Triheptanoin for seizure reduction disappoints in patients with Glut1DS

Treatment with triheptanoin does not lead to a significant reduction in the frequency of seizures associated with glucose transporter 1 deficiency syndrome (Glut1DS), as shown in a study.

A total of 36 patients (15 children, 13 adolescents, eight adults) were randomized to receive either triheptanoin or placebo following a 6-week baseline period. Over the first 2 weeks of treatment, dosing was titrated to 35 percent of total daily calories. After an 8-week placebo-controlled period, all patients received open-label triheptanoin through week 52.

The mean age of the population was 14.3 years; 61.1 percent of patients were women and 88.9 percent were White. At week 52, median overall seizure frequency dropped by 12.6 percent in the triheptanoin arm as compared with a reduction of 13.5 percent in the placebo arm (p=0.58).

In the subgroup of patients with absence seizures only (n=9), triheptanoin produced a greater reduction, a median of 62.2 percent, in seizure frequency relative to baseline. Between-group comparison was not possible due to the placebo arm having only one patient with absence seizures.

Commonly reported treatment-emergent adverse events were diarrhoea, vomiting, abdominal pain, and nausea. These events were mostly mild or moderate in severity. No serious adverse events were considered related to treatment. One patient ceased treatment due to status epilepticus.