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

Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Clinical Trial

Official title: Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program (LC-FAOD DMP)

Status Recruiting

Clinical Trial 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.

Clinical Trial Description

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. ;

Related Conditions & MeSH terms

• Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)

• NCT number: NCT04632953
• Study type: Observational
• Source: Ultragenyx Pharmaceutical Inc
• Contact: Patients Contact: Trial Recruitment
• Phone: 1-888-756-8657
• Email: trialrecruitment@ultragenyx.com
• Status: Recruiting
• Start date: November 30, 2021
• Completion date: December 2035