A Randomised study to Determine the Effect of Triheptanoin in Paediatric Patients with LC-FAOD
Research type
Research Study
Full 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)
IRAS ID
1006078
Contact name
Rosy Veliz
Contact email
Sponsor organisation
Ultragenyx Pharmaceutical Inc.
Eudract number
2022-001539-10
Clinicaltrials.gov Identifier
Research summary
Long-chain fatty acid oxidation disorders (LC-FAOD) are a group of 6 rare and life-threatening disorders caused by defects in genes critically involved in the conversion of long-chain fatty acids into energy. The resulting defects in energy metabolism, with increased fatty acid metabolites and ineffective production of ketones in the liver, manifest as acute crises of energy deficiency. These metabolic crises may often require emergency medical management.
Metabolic crises can be induced or aggravated by fasting, exercise, exposure to cold, or metabolic stress (eg, infection). Between these events, patients may have periods of normal function; however, minor events such as viral infections or overexertion may trigger profound metabolic crises in these vulnerable patients, initiating vital organ damage, that can ultimately lead to death. Therefore, reducing Major Clinical Events (MCEs) in patients with LC-FAOD would fulfil a profound unmet need.
Triheptanoin (UX007) is being developed for the treatment of LC-FAOD. The purpose of this study is to investigate the effect of triheptanoin versus Triglycerides (known as MCTs) on the frequency of MCEs in paediatric patients with LC-FAOD.
The study will recruit 60 paediatric participants with LC-FAOD age 0 (including newborns) to 17 years of age with a caregiver willing/able to assist in the study requirements.
Participants will be randomly assigned 1:1 to receive triheptanoin or MCT oil and will be kept blinded to treatment assignment for the entire study. The planned duration of participation is a minimum of 2 years of treatment. Participants will be closely monitored by a dietician to assist them and their families with a nutritional plan to support and standardize study treatment.
A Data Monitoring Committee (DMC), which is independent of the sponsor, will assess safety data on an ongoing basis to ensure patient safety at all times.
Ultragenyx Pharmaceutical, Inc. is the sponsor of this study.REC name
London - Brent Research Ethics Committee
REC reference
22/LO/0758
Date of REC Opinion
9 Feb 2023
REC opinion
Further Information Favourable Opinion