Fatty acid oxidation disorder – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Fri, 12 Jun 2026 10:32:20 +0000 en-US hourly 1 https://wordpress.org/?v=7.0 https://clinicaltrials.eu/wp-content/uploads/2024/12/cropped-EU_icon-32x32.png Fatty acid oxidation disorder – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 Study on the Effects of Triheptanoin and Medium-chain Triglycerides in Children with Long-chain Fatty Acid Oxidation Disorders https://clinicaltrials.eu/trial/study-on-the-effects-of-triheptanoin-and-medium-chain-triglycerides-in-children-with-long-chain-fatty-acid-oxidation-disorders/ Wed, 29 Apr 2026 14:29:01 +0000 https://clinicaltrials.eu/trial/study-on-the-effects-of-triheptanoin-and-medium-chain-triglycerides-in-children-with-long-chain-fatty-acid-oxidation-disorders/ This clinical trial is focused on studying a group of rare genetic conditions known as Long-chain Fatty Acid Oxidation Disorders (LC-FAOD). These disorders affect the body’s ability to break down certain fats into energy, especially during periods without food. The study will compare the effects of a treatment called Triheptanoin, also known by its code name UX007 and marketed as DOJOLVI®, with another treatment involving Even-chain, Medium-chain Triglycerides (MCT). Triheptanoin is an oral liquid that provides an alternative energy source for patients with LC-FAOD.

The purpose of the study is to evaluate how Triheptanoin affects the frequency of major clinical events, such as episodes of low blood sugar or muscle breakdown, in children with LC-FAOD. Participants in the study will be randomly assigned to receive either Triheptanoin or MCT. The study will be conducted in a double-blind manner, meaning neither the participants nor the researchers will know which treatment is being given to each participant. This helps ensure that the results are not biased.

The study will last for up to 24 months, during which participants will take the assigned treatment orally. Throughout the study, participants will be monitored for any changes in their condition, including the occurrence of major clinical events. The study aims to provide valuable information on the effectiveness of Triheptanoin in managing LC-FAOD and improving the quality of life for affected individuals.

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