Phase 3 Study of Oral Clemizole Hydrochloride EPX-100 as Add-On Treatment for Children and Adults With Dravet Syndrome

What this Trial Is About

Researchers are evaluating clemizole hydrochloride EPX-100 as an additional treatment for children and adults with Dravet syndrome DS, a severe form of epilepsy. This Phase 3, multicenter, randomized, double-blind, placebo-controlled study aims to assess the safety and effectiveness of clemizole hydrochloride in reducing seizures. The study includes participants with confirmed DS who continue to have seizures despite other treatments and have a documented SCN1A genetic mutation. Participants first undergo a 4-week Observational Period to establish baseline seizure activity. Then, they enter a 16-week Double-Blind Period where they receive either clemizole hydrochloride or a placebo as an oral solution. Following this, eligible participants who complete the double-blind phase may continue to an Open-Label Extension Period, receiving clemizole hydrochloride for up to 3 years. During the study, participants are regularly monitored for changes in countable motor seizures per 28 days and other seizure-related outcomes. Assessments include clinical evaluations and seizure tracking, with safety monitored throughout the trial. The primary outcome focuses on seizure reduction over 16 weeks, while secondary outcomes evaluate seizure frequency, seizure-free days, and clinical impressions of improvement. Participants are followed for up to approximately 172 weeks, ensuring long-term safety and efficacy data are collected.

A Study of EPX-100 (Clemizole Hydrochloride) in Participants With Dravet Syndrome