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

Last updated: May 13, 2025
Sponsor: Epygenix
Overall Status: Active - Recruiting

Phase

3

Condition

Seizure Disorders (Pediatric)

Unverricht-lundborg Syndrome

Dravet Syndrome

Treatment

EPX-100 (clemizole HCl)

EPX-100 (Clemizole HCl)

Clemizole HCl

Clinical Study ID

NCT04462770
EPX-100-001
  • Ages > 2
  • All Genders

Study Summary

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

Eligibility Criteria

Inclusion

Key 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 toprovide written informed consent, assent (if applicable) prior to initiation of anystudy related procedures.

  3. Clinical diagnosis of Dravet syndrome. Participants must have seizures which are notcompletely 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 associatedwith the natural progression of Dravet syndrome),

  • Genetic mutation of the SCN1A gene must be documented.

Exclusion

Key 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 plansto participate in another drug or device trial at any time during the study.

  3. Seizures secondary to illicit drug (this includes concomitant use oftetrahydrocannabinol [THC] and nonprescription cannabidiol preparations) or alcoholuse, infection, neoplasm, demyelinating disease, degenerative neurological disease,or central nervous system (CNS) disease deemed progressive, metabolic illness, orprogressive degenerative disease.

  4. Prior or concurrent use of lorcaserin.

  5. Concurrent use of fenfluramine.

  6. Epilepsy surgery planned during the study or epilepsy surgery within 6 months priorto Screening.

Study Design

Total Participants: 150
Treatment Group(s): 4
Primary Treatment: EPX-100 (clemizole HCl)
Phase: 3
Study Start date:
September 15, 2020
Estimated Completion Date:
May 01, 2029

Study Description

This is a global multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of clemizole HCl as adjunctive therapy in children and adult participants with Dravet syndrome. 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.

Connect with a study center

  • Children's and Women's Health Centre of British Columbia

    Vancouver, British Columbia V6H 3V4
    Canada

    Active - Recruiting

  • UBC Children's Hospital Research Institute

    Vancouver, British Columbia V6H 3V4
    Canada

    Active - Recruiting

  • Children's Hospital of Eastern Ontario

    Ottawa, Ontario K1H 8L1
    Canada

    Active - Recruiting

  • Children's Hospital of Eastern Ontario Research Institute Inc.

    Ottawa, Ontario K1H 8L1
    Canada

    Active - Recruiting

  • The Hospital for Sick Children

    Toronto, Ontario M5G 1X8
    Canada

    Active - Recruiting

  • Toronto Western Hospital, University Health Network

    Toronto, Ontario M5T 2S8
    Canada

    Active - Recruiting

  • Institute of Neurology and Neuropsychology

    Tbilisi, 0186
    Georgia

    Active - Recruiting

  • LEPL Tbilisi State Medical University, Givi Zhvania Academic, Clinic of Pediatry

    Tbilisi, 0159
    Georgia

    Active - Recruiting

  • LTD Institute of Neurology and Neuropsychology

    Tbilisi, 0186
    Georgia

    Active - Recruiting

  • LTD Medi Club Georgia LLC

    Tbilisi, 0160
    Georgia

    Site Not Available

  • Medi Club Georgia LLC

    Tbilisi, 0160
    Georgia

    Active - Recruiting

  • Tbilisi State Medical University, Givi Zhvania Academic, Clinic of Pediatry

    Tbilisi, 0159
    Georgia

    Active - Recruiting

  • Semmelweis University

    Budapest, 1085
    Hungary

    Active - Recruiting

  • Semmelweis University First Department of Pediatrics, Division of Child Neurology

