Long-Chain Fatty Acid Oxidation Disorder Monitoring Program

Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program (LC-FAOD DMP)

Protocol Description

The purpose of this multi-center, international observational study is to help researchers understand the progression of long-chain fatty acid oxidation disorders (LC-FAOD) over time. LC-FAOD are inherited conditions in which the body is unable to break down fats present in normal foods to produce energy. The study will collect information for up to 10 years from adult and pediatric participants with a confirmed diagnosis of LC-FAOD. Although no drug will be provided to participants as part of this study, researchers will assess the long-term safety and effectiveness of Dojolvi® (triheptanoin) in participants who use Dojolvi as part of their treatment for LC-FAOD.

Eligibility Criteria

Subject to certain exclusion criteria, this study is accepting people of all ages who have been diagnosed with a LC-FAOD.
Males and Females: All ages


Study participants will visit the study site 8 times for examinations and have 4 telephone follow-up visits during the 10 years of their enrollment.
Visits: 8
Duration: 10 years

Status: Open to Enrollment

Source(s) of Support

Ultragenyx Pharmaceutical, Inc.

Primary Investigator

Gerard Vockley, MD, PhD

Contact Information

For more information about the study or enrollment, please contact:
Danielle Black, MS