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

Last updated: June 11, 2025
Sponsor: Ultragenyx Pharmaceutical Inc
Overall Status: Active - Recruiting

Phase

N/A

Condition

N/A

Treatment

No Intervention

Clinical Study ID

NCT04632953
UX007-CL401
  • All Genders

Study Summary

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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Confirmed diagnosis of any LC-FAOD subtype. Diagnosis must be confirmed by resultsof acylcarnitine profiles and/or genetic testing results obtained from medicalrecords 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 informedconsent by their legally authorized representative.

  • Females of childbearing potential who become pregnant during the study will beinvited to remain in the study. Pregnant females with LC-FAOD will be informed ofthe study and invited to enroll.

Exclusion

Exclusion Criteria:

  • Presence of a concurrent disease or condition that would interfere with studyparticipation or affect patient's safety in the opinion of the Investigator.

  • Presence or history of any condition that, in the view of the Investigator, placesthe patient at high risk of not completing the study or would affect theinterpretation of study results.

Study Design

Total Participants: 150
Treatment Group(s): 1
Primary Treatment: No Intervention
Phase:
Study Start date:
November 30, 2021
Estimated Completion Date:
December 31, 2035

Study Description

The LC-FAOD Disease Monitoring Program (DMP) is an international, long-term, retrospective and prospective outcomes study aiming to collect safety and effectiveness data for triheptanoin and the natural history of LC-FAOD for a study duration of up to 10 years from adult and pediatric patients with LC-FAOD, with any previous disease management, regardless of prior treatment with triheptanoin, those who have previously participated in triheptanoin clinical trials, and those who received triheptanoin through an Expanded Access Program (EAP).

Patients enrolling in the LC-FAOD DMP will be managed at the discretion of their physicians and may or may not be treated with triheptanoin during the course of the study. Patients will have access to triheptanoin only through authorized commercial use (if approved in their country) or available EAP but not from the LC-FAOD DMP itself.

Connect with a study center

  • University of Alberta

    Edmonton, Alberta T6G 1C9
    Canada

    Active - Recruiting

  • CHEO (Children's Hospital Eastern Ontario)

    Ottawa, Ontario K1H 8L1
    Canada

    Active - Recruiting

  • SickKids (The Hospital for Sick Children)

    Toronto, Ontario M5G 1X8
    Canada

    Active - Recruiting

  • The Hospital for Sick Children

    Toronto, Ontario M5G1X8
    Canada

    Active - Recruiting

  • Phoenix Children's Hospital

    Phoenix, Arizona 85016
    United States

    Active - Recruiting

  • University of California San Francisco

    San Francisco, California 94158
    United States

    Active - Recruiting

  • Children's Hospital of Colorado

    Aurora, Colorado 80045
    United States

    Active - Recruiting

  • University of South Florida

    Tampa, Florida 33606
    United States

    Active - Recruiting

  • The Emory Clinic

    Atlanta, Georgia 30322
    United States

    Active - Recruiting

  • Ann & Robert H. Lurie Children's Hospital

    Chicago, Illinois 60611
    United States

    Active - Recruiting

  • Boston Children's Hospital

    Boston, Massachusetts 02215
    United States

    Active - Recruiting

  • University of Minnesota

    Minneapolis, Minnesota 55454
    United States

    Active - Recruiting

  • Columbia University

    New York, New York 10032
    United States

    Active - Recruiting

  • Nationwide Children's Hospital

    Columbus, Ohio 43205
    United States

    Active - Recruiting

  • Children's Hospital of Pittsburgh of UPMC

    Pittsburgh, Pennsylvania 15224
    United States

    Active - Recruiting

  • University of Utah

    Salt Lake City, Utah 84108
    United States

    Active - Recruiting

  • Seattle Children's Hospital

    Seattle, Washington 98020
    United States

    Site Not Available

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