Dravet SyndromePhase 3ARGUSNCT04462770
A Study of EPX-100 (Clemizole Hydrochloride) in Participants With Dravet Syndrome
A Phase 3 study testing Clemizole HCl in people with Dravet Syndrome, run by Epygenix at 68 sites in 10 countries.
Trial 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). Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Dravet Syndrome
- What is being tested?
- Clemizole HCl
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 150 participants
- Where
- 68 sitesin 10 countries
- Study dates
- 15 Sep 2020to May 2029
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male and female participants 2 years and older at time of consent.
- 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.
- 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.
Who cannot join
- Known sensitivity, allergy, or previous exposure to clemizole HCl.
- 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.
- 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.
- Concurrent use of lorcaserin. Note: Prior use of lorcaserin is permitted if at least 30 days have passed since the last dose.
- Concurrent use of fenfluramine.
- Epilepsy surgery planned during the study or epilepsy surgery within 6 months prior to Screening.
Trial locations and contacts
68 sites in 10 countries. Contact the study team or a site near you to ask about taking part.
Eric Bauer contact
68 sites in 10 countries
United States31 sites, 18 recruiting
- Terminated
University of Michigan- Mott Children's Hospital
Ann Arbor, Michigan, 48109
- Withdrawn
Clinical Integrative Research Center of Atlanta (CIRCA)
Atlanta, Georgia, 30328
- Recruiting
Child Neurology Consultants of Austin
Austin, Texas, 78757
Victoria Henderson, BS, 210-416-4163, Victoria.Henderson@childneurotx.com
Talitha Durgin, talitha.durgin@childneurotx.com
- Recruiting
University of Alabama at Birmingham
Birmingham, Alabama, 35233
Kathryn Lalor, MD, 205-586-4464, klalor@uabmc.edu
Kathryn Lalor, MD
- Terminated
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
- Recruiting
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Samantha Ballard, BSN, RN, CPN, 513-803-3177, samantha.ballard@cchmc.org
Jessica Michael, jessica.michael@cchmc.org
- Recruiting
Cleveland Clinic
Cleveland, Ohio, 44195
Honglian Huang, PhD, 216-445-2366, huangh2@ccf.org
Elia Pestana Knight, MD
- Withdrawn
Nationwide Children's Hospital
Columbus, Ohio, 43205
- Withdrawn
UT Southwestern/Children's Health
Dallas, Texas, 75207
- Recruiting
Duke University Health System
Durham, North Carolina, 27710
Amanda Fristoe, 919-613-0767, amanda.fristoe@duke.edu
Muhammad Zafar, MD
- Withdrawn
Northeast Regional Epilepsy Group
Hackensack, New Jersey, 07601
- Terminated
Rare Disease Research FL
Kissimmee, Florida, 34746
- Recruiting
Arkansas Children's Hospital
Little Rock, Arkansas, 72202
Debopam Samanta, MD, 501-364-1850, dsamanta@uams.edu
Debopam Samanta, MD
