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

Last updated: March 4, 2026
Sponsor: Ultragenyx Pharmaceutical Inc
Overall Status: Active - Not 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

    Site Not Available

  • University of Alberta

    Edmonton 5946768, Alberta 5883102 T6G 1C9
    Canada

    Site Not Available

  • CHEO (Children's Hospital Eastern Ontario)

    Ottawa, Ontario K1H 8L1
    Canada

    Site Not Available

  • SickKids (The Hospital for Sick Children)

    Toronto, Ontario M5G 1X8
    Canada

    Site Not Available

  • The Hospital for Sick Children

    Toronto, Ontario M5G1X8
    Canada

    Active - Recruiting

  • CHEO (Children's Hospital Eastern Ontario)

    Ottawa 6094817, Ontario 6093943 K1H 8L1
    Canada

    Site Not Available

  • SickKids (The Hospital for Sick Children)

    Toronto 6167865, Ontario 6093943 M5G 1X8
    Canada

    Site Not Available

  • Phoenix Children's Hospital

    Phoenix, Arizona 85016
    United States

    Site Not Available

  • Phoenix Children's Hospital

    Phoenix 5308655, Arizona 5551752 85016
    United States

    Site Not Available

  • University of California San Francisco

    San Francisco, California 94158
    United States

    Site Not Available

  • University of California San Francisco

    San Francisco 5391959, California 5332921 94158
    United States

    Site Not Available

  • Children's Hospital of Colorado

    Aurora, Colorado 80045
    United States

    Site Not Available

  • Children's Hospital of Colorado

    Aurora 5412347, Colorado 5417618 80045
    United States

    Site Not Available

  • University of South Florida

    Tampa, Florida 33606
    United States

    Site Not Available

  • University of South Florida

    Tampa 4174757, Florida 4155751 33606
    United States

    Site Not Available

  • The Emory Clinic

    Atlanta, Georgia 30322
    United States

    Site Not Available

  • The Emory Clinic

    Atlanta 4180439, Georgia 4197000 30322
    United States

    Site Not Available

  • Ann & Robert H. Lurie Children's Hospital

    Chicago, Illinois 60611
    United States

    Site Not Available

  • Ann & Robert H. Lurie Children's Hospital

    Chicago 4887398, Illinois 4896861 60611
    United States

    Site Not Available

  • Boston Children's Hospital

    Boston, Massachusetts 02215
    United States

    Site Not Available

  • Boston Children's Hospital

    Boston 4930956, Massachusetts 6254926 02215
    United States

    Site Not Available

  • University of Minnesota

    Minneapolis, Minnesota 55454
    United States

    Site Not Available

  • University of Minnesota

    Minneapolis 5037649, Minnesota 5037779 55454
    United States

    Site Not Available

  • Columbia University

    New York, New York 10032
    United States

    Site Not Available

  • Columbia University

    New York 5128581, New York 5128638 10032
    United States

    Site Not Available

  • Nationwide Children's Hospital

    Columbus, Ohio 43205
    United States

    Site Not Available

  • Nationwide Children's Hospital

    Columbus 4509177, Ohio 5165418 43205
    United States

    Site Not Available

  • Children's Hospital of Pittsburgh of UPMC

    Pittsburgh, Pennsylvania 15224
    United States

    Site Not Available

  • Children's Hospital of Pittsburgh of UPMC

    Pittsburgh 5206379, Pennsylvania 6254927 15224
    United States

    Site Not Available

  • University of Utah

    Salt Lake City, Utah 84108
    United States

    Site Not Available

  • University of Utah

    Salt Lake City 5780993, Utah 5549030 84108
    United States

    Site Not Available

  • Seattle Children's Hospital

    Seattle, Washington 98020
    United States

    Site Not Available

  • Seattle Children's Hospital

    Seattle 5809844, Washington 5815135 98020
    United States

    Site Not Available

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