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Active Not Recruiting
NCT04632953
Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program
Conditions: Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
Sex: All
Healthy volunteers: No
Enrollment: 150
Sponsor: Ultragenyx Pharmaceutical Inc
Location: Phoenix Children's Hospital Phoenix Arizona
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 Criteria:
* 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.
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.
Source: ClinicalTrials.gov (NCT04632953). StuddyBuddy aggregates publicly available trial information.