- Terminated
Children's Hospital of Los Angeles
Los Angeles, California, 90027
- Recruiting
Norton Children's Research Institute
Louisville, Kentucky, 40202
Katie Harris, 502-629-5820 ext. 50920, katherine.bellamy@louisville.edu
Ines Roy, ines.roy@nortonhealthcare.org
- Recruiting
Neurology Center for Epilepsy and Seizures
Marlboro, New Jersey, 07746
Aimee Kinlan, 732-201-6179, Amy.Kinlan@tektonresearch.com
Amor Mehta, MD
- Terminated
Le Bonheur Children's Hospital
Memphis, Tennessee, 38103
- Not yet recruiting
Medical College of Wisconsin, Children's Wisconsin
Milwaukee, Wisconsin, 53266
Adriane Mueller, 414-266-7543, admueller@mcw.edu
Avantika Singh
- Recruiting
Science 37
Morrisville, North Carolina, 27560
- Recruiting
Yale School of Medicine - Yale-New Haven Hospital
New Haven, Connecticut, 06511
Aline Herlopian, MD
- Terminated
Weill Cornell Medical Center
New York, New York, 10021
- Withdrawn
Northwell Health - Lenox Hill Hospital
New York, New York, 10075
- Recruiting
Children's Nebraska
Omaha, Nebraska, 68114
Kym Abraham, 402-559-2977, kabraham@unmc.edu
Malayna Unkel, 402-559-0686
- Recruiting
University of California Irvine
Orange, California, 92868
Madeline Pak, 714-456-8413, mspak@hs.uci.edu
Marie Wencel, mwencel@hs.usci.edu
- Recruiting
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Rashmi Risbud, Mbio, 215-419-3212, risbudr@chop.edu
Eric Marsh, MD, PhD
- Recruiting
University of Utah
Salt Lake City, Utah, 84112
Laura Beeler, BS, 801-585-2121, laura.beeler@hsc.utah.edu
Daniel Ashman, daniel.ashman@hsc.utah.edu
- Terminated
UCSF Medical Center
San Francisco, California, 94158
- Recruiting
Seattle Children's Hospital
Seattle, Washington, 98105
Laurie Guidry, RN, 206-987-0058, laurie.guidry@seattlechildrens.org
Alex Collins, alex.collins@seattlechildrens.org
- Terminated
The Nemours Foundation
Wilmington, Delaware, 19803
- Recruiting
Wake Forest Baptist Medical Center
Winston-Salem, North Carolina, 27157
Tonya Woosley, 336-716-8940, twoosley@wakehealth.edu
Gautam S Popli, MD, MBA
- Recruiting
Pediatric Neurology and Epilepsy Specialists
Winter Park, Florida, 32789
Alex Rivera, 407-293-1122 ext. 121, ariveradelvalle@pediatricneurologyandepilepsy.com
Kathryn Bruner, kathryn.skilesbruner@pediatricneurologyandepilepsy.com
India12 sites, 12 recruiting
- Not yet recruiting
KLES Dr Prabhakar Kore Hospital and Medical Research Centre
Belagavi, Karnataka, 590010
Jyoti Guruputranavar, +91- 76195 48224, guruputranavarjyoti@gmail.com
Mahesh Kamate
- Not yet recruiting
National Institute of Mental Health and Neurosciences (NIMHANS)
Bengaluru, Karnataka, 560029
Ajay Asranna, 91-8943612626, ajayasranna@gmail.com
Ajay Asranna
- Not yet recruiting
Rainbow Children's Hospital
Hyderabad, Andhra Pradesh, 500034
Ramesh Konanki, 7386668959, drkonankiramesh@rainbowhospitals.in
Ramesh Konanki
- Not yet recruiting
Yashoda Hospitals Hitec City
Hyderabad, Andhra Pradesh, 500081
