Loading clinical trials...

RECRUITINGPHASE3

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

A 20-Week Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial of EPX-100 (Clemizole Hydrochloride) as Adjunctive Therapy in Children and Adult Participants With Dravet Syndrome (ARGUS Trial)

NCT04462770•Epygenix

View on ClinicalTrials.gov

Summary

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).

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 for up to 156 weeks.

Eligibility

Age

2 - No limit years

Sex

ALL

Healthy Volunteers

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, 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.

Locations (46)

Children's Hospital of Los Angeles

Los Angeles, California, United States

University of California Irvine

Orange, California, United States

UCSF Medical Center

San Francisco, California, United States

Yale University School of Medicine

New Haven, Connecticut, United States

The Nemours Foundation

Wilmington, Delaware, United States

Rare Disease Research FL

Kissimmee, Florida, United States

Pediatric Neurology and Epilepsy Specialists

Winter Park, Florida, United States

Clinical Integrative Research Center of Atlanta (CIRCA)

Atlanta, Georgia, United States

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, United States

Norton Children's Research Institute

Louisville, Kentucky, United States

Key Dates

Start Date

September 15, 2020

Primary Completion Date

April 1, 2026

Completion Date

May 1, 2029

Last Updated

August 28, 2025

Enrollment

150

ESTIMATED participants

Conditions

Dravet Syndrome

Interventions

Clemizole HCl

DRUG

Placebo

DRUG

Contacts

Krystle Rapchak

CONTACT

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

Eric Bauer

CONTACT

clinicaltrials@harmonybiosciences.com

A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and Children With SCN1A-Positive Dravet Syndrome

NCT05419492

Dravet Syndrome

View study

RECRUITINGPHASE4

Assessment of Safety of the Use of Fenfluramine in Children With Dravet Syndrome Under 24 Months of Age

NCT06598449

Dravet Syndrome (DS)Children Under 2 Years

View study

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: August 28, 2025Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

View ClinicalTrials.gov Terms and Conditions

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

Inclusion Criteria

Exclusion Criteria

A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and Children With SCN1A-Positive Dravet Syndrome

Assessment of Safety of the Use of Fenfluramine in Children With Dravet Syndrome Under 24 Months of Age

A Study to Investigate the Transition of Children From 'Artisanal" Cannabidiol (CBD) to Epidiolex

A Study to Investigate the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution in Children and Adults With Epileptic Encephalopathy Including Dravet Syndrome and Lennox-Gastaut Syndrome

Bioavailability of Stiripentol After Single Oral Dose of Capsule vs Suspension in Healthy Subjects (STILIQ)

A Study to Gather Information About Overall Occurrence and New Cases of Dravet and Lennox-Gastaut Syndromes in Children, Teenagers and Adults in Spain