Study of Triheptanoin for Treatment of Long-Chain Fatty Acid Oxidation Disorder

• Status: Completed
• Trial end date: 2015-04-01
• Target enrollment: 0
• Participant gender: All

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

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Oregon Health and Science University
• Collaborator: University of Pittsburgh

Criteria

Inclusion Criteria:

• Confirmed diagnosis of Very long-chain acylCoA dehydrogenase (VLCAD) Deficiency, Carnitine Palmitoyltransferase 2 (CPT2) Deficiency, Mitochondrial Trifunctional Protein (TFP) Deficiency, or Long-chain 3 hydroxyacylCoA dehydrogenase (LCHAD) deficiency

• > 7 years

• Ability to travel to CRC to participate

• Ability to follow protocol

Exclusion Criteria:

• Hgb < 10 g/dl

• Peripheral neuropathy that limits ability to complete treadmill studies

• Inclusion in another research study that alters macronutrient intake

• Pregnant females

• history of myocardial infarction or cardiovascular disease