Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program
NCT ID: NCT04632953
Last Updated: 2025-12-02
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
150 participants
OBSERVATIONAL
2021-11-30
2035-12-31
Brief Summary
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Detailed Description
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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.
Conditions
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Study Design
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COHORT
PROSPECTIVE
Study Groups
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Cohort 1: Previously Treated with Triheptanoin
Patients who have been previously treated with triheptanoin in clinical studies: UX007-CL201 (NCT01886378), UX007-CL202 (NCT02214160), UX007-CL302 (2022-001539-10), Investigator Sponsored Trials (ISTs), or UX007-EAP (NCT03773770).
No Intervention
No Intervention
Cohort 2: Currently or Previously Treated with Triheptanoin
New patients enrolling into the DMP currently or previously treated with triheptanoin (excluding those in Cohort 1).
No Intervention
No Intervention
Cohort 3: Triheptanoin Naïve
New patients enrolling into the DMP with no exposure to triheptanoin (naïve).
No Intervention
No Intervention
Interventions
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No Intervention
No Intervention
Eligibility Criteria
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Inclusion Criteria
* 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.
Exclusion Criteria
* 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.
ALL
No
Sponsors
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Ultragenyx Pharmaceutical Inc
INDUSTRY
Responsible Party
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Principal Investigators
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Medical Director
Role: STUDY_DIRECTOR
Ultragenyx Pharmaceutical Inc
Locations
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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
Countries
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Related Links
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Ultragenyx Patient Advocacy/LC-FAOD Disease Information
Ultragenyx Transparency Commitment
Other Identifiers
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UX007-CL401
Identifier Type: -
Identifier Source: org_study_id
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