New Epilespy Treatment Trials

New treatments reduce seizures in pediatric patients.

Multiple studies at Hackensack Meridian Joseph M. Sanzari Children’s Hospital, led by Eric. B Segal, M.D., pediatric epileptologist, are focused on reducing seizures for patients with severe forms of epilepsy.

• The hospital is participating in a study of fenfluramine hydrochloride, a diet drug from the 1990s, prescribed to patients with Dravet Syndrome. Initial findings suggest patients can experience a medium reduction of 70% in seizure frequency. Low-dose fenfluramine was just recently approved for the treatment of Dravet Syndrome.
• Dr. Segal is serving as principal investigator for Soticlestat, which inhibits the activity of an enzyme involved in cholesterol metabolism and has shown an effect of regulating the neurotransmitter glutamate, which can increase the initiation and spread of seizures. He is also the principal investigator for a Phase 1b/2a clinical trial in Lennox-Gastaut, Dravet Syndrome, and Tuberous Sclerosis Complex.
• The hospital is currently recruiting patients with Dravet Syndrome for two studies including utilizing a first-generation antihistamine that has been found to be very effective in Dravet Syndrome animal models. Another study is an observational study to better understand outcomes for a gene therapy for Dravet.

In addition, Joseph M. Sanzari Children’s Hospital has been the largest pediatric recruiter for the Valtoco® studies. Valtoco® is the only intranasal diazepam rescue medication found to be clinically superior to Diastat®, which was the only rescue medication available before 2020.