Actively Recruiting
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)
Led by Epygenix · Updated on 2026-05-14
150
Participants Needed
58
Research Sites
108 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
Researchers are evaluating clemizole hydrochloride (EPX-100) as an additional treatment for children and adults with Dravet syndrome (DS), a rare form of epilepsy. This Phase 3 study is designed to assess the safety and effectiveness of clemizole hydrochloride compared to a placebo, focusing on reducing motor seizures. The study includes participants who have seizures not fully controlled by current anti-epileptic drugs and have a confirmed genetic mutation related to DS. Participants will first go through a 4-week observational period before being randomly assigned to receive either clemizole hydrochloride or a placebo as an oral solution during a 16-week double-blind period. After completing this phase, eligible participants can join an open-label extension where they receive clemizole hydrochloride for up to three years. This design allows researchers to monitor both short-term and longer-term effects of the treatment. During the study, participants will be closely monitored for changes in their motor seizure frequency using daily counts and clinical assessments. Researchers will also evaluate seizure-free days and overall seizure reduction, along with the use of rescue medications. Safety will be tracked throughout the study and the extension period, which may last up to approximately 172 weeks. Participants will have regular visits to assess their response and well-being throughout the trial.
CONDITIONS
Brief Title
A Study of EPX-100 (Clemizole Hydrochloride) in Participants With Dravet Syndrome
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male and female participants 2 years and older at time of consent
- Participant or parent/legally authorized representative willing and able to provide written informed consent and assent if applicable
- Clinical diagnosis of Dravet syndrome with seizures not fully controlled by anti-epileptic drugs
- Onset of seizures before 18 months of age
- Normal development at seizure onset
- History of at least one type of countable motor seizure
- Brain MRI showing no cortical malformation except mild atrophy related to Dravet syndrome progression
- Documented genetic mutation of the SCN1A gene
You will not qualify if you...
- Known sensitivity, allergy, or previous exposure to clemizole hydrochloride
- Exposure to any investigational drug or device within 90 days prior to screening or plans to participate in another drug or device trial during the study
- Seizures caused by illicit drugs, alcohol use, infection, neoplasm, demyelinating or degenerative neurological disease, metabolic illness, or other progressive diseases
- Current use of lorcaserin or fenfluramine (prior lorcaserin use allowed if last dose was at least 30 days ago)
- Planned epilepsy surgery during the study or epilepsy surgery within 6 months prior to screening
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 4 weeks
Participants are observed without receiving the study drug to establish baseline seizure frequency.
Weekly visits for up to 4 weeks
Duration - 16 weeks
Participants receive either clemizole hydrochloride or placebo as adjunctive therapy to evaluate safety and efficacy.
Regular visits throughout 16 weeks
Duration - Up to 3 years
Eligible participants who complete the double-blind period receive open-label clemizole hydrochloride for continued treatment and safety monitoring.
Visits as scheduled during extension period
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
Research Team
K
Krystle Rapchak
E
Eric Bauer
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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