Shameer Basha Shaik, +91- 9966596459, research2.bnj@rainbowhospitals.in
Katakam Deepthi, researchneuroscience3@gmail.com
- Recruiting
Amrita Institute of Medical Sciences & Research Center
Kochi, Kerala, 682041
Deepa Gijesh, +919995184399, deepagijesh@aims.amrita.edu
Nithya Kumar, nithyankumar2@gmail.com
- Recruiting
SRCC Children's Hospital
Mahālakshmi, Mumbai, 400034
Vijetha Shetty, +91- 8291207577, drgadgilexecutiveassistant@gmail.com
Pradnya Gadgil
- Recruiting
Jaslok Hospital & Research Centre
Mumbai, Maharashtra, 400026
Pradnya Shinde, +919757138607, pradnyashinde2210@gmail.com
Anita Hegde
- Recruiting
Central India Cardiology Hospital and Research Institute
Nagpur, Maharashtra, 440015
Krutika Satokar, +919359723117, Krutika@hrapl.in
Amarjeet Wagh
- Recruiting
All India Institute of Medical Sciences (AIIMS)
New Delhi, National Capital Territory of Delhi, 110029
Shailly Katoch, +919878507624, katochshailly@gmail.com
Sheffali Gulati
- Not yet recruiting
KEM Hospital Research Centre
Pune, Maharashta, 411011
Aishwarya Patil, +91- 98999476422, aishwaryauttampatil@gmail.com
Nandan Yardi
- Recruiting
Saachi Children's Hospital
Surat, Gujarat, 395002
Honey Patel, 73833-33348, honey.patel@visaferesearch.com
Ritesh Shah
- Recruiting
Christian Medical College
Vellore, Tamil Nadu, 632004
G Ponezhil, +918124235630, argustrialpnucmc@gmail.com
Maya Thomas
Argentina4 sites, 4 recruiting
- Not yet recruiting
Fundación Para La Lucha Contra Las Enfermedades Neurológicas de La Infancia - FLENI
Buenos Aires
Walter Silva, 5491138310315, whsilva@fleni.org.ar
Walter Silva
- Not yet recruiting
Conci Carpinella - Santa Rosa N° 748
Córdoba
Hector Martinez, 5493517473258, hectorariel_martinez@hotmail.com
Hector Martinez
- Not yet recruiting
Instituto Privado Kremer
Córdoba
Carlos Deabato, 0351 3303166, deabatoarg@gmail.com
Carlos Deabato
- Not yet recruiting
Instituto Médico Rivarola (GIEN SRL)
San Luis
Pablo Lopez, (54115) 239-6010, pabloadrian.lopez@swissmedical.com.ar
Pablo Lopez
Brazil4 sites, 4 recruiting
- Not yet recruiting
Hospital Universitário da Universidade Estadual de Campinas - UNICAMP
Campinas
Fernando Cendes, (55193) 521-7372, fcendes@unicamp.br
Fernando Cendes
- Not yet recruiting
Fundacao Faculdade Regional de Medicina de Sao Jose Do Rio Preto Hospital de Base - PPDS
São José do Rio Preto
Lucia Neves Marques, 55 17 99608-4244, luciahelenanevesmarques@gmail.com
Lucia Neves Marques
- Not yet recruiting
Hospital São Paulo da Universidade Federal de Sao Paulo - Escola Paulista de Medicina UNIFESP-EPM
São Paulo
Laura Maria Guilhoto, (55115) 579-2581, lauragui@gmail.com
Laura Maria Guilhoto
- Not yet recruiting
Instituto de Psiquiatria HC FMUSP
São Paulo
Kette Dualibi Ramos Valente, 551126616518, kette.valente@hc.fm.usp.br
Kette Dualibi Ramos Valente
Canada4 sites, 4 recruiting
- Recruiting
Children's Hospital of Eastern Ontario Research Institute Inc.
