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

Completed · Phase 2

Conditions studied: Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency, Carnitine Palmitoyltransferase 2 (CPT2) Deficiency, Mitochondrial Trifunctional Protein (TFP) Deficiency, Long-chain 3 hydroxyacylCoA Dehydrogenase (LCHAD) Deficiency

In brief

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

Key facts

Study ID

NCT01379625

Run by

Oregon Health and Science University

People needed

32

Starts

2011-09-01

Expected to finish

2015-04-01

Last updated by the study team

2017-03-23

Who can join

Age: 7 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• Oregon Health & Science University — Portland, Oregon, United States
• University of Pittsburgh — Pittsburgh, Pennsylvania, United States

Full record on ClinicalTrials.gov

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