Table of Contents
- Clinical trial overview
- Study design and treatment groups
- Who can participate
- What the trial measures
- Trial phase and status
- Key terms explained
Clinical trial overview
The available study is testing Triheptanoin in pediatric patients with long-chain fatty acid oxidation disorders (LC-FAOD).[1] The study is designed to see whether Triheptanoin changes the rate of major clinical events (MCEs) compared with even-chain medium-chain triglycerides (MCT).[1]
Study design and treatment groups
This is an interventional study, which means researchers assign a treatment and then compare results between groups.[1] The study includes oral use of Triheptanoin and oral use of fat emulsions described as even-chain MCT in the source data.[1]
Who can participate
The trial is focused on children with LC-FAOD, so the target group is pediatric patients with this condition.[1] The source data does not give more detailed inclusion or exclusion rules.[1]
What the trial measures
The main outcome is the annualized event rate of MCEs.[1] This means the study counts how often major clinical events happen over one year, so researchers can compare the two treatment groups.[1]
Trial phase and status
The study is a Phase 3 trial.[1] 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.[1]
Key terms explained
LC-FAOD is a group of rare disorders where the body has trouble using long-chain fats for energy.[1] MCT means medium-chain triglycerides, which are being used as the comparison treatment in this study.[1] Enrollment is the planned number of people in the study, and here it is 70.[1]