Ottawa, Ontario, K1H 8L1
Sarah Healy, MSc, 613-737-7600 ext. 1605, shealy@cheo.on.ca
Aristhana Kathiresu, AKathiresu@cheo.on.ca
- Recruiting
The Hospital for Sick Children
Toronto, Ontario, M5G 1X8
Laura MacDougall, PhD, CCRP, 416-813-7996, laura.macdougall@sickkids.ca
Elizabeth Donner, MD
- Recruiting
Toronto Western Hospital, University Health Network
Toronto, Ontario, M5T 2S8
Sarah Iskandarani, MD, 416-603-5800 ext. 5906, sarah.iskandarani@uhn.ca
Quratulain Zulfiqar, quaratulain.zulfiqar_ali@uhn.ca
- Recruiting
UBC Children's Hospital Research Institute
Vancouver, British Columbia, V6H 3V1
Dora Xiong, 604-875-2345 ext. 6728, dora.xiong@cw.bc.ca
Vanessa Diamond, MD, vanessa.diamond@cw.bc.ca
Georgia3 sites, 3 recruiting
- Recruiting
Tbilisi State Medical University, Givi Zhvania Academic, Clinic of Pediatry
Tbilisi, 0159
Tinatin Nadiradze, MD, +995558320502, t.nadiradze@tsmu.edu
Sophia Bakhtadze, MD
- Recruiting
Medi Club Georgia LLC
Tbilisi, 0160
Tamar Gachechiladze, MD, +995598357909, tamogachechiladze@yahoo.com
Gia Melikishvili, MD
- Recruiting
Institute of Neurology and Neuropsychology LTD
Tbilisi, 0186
Sopiko Digmelashvili, MD, +995551516066, sopikodigmelashvili@gmail.com
David Kvernadze, MD
Poland3 sites, 3 recruiting
- Recruiting
University Clinical Center in Gdansk, Division of Developmental Neurology
Gdansk, 80-952
Angelika Kamińska, +48 58 349 23 31, ankaminska@uck.gda.pl
Maria Mazuriewicz-Bełdzińska, MD
- Recruiting
Medical Centre Plejady
Krakow, 30-363
Anna Mackowicz, +48 50 107 68 35, anna.mackowicz@plejady.com.pl
Marta Żołnowska, MD
- Recruiting
Institute of Mother and Child
Warsaw, 01-211
Zuzanna Kuczer, +48608338848, zuzanna.kuczer@imid.med.pl
Elżbieta Stawicka, MD
United Kingdom3 sites
- Active, not recruiting
Cardiff and Vale University Health Board
Cardiff, CF14 4XW
- Active, not recruiting
Great Ormond Street Hospital For Children
London, WC1N 1EH
- Active, not recruiting
Sheffield Children's Hospital
Sheffield, S102TH
Hungary2 sites, 2 recruiting
- Recruiting
Semmelweis University
Budapest, 1094
Andrasne Korodi, +06208258208, Korodi.kata@gmail.com
Kristof Farkas, MD
- Recruiting
University of Debrecen
Debrecen, 4032
Monika Bessenyei, MD, +0036709450368, besenyei.monika@med.unideb.hu
Mónika Bessenyei, MD
Spain2 sites, 2 recruiting
- Recruiting
Hospital de la Santa Creu i Sant Pau
Barcelona, 08025
Angela Fraga, +34633160818, AFraga@santpau.cat
Marc Alegre, MAlegreH@santpau.cat
- Recruiting
Hospital Infantil Universitario Niño Jesús
Madrid, Madrid Provincia, 28009
Nuria Ventosino, +346620708323 ext. 7351, nuria.ventosino@salud.madrid.org
Victor Soto, MD
About this study
Official title: 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)
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).
Read the 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.
- Conditions
- Dravet Syndrome
- Being tested
- Clemizole HCl, Placebo
- Keywords
- Clemizole hydrochloride, Convulsive seizure, Pediatric epilepsy, Dravet syndrome
- Medical subject headings
- Epilepsies, Myoclonic, Seizures
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Clemizole HClDrug
Also called EPX-100
Clemizole HCl will be administered as an oral solution.
PlaceboDrug
Placebo will be administered as an oral solution.
| Study group | Role | What they receive |
|---|---|---|
| Double-blind clemizole HCl | Experimental | Participants will receive their first dose of study drug following randomization. Receives: Drug: Clemizole HCl |
| Placebo | Placebo comparator | Participants will receive their first dose of study drug following randomization. Receives: Drug: Placebo |
| Open-label clemizole HCl | Experimental | 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. Receives: Drug: Clemizole HCl |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent Change in Countable Motor Seizures Per 28 Days (CMS-28) in the Titration Plus Maintenance Periods Relative to Baseline
Percent change in CMS-28 from the Baseline Period through the end of the DB period.
Measured: From Baseline Period (Day 1) up to 16 weeks
European Union: Percent Change in Countable Motor Seizures Per 28 Days in the Maintenance Period Relative to Baseline
Percent change in CMS-28 from the Baseline Period through the end of the maintenance period.
