in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)

Brief Summary: This is a medical research study to test a medication in adult patients with a disease called medium-chain acyl-CoA dehydrogenase deficiency (MCADD).The medication is triheptanoin, which is currently FDA approved for the treatment of Long-Chain Fatty Acid Oxidation Disorders.Previous research suggests that triheptanoin may also be effective in the treatment MCADD.This study will investigate the safety and efficacy (how well it works) of triheptanoin in patients with MCADD.

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