Triheptanoin in efficient in the long-term for the treatment of patients with long-chain fatty acid oxidation disorder (LCFAOD), confirms a new study published in the Journal of Inherited Metabolic Disease.
This finding is based on the results of the open-label extension of a phase 2 study called CL20 that evaluated the impact of triheptanoin on acute clinical pathophysiology associated with LCFAOD.
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The open-label extension assessed the long-term safety and efficacy of triheptanoin in 94 patients, who are at least 6 months of age. Of these, 33 were triheptanoin naïve while the remaining 61 received triheptanoin either as part of the CL201 study (24 patients) or investigator-sponsored trials or expanded access programs (37 patients).
The primary endpoint of the extension study was the annualized rate of major clinical events including cardiomyopathy, hypoglycemia, and rhabdomyolysis.
The results showed that in patients who were triheptanoin-naïve, the median rate of major clinical events decreased significantly from 2 events per patient per year before treatment to 0.28 events per patient per year after treatment corresponding to an 86% reduction.
In patients who had received triheptanoin as part of the CL201 trial, the mean rate of major clinical events also significantly decreased from 1.76 events per patient per year to 1 event per patient per year, a reduction of 43%.
In patients who previously received triheptanoin as part of investigator-sponsored trials or expanded access programs, the mean rate of major clinical events was 1.4 events per patient per year with triheptanoin treatment.
The researchers reported that safety data were consistent with previous observations with treatment-related treatment-emergent adverse events occurring in 68.1% of patients. These were mostly mild or moderate in severity, they said. A total of 5 patients had 7 serious treatment-related treatment-emergent adverse events but these were all resolved.
Triheptanoin is a medium-chain triglyceride that is used for the treatment of children and adults with LCFAOD.
References
Vockley J, Burton BK, Berry G, et al. Triheptanoin for the treatment of long-chain fatty acid oxidation disorders: Final results of an open-label, long-term extension study. J Inherit Metab Dis. June 5, 2023. doi:10.1002/jimd.12640
Long-chain fatty acid oxidation disorders (LC-FAOD) extension study for subjects previously enrolled in triheptanoin studies. US National Library of Medicine. Updated December 8, 2021. Accessed June 12, 2023.
