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

Purpose

This is a multicenter, Phase 3, randomized, double-blind, placebo-controlled study designed to evaluate the efficacy and safety of clemizole hydrochloride (EPX-100) as adjunctive therapy in children and adult participants with Dravet syndrome (DS).

Condition

  • Dravet Syndrome

Eligibility

Eligible Ages
Over 2 Years
Eligible Sex
All
Accepts Healthy Volunteers
No

Inclusion Criteria

  1. Male and female participants 2 years and older at time of consent. 2. Participant or parent/legally authorized representative (LAR) willing and able to provide written informed consent and assent (if applicable) prior to initiation of any study related procedures. 3. Clinical diagnosis of DS. Participants must have seizures which are not completely controlled by AEDs with the following criteria: - Onset of seizures prior to 18 months of age, - Normal development at onset, - History of at least one type of countable motor seizure (CMS), - Brain MRI without cortical malformation (not including mild atrophy associated with the natural progression of DS), - Genetic mutation of the SCN1A gene must be documented.

Exclusion Criteria

  1. Known sensitivity, allergy, or previous exposure to clemizole HCl. 2. Exposure to any investigational drug or device <90 days prior to screening or plans to participate in another drug or device trial at any time during the study. 3. Seizures secondary to illicit drug (this includes concomitant use of tetrahydrocannabinol [THC] and nonprescription cannabidiol preparations) or alcohol use, infection, neoplasm, demyelinating disease, degenerative neurological disease, or central nervous system disease deemed progressive, metabolic illness, or progressive degenerative disease. 4. Concurrent use of lorcaserin. Note: Prior use of lorcaserin is permitted if at least 30 days have passed since the last dose. 5. Concurrent use of fenfluramine. 6. Epilepsy surgery planned during the study or epilepsy surgery within 6 months prior to Screening.

Study Design

Phase
Phase 3
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Intervention Model Description
Patients are randomized 1:1 to clemizole HCl (EPX-100) or placebo.
Primary Purpose
Treatment
Masking
Triple (Participant, Care Provider, Investigator)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
Double-blind clemizole HCl 		Participants will receive their first dose of study drug following randomization.
  • Drug: Clemizole HCl
    Clemizole HCl will be administered as an oral solution.
    Other names:
    • EPX-100
Placebo Comparator
Placebo 		Participants will receive their first dose of study drug following randomization.
  • Drug: Placebo
    Placebo will be administered as an oral solution.
Experimental
Open-label clemizole HCl 		Eligible participants who complete the DB Period will have the option to continue in the OLE Period, during which they will receive clemizole HCL for up to 3 years.
  • Drug: Clemizole HCl
    Clemizole HCl will be administered as an oral solution.
    Other names:
    • EPX-100

Recruiting Locations

University of Alabama at Birmingham
Birmingham, Alabama 35233
Contact:
Kathryn Lalor, MD
205-586-4464
klalor@uabmc.edu

More Details

Status
Recruiting
Sponsor
Epygenix

Study Contact

Krystle Rapchak
+1 (312) 847-1289
clinicaltrials@harmonybiosciences.com

Detailed Description

This is a global, multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of clemizole hydrochloride as adjunctive therapy in children and adult participants with DS. The study consists of a 4-week Observational Period, a 16-week Double-Blind (DB) Period and an Open-Label Extension (OLE) Period.