Actively Recruiting

Phase 3
Age: 2Years +
All Genders
NCT04462770

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

Led by Epygenix · Updated on 2026-05-14

150

Participants Needed

58

Research Sites

450 weeks

Total Duration

On this page

Sponsors

E

Epygenix

Lead Sponsor

H

Harmony Biosciences Management, Inc.

Collaborating Sponsor

AI-Summary

What this Trial Is About

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).

CONDITIONS

Official Title

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

Who Can Participate

Age: 2Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Male and female participants 2 years and older at time of consent
  • Participant or parent/legal representative able to provide written informed consent or assent
  • Clinical diagnosis of Dravet syndrome with seizures not fully controlled by anti-epileptic drugs
  • Seizure onset before 18 months of age
  • Normal development at seizure onset
  • History of at least one type of countable motor seizure
  • Brain MRI without cortical malformation (except mild atrophy related to Dravet syndrome progression)
  • Documented SCN1A gene mutation
Not Eligible

You will not qualify if you...

  • Known allergy, sensitivity, or prior exposure to clemizole hydrochloride
  • Participation in another investigational drug or device study within 90 days prior to screening or planned during this study
  • Seizures caused by illicit drug or alcohol use, infection, tumor, demyelinating disease, degenerative neurological disease, progressive CNS disease, metabolic illness, or other progressive degenerative diseases
  • Current use of lorcaserin (prior use allowed if last dose was at least 30 days ago)
  • Current use of fenfluramine
  • Epilepsy surgery planned during the study or performed within 6 months before screening

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 58 locations

1

University of Alabama at Birmingham

Birmingham, Alabama, United States, 35233

Active, Not Recruiting

2

Arkansas Children's Hospital

Little Rock, Arkansas, United States, 72202

Active, Not Recruiting

3

Children's Hospital of Los Angeles

Los Angeles, California, United States, 90027

Terminated

4

University of California Irvine

Orange, California, United States, 92868

Actively Recruiting

5

UCSF Medical Center

San Francisco, California, United States, 94158

Actively Recruiting

6

Yale University School of Medicine

New Haven, Connecticut, United States, 06510

Not Yet Recruiting

7

The Nemours Foundation

Wilmington, Delaware, United States, 19803

Terminated

8

Rare Disease Research FL

Kissimmee, Florida, United States, 34746

Actively Recruiting

9

Pediatric Neurology and Epilepsy Specialists

Winter Park, Florida, United States, 32789

Actively Recruiting

10

Clinical Integrative Research Center of Atlanta (CIRCA)

