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Through this investigation, researchers will study longer-term results from patients who take UX007 for treatment of a long-chain fatty acid oxidation disorder (FAOD). Participants will be followed for about 5 years or until the drug is approved. FAODs are inherited disorders in which the body is unable to breakdown fats in normal foods to convert to energy. People with a long-chain FAOD are usually placed on diets that limit their fat intake while providing a supplement of medium-chain triglycerides (MCT) that can be absorbed normally and used by the body. UX007, also referred to as triheptanoin or C7 oil, is an MCT that may act as a good source of energy for people with LC-FAOD.
Individuals of both genders, ages 6 months and up, who received UX007 or triheptanoin in a previous study, or who have failed conventional therapy and have a severe unmet need will be invited to enroll in this study extension.
Males and Females: All ages
All study participants will take a prescribed amount of UX007, a clear oil, orally at least 4 times per day with food or drink. Participants will return to the clinic about every 6 months over 5 years for physical examinations and tests by the study team.
Duration: 5 years
Study Description at National Institutes of Health
Medical Genetics Research
Gerard Vockley, MD, PhD
For more information about the study or enrollment, please contact:
Elizabeth McCracken, MS, LCGC
Children's Hospital's main campus is located in the Lawrenceville neighborhood. Our main hospital address is:
UPMC Children’s Hospital of Pittsburgh
One Children’s Hospital Way
4401 Penn Ave.
Pittsburgh, PA 15224
In addition to the main hospital, Children's has many convenient locations in other neighborhoods throughout the greater Pittsburgh region.
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Interested in giving to Children's Hospital? Visit Children's Hospital of Pittsburgh Foundation's website to make a donation online.