    Budapest, 1083
    Hungary

    Site Not Available

  • University of Debrecen

    Debrecen, 4032
    Hungary

    Active - Recruiting

  • University Clinical Center in Gdansk, Division of Developmental Neurology

    Gdańsk,
    Poland

    Site Not Available

  • Medical Centre Plejady

    Kraków, 30-363
    Poland

    Active - Recruiting

  • Institute of Mother and Child

    Warsaw, 01-211
    Poland

    Active - Recruiting

  • "Prof. Dr. Al. Obregia" Psychiatry Clinical Hospital

    Bucharest, 041914
    Romania

    Site Not Available

  • Hospital Universitario Infantil Niño Jesús

    Madrid, Madrid Provincia 28009
    Spain

    Active - Recruiting

  • Hospital Infantil Universitario Niño Jesús

    Madrid, Madrid provincia 28009
    Spain

    Active - Recruiting

  • Hospital de la Santa Creu i Sant Pau

    Barcelona, 08025
    Spain

    Active - Recruiting

  • Cardiff and Vale University Health Board

    Cardiff, CF14 4XW
    United Kingdom

    Site Not Available

  • Alder Hey Children's NHS Foundation Trust

    Liverpool, L12 2AP
    United Kingdom

    Site Not Available

  • Great Ormond Street Hospital For Children

    London, WC1N 1EH
    United Kingdom

    Active - Recruiting

  • Sheffield Children's Hospital

    Sheffield, S102TH
    United Kingdom

    Active - Recruiting

  • Children's Hospital of Los Angeles

    Los Angeles, California 90027
    United States

    Terminated

  • University of California Irvine

    Orange, California 92868
    United States

    Site Not Available

  • UCSF Medical Center

    San Francisco, California 94158
    United States

    Active - Recruiting

  • Nemours Children's Hospital-DE

    Wilmington, Delaware 19803
    United States

    Active - Recruiting

  • The Nemours Foundation

    Wilmington, Delaware 19803
    United States

    Active - Recruiting

  • Rare Disease Research FL

    Kissimmee, Florida 34746
    United States

    Site Not Available

  • Pediatric Neurology and Epilepsy Specialists

    Winter Park, Florida 32789
    United States

    Site Not Available

  • Clinical Integrative Research Center of Atlanta (CIRCA)

    Atlanta, Georgia 30328
    United States

    Site Not Available

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

    Chicago, Illinois 60611
    United States

    Site Not Available

  • Norton Children's Research Institute

    Louisville, Kentucky 40202
    United States

    Site Not Available

  • University of Michigan- Mott Children's Hospital

    Ann Arbor, Michigan 48109
    United States

    Site Not Available

  • Children's Hospital & Medical Center

    Omaha, Nebraska 68114
    United States

    Site Not Available

  • Children's Nebraska

    Omaha, Nebraska 68114
    United States

    Active - Recruiting

  • NorthEast Epilepsy Group

    Hackensack, New Jersey 07601
    United States

    Site Not Available

  • Northeast Regional Epilepsy Group

    Hackensack, New Jersey 07601
    United States

    Terminated

  • Neurology Center for Epilepsy and Seizures

    Marlboro, New Jersey 07746
    United States

    Site Not Available

  • Weill Cornell Medical Center

    New York, New York 10021
    United States

    Active - Recruiting

  • Weill Cornell Medicine/New York-Presbyterian Hospital

    New York, New York 10065
    United States

    Site Not Available

  • OnSite Clinical Solutions, LLC

    Charlotte, North Carolina 28211
    United States

    Site Not Available

  • Duke University Health System

    Durham, North Carolina 27710
    United States

    Site Not Available

  • Wake Forest Baptist Health Sciences Department of Neurology

    Winston-Salem, North Carolina 27157
    United States

    Active - Recruiting

  • Wake Forest Baptist Medical Center

    Winston-Salem, North Carolina 27157
    United States

    Active - Recruiting

  • Cincinnati Children's Hospital

    Cincinnati, Ohio 45229
    United States

    Active - Recruiting

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio 45229
    United States

    Active - Recruiting

  • Cleveland Clinic

    Cleveland, Ohio 44195
    United States

    Active - Recruiting

  • Nationwide Children's Hospital

    Columbus, Ohio 43205
    United States

    Site Not Available

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania 19104
    United States

    Active - Recruiting

  • The Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania 19104
    United States

    Active - Recruiting

  • Le Bonheur Children's Hospital

    Memphis, Tennessee 38103
    United States

    Terminated

  • Child Neurology Consultants of Austin

    Austin, Texas 78757
    United States

    Active - Recruiting

  • UT Southwestern/Children's Health

    Dallas, Texas 75207
    United States

    Site Not Available

  • University of Utah

    Salt Lake City, Utah 84112
    United States

    Active - Recruiting

  • Seattle Children's

    Seattle, Washington 98105
    United States

    Active - Recruiting

  • Seattle Children's Hospital

    Seattle, Washington 98105
    United States

    Active - Recruiting

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