Trial reportBMC medical genomics2026
Long-term safety and efficacy of triheptanoin in Korean patients with long-chain fatty acid oxidation disorders: a prospective, open-label, single-center, phase II clinical study.
Trial report in BMC medical genomics, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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11 authors.
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Abstract
backgroundLong-chain fatty acid oxidation disorders (LC-FAOD) are inherited metabolic conditions caused by impaired mitochondrial β-oxidation of long-chain fatty acids, leading to rhabdomyolysis, cardiomyopathy, and hypoglycemia. Triheptanoin, a medium odd-chain triglyceride, provides anaplerotic substrates that replenish tricarboxylic acid cycle intermediates, offering alternative energy sources and potential therapeutic benefit.
methodsThis open-label, single-center phase II study evaluated the safety and efficacy of triheptanoin in 10 Korean patients with genetically confirmed LC-FAOD who experienced recurrent rhabdomyolysis despite conventional treatment. Triheptanoin was titrated to 35% of total daily caloric intake, and all patients completed 3 years of treatment. The primary outcome was safety, assessed by adverse events. Secondary outcomes included changes in the major clinical event (MCE) rate and duration, functional capacity, health-related quality of life (HRQoL), and organ function.
resultsThe mean study duration was 3.5 ± 0.3 years. Serious adverse events occurred in nine patients (90%), most commonly rhabdomyolysis (100%), and were considered unrelated to triheptanoin. Mild gastrointestinal symptoms during titration were the most frequent treatment-related adverse events (80%). The median annualized MCE rate and duration decreased by 42.4% and 43.6%, respectively (P = 0.114 and P = 0.059). The duration per MCE was significantly reduced by 3.5 days, as assessed by a linear mixed-effects model (P = 0.022), with a significant reduction observed in the child subgroup (P < 0.001). In eight patients, the 12-minute walk test distance increased by 12.4% at 6 months and was maintained over 3 years (P = 0.385). HRQoL improved to within the normative range in adults, whereas child self-reported and parent proxy-reported scores showed an initial increase but remained suboptimal at 3 years. Progressive neuropathy and newly developed retinopathy and attention-deficit/hyperactivity disorder were observed during treatment.
conclusionTriheptanoin significantly reduced hospitalization burden in Korean patients with LC-FAOD and was well-tolerated, with potential benefits in physical function.
trial registrationClinical Research Information Service (CRIS), Republic of Korea, KCT0006756, 15 November 2021.
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