A Study to Determine the Effect of Triheptanoin Compared With Even-chain, MCT on MCEs in Pediatric Patients With LC-FAOD

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

Official title: A Randomized, Double-blind, Multicenter Study to Determine the Effect of Triheptanoin Compared With Even-chain, Medium-chain Triglycerides (MCT) on Major Clinical Events (MCEs) in Pediatric Patients With Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)

Status Recruiting

Clinical Trial Summary

The main goal of this study is to evaluate the effects of triheptanoin versus Medium-chain Triglycerides (MCT) on frequency of Major Clinical Events (MCEs).

Clinical Trial Description

Participants will be randomly assigned 1:1 to receive triheptanoin or MCT oil. The duration of the study, which is estimated to be 3.5 to 4 years and comprises of the following: Screening Period, Baseline (Month 0), Double-blind Treatment Period (including Titration and End of Study Visit), and Safety Follow-up Phone Visit.

In addition, a substudy will examine the effect of triheptanoin versus MCT on decreasing hepatic lipid content to avoid or improve steatosis in subjects with LC-FAOD. Participants older than 2 years of age at selected sites will be invited to screen for the Liver Substudy. ;

Related Conditions & MeSH terms

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

• NCT number: NCT05933200
• Study type: Interventional
• Source: Ultragenyx Pharmaceutical Inc
• Contact: Patients Contact: Trial Recruitment
• Phone: (+1) 888-756-8657
• Email: trialrecruitment@ultragenyx.com
• Status: Recruiting
• Phase: Phase 3
• Start date: February 28, 2023
• Completion date: September 2026