Study of Triheptanoin for Treatment of Long-Chain Fatty Acid Oxidation Disorder
Status:
Completed
Trial end date:
2015-04-01
Target enrollment:
Participant gender:
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