At a glance
ClinicalIndex Comparison RecordStandardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
Phase 2 Study of Triheptanoin for Treatment of Long-Chain Fatty Acid Oxidation Disorders
In Brief
A Phase 2 clinical trial evaluating Triheptanoin for Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency and 3 related conditions. Completed, enrolled 32 participants across 2 sites.
Detailed Summary
Humans eat long-chain fat in their diet and use it for energy during exercise and during periods of fasting. Patients with long-chain fatty acid oxidation disorders cannot use dietary fat for energy. They sometimes develop muscle breakdown, and severe pain with exercise or illness. They can also develop a heart that does not function properly. These patients are tired and expend less energy than people who do not have a long-chain fatty acid oxidation disorder. However, they can use a supplement oil called medium chain triglyceride or MCT. This study will determine if a new experimental oil called Triheptanoin can decrease the muscle pain and increase the heart function and the amount of energy in patients with long-chain fatty acid oxidation disorders. Funding source - FDA's OOPD
Study Details
Timeline
Interventions
Triglyceride with three heptanoin or 7 carbon fatty acids esterified to a glycerol backbone