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dc.contributor.authorStriano, Pasquale-
dc.contributor.authorAuvin, Stéphane-
dc.contributor.authorCollins, Abigail-
dc.contributor.authorHorvath, Rita-
dc.contributor.authorScheffer, Ingrid E-
dc.contributor.authorTzadok, Michal-
dc.contributor.authorMiller, Ian-
dc.contributor.authorKay Koenig, Mary-
dc.contributor.authorLacy, Adrian-
dc.contributor.authorDavis, Ronald-
dc.contributor.authorGarcia-Cazorla, Angela-
dc.contributor.authorSaneto, Russell P-
dc.contributor.authorBrandabur, Melanie-
dc.contributor.authorBlair, Susan-
dc.contributor.authorKoutsoukos, Tony-
dc.contributor.authorDe Vivo, Darryl-
dc.identifier.citationEpilepsia 2022; 63(7): 1748-1760en
dc.description.abstractThis study was undertaken to evaluate efficacy and long-term safety of triheptanoin in patients >1 year old, not on a ketogenic diet, with drug-resistant seizures associated with glucose transporter 1 deficiency syndrome (Glut1DS). UX007G-CL201 was a randomized, double-blind, placebo-controlled trial. Following a 6-week baseline period, eligible patients were randomized 3:1 to triheptanoin or placebo. Dosing was titrated to 35% of total daily calories over 2 weeks. After an 8-week placebo-controlled period, all patients received open-label triheptanoin through Week 52. The study included 36 patients (15 children, 13 adolescents, eight adults). A median 12.6% reduction in overall seizure frequency was observed in the triheptanoin arm relative to baseline, and a 13.5% difference was observed relative to placebo (p = .58). In patients with absence seizures only (n = 9), a median 62.2% reduction in seizure frequency was observed in the triheptanoin arm relative to baseline. Only one patient with absence seizures only was present in the control group, preventing comparison. No statistically significant differences in seizure frequency were observed. Common treatment-emergent adverse events included diarrhea, vomiting, abdominal pain, and nausea, mostly mild or moderate in severity. No serious adverse events were considered to be treatment related. One patient discontinued due to status epilepticus. 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.en
dc.subjectdiet treatmenten
dc.subjectdrug resistanceen
dc.subjectglucose transporter 1 deficiency syndromeen
dc.titleA randomized, double-blind trial of triheptanoin for drug-resistant epilepsy in glucose transporter 1 deficiency syndrome.en
dc.typeJournal Articleen
dc.identifier.affiliationAustin Healthen
dc.identifier.affiliationThe Florey Institute of Neuroscience and Mental Healthen
dc.identifier.affiliationUniversity of Melbourne, Parkville, Victoria, Australiaen
dc.identifier.affiliationDepartment of Clinical Neurosciences, University of Cambridge, Cambridge, UKen
dc.identifier.affiliationPediatric Neurology Units, Edmond and Lily Safra Children's Hospital, Sheba Medical Center, Tel HaShomer, Affiliated with the Sackler Faculty of Medicine, Tel Aviv University, Ramat Gan, Israelen
dc.identifier.affiliationMiami Children's Research Institute, Miami, Florida, USAen
dc.identifier.affiliationUniversity of Texas Health Science Center at Houston, Houston, Texas, USAen
dc.identifier.affiliationCook Children's Medical Center, Fort Worth, Texas, USAen
dc.identifier.affiliationNeurology and Epilepsy Research Center, Pediatric Neurology, Orlando, Florida, USAen
dc.identifier.affiliationSant Joan de Déu Hospital, Barcelona, Spainen
dc.identifier.affiliationDepartment of Neurology, Division of Pediatric Neurology, University of Washington/Seattle Children's Hospital, Seattle, Washington, USAen
dc.identifier.affiliationUltragenyx Pharmaceutical Inc, Novato, California, USAen
dc.identifier.affiliationColumbia University Irving Medical Center, New York, New York, USAen
dc.identifier.affiliationDepartments of Neurosciences, Rehabilitation, Ophthalmology, Genetics, and Maternal and Child Health, University of Genoa, Genoa, Italyen
dc.identifier.affiliationRobert Debré University Hospital and University of Paris, Paris, Franceen
dc.identifier.affiliationUniversity Institute of France, Paris, Franceen
dc.identifier.affiliationG. Gaslini Institute, Scientific Institute for Research and Health Care, Genoa, Italyen
dc.identifier.affiliationChildren's Hospital Colorado, Aurora, Colorado, USAen
dc.identifier.affiliationMurdoch Children's Research Institute, Parkville, VIC, Australiaen
dc.identifier.affiliationRoyal Children's Hospital, Melbourne, VIC, Australiaen
dc.identifier.pubmedid35441706-, Ingrid E
item.openairetypeJournal Article-
item.fulltextNo Fulltext-
item.openairecristype Research Centre-
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