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New hope for dravet syndrome: phase 3 trial of EPX-100 aims to cut seizures

NCT ID NCT04462770

Disease control Sponsor: Epygenix Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Aug 14, 2026 · Updated 4 times

Summary

This study tests an experimental drug called EPX-100 (clemizole) in 150 children and adults with Dravet syndrome, a severe form of epilepsy. The goal is to see if adding EPX-100 to current treatments can safely reduce the number of motor seizures. Participants will be randomly assigned to receive either the drug or a placebo for 20 weeks.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 150 people

The number the study aims to enrol. It can still change while the study runs.

Started

Sep 2020

Expected to finish

May 2029

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

2 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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 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. 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 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.

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Conditions

The condition(s) this trial relates to.

Dravet syndrome Epilepsies, Myoclonic visual epilepsy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Dravet syndrome

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    55 sites in 9 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

  • Contact

    Email: •••••@•••••

Locations

  • All India Institute of Medical Sciences (AIIMS)

    RECRUITING

    New Delhi, National Capital Territory of Delhi, 110029, India

  • Amrita Institute of Medical Sciences & Research Center

    RECRUITING

    Kochi, Kerala, 682041, India

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

    TERMINATED

    Chicago, Illinois, 60611, United States

  • Arkansas Children's Hospital

    RECRUITING

    Little Rock, Arkansas, 72202, United States

  • Cardiff and Vale University Health Board

    ACTIVE_NOT_RECRUITING

    Cardiff, CF14 4XW, United Kingdom

  • Central India Cardiology Hospital and Research Institute

    RECRUITING

    Nagpur, Maharashtra, 440015, India

  • Child Neurology Consultants of Austin

    RECRUITING

    Austin, Texas, 78757, United States

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

    RECRUITING

    Ottawa, Ontario, K1H 8L1, Canada

  • Children's Hospital of Los Angeles

    TERMINATED

    Los Angeles, California, 90027, United States

  • Children's Nebraska

    RECRUITING

    Omaha, Nebraska, 68114, United States

  • Christian Medical College

    RECRUITING

    Vellore, Tamil Nadu, 632004, India

    Contact Email: •••••@•••••

  • Cincinnati Children's Hospital Medical Center

    RECRUITING

    Cincinnati, Ohio, 45229, United States

  • Cleveland Clinic

    RECRUITING

    Cleveland, Ohio, 44195, United States

  • Clinical Integrative Research Center of Atlanta (CIRCA)

    WITHDRAWN

    Atlanta, Georgia, 30328, United States

  • Conci Carpinella - Santa Rosa N° 748

    NOT_YET_RECRUITING

    Córdoba, Argentina

  • Duke University Health System

    RECRUITING

    Durham, North Carolina, 27710, United States

  • Fundacao Faculdade Regional de Medicina de Sao Jose Do Rio Preto Hospital de Base - PPDS

    NOT_YET_RECRUITING

    São José do Rio Preto, Brazil

  • Fundación Para La Lucha Contra Las Enfermedades Neurológicas de La Infancia - FLENI

    NOT_YET_RECRUITING

    Buenos Aires, Argentina

  • Great Ormond Street Hospital For Children

    ACTIVE_NOT_RECRUITING

    London, WC1N 1EH, United Kingdom

  • Hospital Infantil Universitario Niño Jesús

    RECRUITING

    Madrid, Madrid Provincia, 28009, Spain

  • Hospital São Paulo da Universidade Federal de Sao Paulo - Escola Paulista de Medicina UNIFESP-EPM

    NOT_YET_RECRUITING

    São Paulo, Brazil

  • Hospital Universitário da Universidade Estadual de Campinas - UNICAMP

    NOT_YET_RECRUITING

    Campinas, Brazil

  • Hospital de la Santa Creu i Sant Pau

    RECRUITING

    Barcelona, 08025, Spain

  • Institute of Mother and Child

    RECRUITING

    Warsaw, 01-211, Poland

  • Institute of Neurology and Neuropsychology LTD

    RECRUITING

    Tbilisi, 0186, Georgia

  • Instituto Médico Rivarola (GIEN SRL)

