A new clinical trial evaluated the efficacy safety of triheptanoin on pediatric patients with long-chain fatty acid oxidation disorders (LCFAOD), comparing its performance with medium-chain triglycerides (MCT) oil. The phase 3 study could provide a novel therapeutic alternative for the condition.
The randomized, multicenter, double-blind study aims to enroll a total of 60 participants. The participants and healthcare professionals involved in the study will be blinded to the assigned treatment of either triheptanoin or MCT oil to produce unbiased results.
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The primary objective of this study is to assess the impact of triheptanoin, a synthetic triglyceride, compared to MCT oil on the frequency of Major Clinical Events (MCEs) experienced by pediatric patients with LCFAOD.
The trial will randomly assign participants in a 1:1 ratio to receive either triheptanoin or MCT oil. The study duration is estimated to be between 3.5 to 4 years and comprises several phases, namely the screening period, baseline, double-blind treatment period (including titration and end of study visit), and safety follow-up phone visit.
Additionally, a liver substudy will investigate the effects of triheptanoin versus MCT oil in reducing hepatic lipid content to prevent or improve steatosis in subjects with LCFAOD. Selected participants who were at least 2 years of age will be invited to participate in this important substudy.
The primary outcome measure is the annualized event rate of MCEs during the double-blind treatment period.
Secondary outcome measures include the annualized duration of MCEs, annualized hypoglycemic event-rate, clinician-reported change in global impression of disease severity, and several other parameters assessing liver health, survival time, and overall quality of life.
The study is set to commence on February 28, 2023, and the estimated completion date is September 2026.
Reference
A study to determine the effect of triheptanoin compared with even-chain MCT on MCEs in pediatric patients with LCFAOD. ClinicalTrials.gov. Updated July 20, 2023. Accessed July 12, 2023
