Clinical Outcome of Triheptanoin Treatment in Patients With Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Treated Under Expanded Access Program

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

Official title: A Retrospective Chart Review Study to Assess the Clinical Outcome of Triheptanoin Treatment in Patients With Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Treated Under Expanded Access Program

Status Completed

Clinical Trial Summary

The primary objective of the study is to evaluate the clinical outcomes for the trigger event of patients who receive triheptanoin in the emergency Investigational New Drug (eIND) program.

Clinical Trial Description

The study is a retrospective chart review of patients in the USA with LC-FAOD who received triheptanoin via eIND request from their metabolic or treating physician before 01 September 2018. Participating study sites will provide the medical records of subjects who meet eligibility criteria, and relevant data regarding the trigger event (the reason the patient was placed on triheptanoin) and key details about hospitalizations during the study period will be extracted. ;

Related Conditions & MeSH terms

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

• NCT number: NCT03768817
• Study type: Observational
• Source: Ultragenyx Pharmaceutical Inc
• Status: Completed
• Start date: January 30, 2019
• Completion date: June 8, 2020