    NOT_YET_RECRUITING

    San Luis, Argentina

  • Instituto Privado Kremer

    NOT_YET_RECRUITING

    Córdoba, Argentina

  • Instituto de Psiquiatria HC FMUSP

    NOT_YET_RECRUITING

    São Paulo, Brazil

  • Jaslok Hospital & Research Centre

    RECRUITING

    Mumbai, Maharashtra, 400026, India

  • KEM Hospital Research Centre

    NOT_YET_RECRUITING

    Pune, Maharashta, 411011, India

  • KLES Dr Prabhakar Kore Hospital and Medical Research Centre

    NOT_YET_RECRUITING

    Belagavi, Karnataka, 590010, India

  • Le Bonheur Children's Hospital

    TERMINATED

    Memphis, Tennessee, 38103, United States

  • Medi Club Georgia LLC

    RECRUITING

    Tbilisi, 0160, Georgia

  • Medical Centre Plejady

    RECRUITING

    Krakow, 30-363, Poland

  • Medical College of Wisconsin, Children's Wisconsin

    NOT_YET_RECRUITING

    Milwaukee, Wisconsin, 53266, United States

  • National Institute of Mental Health and Neurosciences (NIMHANS)

    NOT_YET_RECRUITING

    Bengaluru, Karnataka, 560029, India

  • Nationwide Children's Hospital

    WITHDRAWN

    Columbus, Ohio, 43205, United States

  • Neurology Center for Epilepsy and Seizures

    ACTIVE_NOT_RECRUITING

    Marlboro, New Jersey, 07746, United States

  • Northeast Regional Epilepsy Group

    TERMINATED

    Hackensack, New Jersey, 07601, United States

  • Northwell Health - Lenox Hill Hospital

    NOT_YET_RECRUITING

    New York, New York, 10075, United States

  • Norton Children's Research Institute

    RECRUITING

    Louisville, Kentucky, 40202, United States

  • Pediatric Neurology and Epilepsy Specialists

    RECRUITING

    Winter Park, Florida, 32789, United States

  • Rainbow Children's Hospital

    NOT_YET_RECRUITING

    Hyderabad, Andhra Pradesh, 500034, India

  • Rare Disease Research FL

    RECRUITING

    Kissimmee, Florida, 34746, United States

  • SRCC Children's Hospital

    RECRUITING

    Mahālakshmi, Mumbai, 400034, India

  • Saachi Children's Hospital

    RECRUITING

    Surat, Gujarat, 395002, India

  • Science 37

    ACTIVE_NOT_RECRUITING

    Morrisville, North Carolina, 27560, United States

  • Seattle Children's Hospital

    RECRUITING

    Seattle, Washington, 98105, United States

  • Semmelweis University

    RECRUITING

    Budapest, 1094, Hungary

  • Sheffield Children's Hospital

    ACTIVE_NOT_RECRUITING

    Sheffield, S102TH, United Kingdom

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

    RECRUITING

    Tbilisi, 0159, Georgia

  • The Children's Hospital of Philadelphia

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

  • The Hospital for Sick Children

    RECRUITING

    Toronto, Ontario, M5G 1X8, Canada

  • The Nemours Foundation

    TERMINATED

    Wilmington, Delaware, 19803, United States

  • Toronto Western Hospital, University Health Network

    RECRUITING

    Toronto, Ontario, M5T 2S8, Canada

  • UBC Children's Hospital Research Institute

    RECRUITING

    Vancouver, British Columbia, V6H 3V1, Canada

  • UCSF Medical Center

    RECRUITING

    San Francisco, California, 94158, United States

  • UT Southwestern/Children's Health

    WITHDRAWN

    Dallas, Texas, 75207, United States

  • University Clinical Center in Gdansk, Division of Developmental Neurology

    RECRUITING

    Gdansk, 80-952, Poland

  • University of Alabama at Birmingham

    RECRUITING

    Birmingham, Alabama, 35233, United States

  • University of California Irvine

    RECRUITING

    Orange, California, 92868, United States

  • University of Debrecen

    RECRUITING

    Debrecen, 4032, Hungary

  • University of Michigan- Mott Children's Hospital

    TERMINATED

    Ann Arbor, Michigan, 48109, United States

  • University of Utah

    RECRUITING

    Salt Lake City, Utah, 84112, United States

  • Wake Forest Baptist Medical Center

    RECRUITING

    Winston-Salem, North Carolina, 27157, United States

  • Weill Cornell Medical Center

    RECRUITING

    New York, New York, 10021, United States

  • Yale School of Medicine - Yale-New Haven Hospital

    RECRUITING

    New Haven, Connecticut, 06511, United States

  • Yale University School of Medicine

    NOT_YET_RECRUITING

    New Haven, Connecticut, 06510, United States

  • Yashoda Hospitals Hitec City

    NOT_YET_RECRUITING

    Hyderabad, Andhra Pradesh, 500081, India

    Contact Email: •••••@•••••

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