In short
Clinical trials are studying Triheptanoin in children with long-chain fatty acid oxidation disorders (LC-FAOD). These studies look at whether Triheptanoin can reduce major clinical events and compare it with even-chain medium-chain triglycerides (MCT). The main focus is on effectiveness and trial outcomes in pediatric patients.
Key points
- Clinical trials are studying Triheptanoin in children with long-chain fatty acid oxidation disorders (LC-FAOD). The main trial is a Phase 3 study with 70 planned participants. It compares Triheptanoin with even-chain MCT. The main goal is to measure the annualized rate of major clinical events. This helps researchers see whether Triheptanoin can reduce important health events in pediatric patients.
Clinical trial overview
The available study is testing Triheptanoin in pediatric patients with long-chain fatty acid oxidation disorders (LC-FAOD). The study is designed to see whether Triheptanoin changes the rate of major clinical events (MCEs) compared with even-chain medium-chain triglycerides (MCT).
Study design and treatment groups
This is an interventional study, which means researchers assign a treatment and then compare results between groups. The study includes oral use of Triheptanoin and oral use of fat emulsions described as even-chain MCT in the source data.
Who can participate
The trial is focused on children with LC-FAOD, so the target group is pediatric patients with this condition. The source data does not give more detailed inclusion or exclusion rules.
What the trial measures
The main outcome is the annualized event rate of MCEs. This means the study counts how often major clinical events happen over one year, so researchers can compare the two treatment groups.
Trial phase and status
The study is a Phase 3 trial. Phase 3 studies usually test how well a treatment works in a larger group of patients, and this trial is listed as Authorised with an enrollment of 70 participants.
Key terms explained
LC-FAOD is a group of rare disorders where the body has trouble using long-chain fats for energy. MCT means medium-chain triglycerides, which are being used as the comparison treatment in this study. Enrollment is the planned number of people in the study, and here it is 70.