Measured: From maintenance period Baseline (Day 29) up to Day 85
Secondary outcomes (12)
Proportion of Participants with >=50% reduction in Countable Motor Seizures Per 28 Days in the Titration Plus Maintenance Periods Relative to Baseline
Proportion of participants with >=50% reduction in CMS-28 from the Baseline Period through the end of the DB Period.
Measured: From Baseline Period (Day 1) up to 16 weeks
European Union: Proportion of Participants with >=50% reduction in Countable Motor Seizures Per 28 Days in the Maintenance Period Relative to Baseline
Proportion of participants with >=50% reduction in CMS-28 from the Baseline Period through the end of the maintenance period.
Measured: From maintenance period Baseline (Day 29) up to Day 85
Number of Countable Motor Seizure-free Days in the Titration Plus Maintenance Periods Relative to Baseline
Number of countable motor seizure-free days from the Baseline Period through the end of the DB Period.
Measured: From Baseline Period (Day 1) up to 16 weeks
European Union: Number of Countable Motor Seizure-free Days in the Maintenance Period Relative to Baseline
Number of countable motor seizure-free days from the Baseline Period through the end of the maintenance period.
Measured: From maintenance period Baseline (Day 29) up to Day 85
Clinical Global Impression of Improvement - Clinician (CGII-C) Score
CGII-C score at the end of the maintenance Period. This 1-item scale asks the clinician to rate how the participant's symptoms have improved or worsened relative to baseline.
Measured: Day 85
Clinical Global Impression of Improvement - Participant/Caregiver (CGII-P) Score
CGII-C score at the end of the maintenance Period. This 1-item scale asks the clinician to rate how the participant's symptoms have improved or worsened relative to baseline.
Measured: Day 85
Percent Change in All Seizures in the Titration Plus Maintenance Periods Relative to Baseline
Percent change in all seizures at the end of the DB Period.
Measured: From Baseline Period (Day 1) up to 16 weeks
Percent Change in All Seizures in the Maintenance Period Relative to Baseline
Percent change in all seizures at the end of the maintenance period.
Measured: From maintenance period Baseline (Day 29) up to Day 85
Incidence of Rescue Anti-epileptic Drug (AED) Use in the Titration Plus Maintenance Periods Relative to Baseline
Incidence of rescue AED use as measured by the number of days on rescue AEDs from the Baseline Period through the end of the DB Period.
Measured: From Baseline Period (Day 1) up to 16 weeks
Incidence of Rescue Anti-epileptic Drug Use in the Maintenance Period Relative to Baseline
Incidence of rescue AED use as measured by the number of days on rescue AEDs from the Baseline Period through the end of the maintenance period.
Measured: From maintenance period Baseline (Day 29) up to Day 85
United States FDA: Proportion of Participants with >=50% Reduction in the Countable Motor Seizures Per 28 Days in the Maintenance Period Relative to Baseline
Proportion of participants with ≥50% reduction in CMS-28 from the Baseline Period through the end of the DB Maintenance Phase only.
Measured: From maintenance period Baseline (Day 29) up to Day 85
Incidence of Treatment-Emergent Adverse Events (TEAEs)
Incidence of TEAEs will be compared among the treatment groups.
Measured: From the first dose administration of study drug up to end of the study, approximately up to 172 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jul 2020
- Study start15 Sep 2020
- Primary completionApr 2027 (estimated)
- Study completionMay 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Dravet SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Patients are randomized 1:1 to clemizole HCl (EPX-100) or placebo.
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 150 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Epygenix
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Harmony Biosciences Management, Inc.
- Responsible party
- Sponsor
- Investigators
- Amit Ray, MD, Study director, Harmony Biosciences Management, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04462770
- Sponsor's protocol ID
- EPX-100-001
- Other IDs
- 2024-518628-57-00
- Acronym
- ARGUS
- Status last verified
- Sep 2026
- First submitted
- 6 Jul 2020
- Last updated
- 15 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No