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

Launched by ULTRAGENYX PHARMACEUTICAL INC · Nov 16, 2020

Keywords

ClinConnect Summary

This clinical trial is focused on understanding the long-term safety of patients with Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD). Specifically, it aims to monitor how these disorders affect health outcomes over time, including during pregnancy and breastfeeding. The study is currently recruiting participants of all ages and genders who have a confirmed diagnosis of any subtype of LC-FAOD. This diagnosis must be supported by specific test results from their medical records.

To participate, individuals must be willing to follow the study's procedures and provide consent (or have a parent or guardian do so if they are a minor). If a female participant becomes pregnant during the study, she can continue to be part of the trial. However, some individuals may not be eligible if they have other serious health conditions that could interfere with the study or affect their safety. Participants can expect regular monitoring and support throughout the study, which aims to provide valuable insights into managing LC-FAOD and improving care for patients.

Gender

ALL

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.