NCT05933200

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

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: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 69 (actual)
• Sponsor: Ultragenyx Pharmaceutical Inc · Industry
• Sex: All
• Age: 17 Years
• Healthy volunteers: Not accepted

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).

Detailed description

Participants will be randomly assigned 1:1 to receive triheptanoin or MCT oil. The duration of the study is estimated to be 3.5 to 4 years. The study comprises the following: Screening Period, Baseline (Month 0), Double-blind Treatment Period (including Titration and End of Study Visit), Safety Follow-up Phone Visit, and an Open Access Period. In addition, a substudy will examine the effect of triheptanoin versus MCT on decreasing liver fat fraction 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.

Conditions

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

Interventions

Type	Name	Description
DRUG	Triheptanoin	Liquid for oral (PO) or enteral feeding tube administration
DIETARY_SUPPLEMENT	MCT Oil	Liquid for oral (PO) or enteral feeding tube administration

Timeline

• Start date: 2023-02-28
• Primary completion: 2027-08-01
• Completion: 2027-08-01
• First posted: 2023-07-06
• Last updated: 2026-04-07

Locations

15 sites across 7 countries: Czechia, Germany, Japan, Poland, Saudi Arabia, Spain, Turkey (Türkiye)