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

  • End date
    Dec 20, 2035
  • participants needed
  • sponsor
    Ultragenyx Pharmaceutical Inc
Updated on 20 September 2023


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.


The LC-FAOD DMP is a global observational long-term prospective outcomes study aiming to collect information for up to 10 years from adult and pediatric patients with LC-FAOD, regardless of disease management, including treatment with triheptanoin, and those who have previously participated in triheptanoin clinical trials or 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 only have access to triheptanoin through authorized commercial use (if approved in their country) or available EAP but not from the LC-FAOD DMP itself.

Condition Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
Treatment No intervention
Clinical Study IdentifierNCT04632953
SponsorUltragenyx Pharmaceutical Inc
Last Modified on20 September 2023


Yes No Not Sure

Inclusion Criteria

Confirmed diagnosis of any LC-FAOD sub-type. 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
Female of child-bearing 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
Pregnant females not affected by LC-FAOD carrying a fetus affected by LC-FAOD with confirmed pre-natal diagnosis of LC-FAOD

Exclusion Criteria

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
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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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