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Project Details

E.W. "Al" Thrasher

Status: Funded - Open


Triheptanoin as an add-on treatment for children with medically refractory epilepsy - a pilot study

Karin Borges, Ph.D.

Summary

BACKGROUND: Epilepsy is one of the most common brain disorders in children. Despite many medication and dietary treatment options about 45% of children with epilepsy still suffer from uncontrolled seizures and about 30% of patients find no seizure relief at all.

GAP: We established that triheptanoin, a synthetic, edible tasteless oil, protects mice against different types of seizures and improves brain energy metabolism. The oil is currently being used to treat children and adults with rare metabolic disorders in USA and we are currently testing the oil’s safety, tolerability and efficacy in adult patients with treatment-resistant epilepsy.

HYPOTHESIS: We hypothesize that triheptanoin will be safe and tolerated in children with epilepsy and will reduce the seizure burden in some children.

METHODS: A pilot study for safety and tolerability of triheptanoin as an add-on treatment for children with treatment-resistant epilepsy will be conducted at the Royal Children and Mater Children’s Hospitals in Brisbane, Australia.

RESULTS: Pending

IMPACT: This clinical trial will pave the way for the commercial development of triheptanoin as a new treatment for epilepsy in children and adults. This new treatment approach would be ideal for children with epilepsy in the developed and developing world, because: 1) triheptanoin does not require refrigeration and is straightforward to add to meals or drinks and 2) it is not expected to require close supervision nor to have significant side effects, in contrast to adding on another antiepileptic drug to control seizures.

Keywords:
Drug Therapy, Neurology, Prospective Cohort, Treatment, Human










Supervising Institution:
The University of Queensland

Project Location:
Australia

Award Amount:
$299,941

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