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

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: Phase 2 Study of Triheptanoin for Treatment of Long-Chain Fatty Acid Oxidation Disorders

Brief 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

Detailed Summary:

Recruitment: Patients with a long-chain fatty acid oxidation disorder will be recruited through our clinic, past research participants, a patient support website, and recruitment letters mailed to physicians around the US. We will enroll 16 subjects at OHSU and 16 subjects at the University of Pittsburgh, age 7 to 40, with a disorder in fatty acid oxidation.

Procedures: Subjects will be admitted to the clinical research center for 4 days. They will collect all their urine for 24 hours. Heart function will be measured using ultrasound an electrocardiogram (ECG). The motion of the heart will be measured by magnetic resonance imaging (MRI). For this test, the patient lies in the magnetic field of the MRI machine in the Advanced Imaging Resource Center (AIRC) for about 45 minutes. The amount of muscle and fat in the whole body and inside the liver and muscle will be measured by MRS and by dual X-ray absorptiometry (DEXA). Subjects will walk on a treadmill for about 45 minutes. The amount of Calories they use, their heart rate, and if they burn fat or carbohydrates will be measured. Blood samples will be collected before and after exercise. A meal test will be used to determine how much fat they burn. The subjects will drink a liquid breakfast with a stable isotope labeled fat in the breakfast. Breath and blood samples will be collected before and after the meal. The amount Calories burned by each subject will be measured when they are at rest on a bed by indirect calorimetry. The amount of Calories burned by subjects when they are doing their routine daily activities will be measured at home by doubly labeled water. All of these tests will be done at baseline. Then, subjects will be randomly assigned to consume MCT (current standard of care) or triheptanoin at 20% of their estimated Calorie needs for 4 months. The subject and/or the parent will be taught how to use the supple
Sponsor: Oregon Health and Science University

Current Primary Outcome:

  • Energy Expenditure [ Time Frame: change from baseline after 4 months of treatment ]
    Total energy expenditure will be measured by doubly labeled water and resting energy expenditure will be measured by indirect calorimetry at baseline and again after 4 months of either MCT or trihpetanoin treatment.
  • Ejection Fraction [ Time Frame: 4 months ]
    Change in resting ejection fraction over 4 month treatment period


Original Primary Outcome: Energy Expenditure [ Time Frame: change from baseline after 4 months of treatment ]

Total energy expenditure will be measured by doubly labled water and resting energy expenditure will be measured by indirect calorimetry at baseline and again after 4 months of either MCT or trihpetanoin treatment.


Current Secondary Outcome: Exercise Heart Rate [ Time Frame: change from baseline to 4 months of treatment ]

Subjects will complete a submaximal treadmill exercise study at baseline. Exercise heart heart, ventilation and perceived exertion will be measured. Subjects will be randomized to MCT or triheptanoin supplementation for 4 months. At the end of treatment, the exercise test will be repeated keeping work performed constant. Change in exercise heart rate, ventilation and exertion will be compared between groups.


Original Secondary Outcome: Exercise Heart Rate [ Time Frame: change from baseline to 4 months of treatment ]

Subjects will complete a submaximal treadmill exercise study at baseline. Exercise heart heart, ventilation and percieved exertion will be measured. Subjects will be randomized to MCT or triheptanoin supplementation for 4 months. At the end of treatment, the exercise test will be repeated keeping work performed constant. Change in exercise heart rate, ventilation and exercition will be compared between groups.


Information By: Oregon Health and Science University

Dates:
Date Received: June 21, 2011
Date Started: September 2011
Date Completion:
Last Updated: March 21, 2017
Last Verified: March 2017