Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program

Running, not enrolling

Conditions studied: Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)

In brief

The primary objective of this study is to assess the long-term safety, including pregnancy, infant, and lactation outcomes, of patients with LC-FAOD who are enrolled in the DMP.

Key facts

Study ID

NCT04632953

Run by

Ultragenyx Pharmaceutical Inc

People needed

150

Starts

2021-11-30

Expected to finish

2035-12-01

Last updated by the study team

2026-06-03

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Confirmed diagnosis of any LC-FAOD subtype. Diagnosis must be confirmed by results of acylcarnitine profiles and/or genetic testing results obtained from medical records or equivalent documentation.
• Willing and able to comply with all study procedures.
• Willing and able to provide consent or, if a minor, provide assent and informed consent by their legally authorized representative.
• Females of childbearing potential who become pregnant during the study will be invited to remain in the study. Pregnant females with LC-FAOD will be informed of the study and invited to enroll.

You may not qualify if…

• Presence of a concurrent disease or condition that would interfere with study participation or affect patient's safety in the opinion of the Investigator.
• Presence or history of any condition that, in the view of the Investigator, places the patient at high risk of not completing the study or would affect the interpretation of study results.

Where it is running

• Phoenix Children's Hospital — Phoenix, Arizona, United States
• University of California San Francisco — San Francisco, California, United States
• Children's Hospital of Colorado — Aurora, Colorado, United States
• University of South Florida — Tampa, Florida, United States
• The Emory Clinic — Atlanta, Georgia, United States
• Ann & Robert H. Lurie Children's Hospital — Chicago, Illinois, United States
• Boston Children's Hospital — Boston, Massachusetts, United States
• University of Minnesota — Minneapolis, Minnesota, United States
• Columbia University — New York, New York, United States
• Nationwide Children's Hospital — Columbus, Ohio, United States
• Children's Hospital of Pittsburgh of UPMC — Pittsburgh, Pennsylvania, United States
• University of Utah — Salt Lake City, Utah, United States
• Seattle Children's Hospital — Seattle, Washington, United States
• University of Alberta — Edmonton, Alberta, Canada
• CHEO (Children's Hospital Eastern Ontario) — Ottawa, Ontario, Canada
• SickKids (The Hospital for Sick Children) — Toronto, Ontario, Canada

Full record on ClinicalTrials.gov

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