A randomized, double-blind trial of triheptanoin for drug-resistant epilepsy in glucose transporter 1 deficiency syndrome.
Pasquale StrianoStėphane AuvinAbigail CollinsRita HorvathIngrid Eileen SchefferMichal TzadokIan MillerMary Kay KoenigAdrian LacyRonald DavisAngela Garcia-CazorlaRussell P SanetoMelanie BrandaburSusan BlairTony KoutsoukosDarryl De VivoPublished in: Epilepsia (2022)
Triheptanoin did not significantly reduce seizure frequency in patients with Glut1DS not on the ketogenic diet. Treatment was associated with mild to moderate gastrointestinal treatment-related events; most resolved following dose reduction or interruption and/or medication for treatment. Triheptanoin was not associated with any long-term safety concerns when administered at dose levels up to 35% of total daily caloric intake for up to 1 year.