Long-Chain Fatty Acid Oxidation Disorder Therapy With Triheptanoin (C7) - Phase II

Extension Study in Subjects with Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Previously Enrolled in UX007 or Triheptanoin Studies

Protocol Description

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.

Study Level: Phase II

Eligibility Criteria

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.
Visits: 11
Duration: 5 years

Status: Enrolling by Invitation

Source(s) of Support

Ultragenyx Pharmaceutical

Primary Investigator

Gerard Vockley, MD, PhD

Contact Information

For more information about the study or enrollment, please contact:
Elizabeth McCracken, MS, LCGC