Atlanta, Georgia, United States, 30328

Withdrawn

11

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

Chicago, Illinois, United States, 60611

Terminated

12

Norton Children's Research Institute

Louisville, Kentucky, United States, 40202

Actively Recruiting

13

University of Michigan- Mott Children's Hospital

Ann Arbor, Michigan, United States, 48109

Terminated

14

Children's Nebraska

Omaha, Nebraska, United States, 68114

Actively Recruiting

15

Northeast Regional Epilepsy Group

Hackensack, New Jersey, United States, 07601

Terminated

16

Neurology Center for Epilepsy and Seizures

Marlboro, New Jersey, United States, 07746

Active, Not Recruiting

17

Weill Cornell Medical Center

New York, New York, United States, 10021

Actively Recruiting

18

Northwell Health - Lenox Hill Hospital

New York, New York, United States, 10075

Not Yet Recruiting

19

Duke University Health System

Durham, North Carolina, United States, 27710

Actively Recruiting

20

Science 37

Morrisville, North Carolina, United States, 27560

Active, Not Recruiting

21

Wake Forest Baptist Medical Center

Winston-Salem, North Carolina, United States, 27157

Actively Recruiting

22

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States, 45229

Actively Recruiting

23

Cleveland Clinic

Cleveland, Ohio, United States, 44195

Actively Recruiting

24

Nationwide Children's Hospital

Columbus, Ohio, United States, 43205

Withdrawn

25

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States, 19104

Actively Recruiting

26

Le Bonheur Children's Hospital

Memphis, Tennessee, United States, 38103

Terminated

27

Child Neurology Consultants of Austin

Austin, Texas, United States, 78757

Actively Recruiting

28

UT Southwestern/Children's Health

Dallas, Texas, United States, 75207

Withdrawn

29

University of Utah

Salt Lake City, Utah, United States, 84112

Actively Recruiting

30

Seattle Children's Hospital

Seattle, Washington, United States, 98105

Actively Recruiting

31

Medical College of Wisconsin, Children's Wisconsin

Milwaukee, Wisconsin, United States, 53266

Not Yet Recruiting

32

UBC Children's Hospital Research Institute

Vancouver, British Columbia, Canada, V6H 3V1

Actively Recruiting

33

Children's Hospital of Eastern Ontario Research Institute Inc.

Ottawa, Ontario, Canada, K1H 8L1

Actively Recruiting

34

The Hospital for Sick Children

Toronto, Ontario, Canada, M5G 1X8

Actively Recruiting

35

Toronto Western Hospital, University Health Network

Toronto, Ontario, Canada, M5T 2S8

Actively Recruiting

36

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

Tbilisi, Georgia, 0159

Actively Recruiting

37

Medi Club Georgia LLC

Tbilisi, Georgia, 0160

Actively Recruiting

38

Institute of Neurology and Neuropsychology LTD

Tbilisi, Georgia, 0186

Actively Recruiting

39

Semmelweis University

Budapest, Hungary, 1085

Actively Recruiting

40

University of Debrecen

Debrecen, Hungary, 4032

Actively Recruiting

41

Saachi Children's Hospital

Surat, Gujarat, India, 395002

Active, Not Recruiting

42

National Institute of Mental Health and Neurosciences (NIMHANS)

Adugodi, Karnatka, India, 560029

Not Yet Recruiting

43

Central India Cardiology Hospital and Research Institute

Nagpur, Maharashtra, India, 44015

Active, Not Recruiting

44

Christian Medical College

Vellore, Tamil Nadu, India, 632004

Not Yet Recruiting

45

Rainbow Children's Hospital

Hyderabad, India, 500034

Not Yet Recruiting

46

Amrita Institute of Medical Sciences & Research Center

Kochi, India, 682041

Not Yet Recruiting

47

Jaslok Hospital & Research Centre

Mumbai, India, 400026

Not Yet Recruiting

48

SRCC Children's Hospital by NH

Mumbai, India, 400034

Not Yet Recruiting

49

All India Institute of Medical Sciences

New Delhi, India

Not Yet Recruiting

50

University Clinical Center in Gdansk, Division of Developmental Neurology

Gdansk, Poland

Actively Recruiting

51

Medical Centre Plejady

Krakow, Poland, 30-363

Actively Recruiting

52

Institute of Mother and Child

Warsaw, Poland, 01-211

Actively Recruiting

53

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

Bucharest, Romania, 041914

Not Yet Recruiting

54

Hospital Infantil Universitario Niño Jesús

Madrid, Madrid Provincia, Spain, 28009

Actively Recruiting

55

Hospital de la Santa Creu i Sant Pau

Barcelona, Spain, 08025

Actively Recruiting

56

Cardiff and Vale University Health Board

Cardiff, United Kingdom, CF14 4XW

Actively Recruiting

57

Great Ormond Street Hospital For Children

London, United Kingdom, WC1N 1EH

Actively Recruiting

58

Sheffield Children's Hospital

Sheffield, United Kingdom, S102TH

Actively Recruiting

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Research Team

K

Krystle Rapchak

CONTACT

E

Eric Bauer

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

TRIPLE

Allocation

RANDOMIZED

Model

PARALLEL

Primary Purpose

TREATMENT

Number of Arms

3

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