Energy Metabolism Disorder Therapy Using Triheptanoin (C7)

Dietary Therapy for Inherited Disorders of Energy Metabolism

Protocol Description

The goal of this study is to better understand the therapeutic effects of triheptanoin oil (C7 oil) in treatment of disorders of energy metabolism, including mitochondrial fat oxidation and glycogenoses.

Eligibility Criteria

The study is open to participants of both genders, ages 1 month through adulthood, who have been diagnosed with an inherited disorder of fatty acid oxidation and energy metabolism.
Males and Females: Age 1 month through adulthood


Participants will receive a complete metabolic evaluation, including blood sampling and heart monitoring at the study onset, after which they will be prescribed C7 oil and diet modification guidelines to maintain appropriate caloric intake and balance. Participants will closely monitor weight in a weekly journal and will be contacted at intervals by researchers. Blood tests will be done at 2, 6, 12 and 18 months.
Visits: 2 to 5
Duration: 18 months, and if the drug is found to be therapeutic, enrollment in a long-term maintenance program may follow.

Status: Open for Enrollment

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, CGC