NCT04462770 · Epygenix
A Study of EPX-100 (Clemizole Hydrochloride) in Participants With Dravet Syndrome
(ARGUS)
What this study is about
This is a conducted at multiple hospitals, Phase 3, randomly assigned, where neither patients nor doctors know which treatment is given, compared against an inactive treatment study designed to evaluate the effectiveness and safety of clemizole hydrochloride (EPX-100) as adjunctive therapy in children and adult participants with Dravet syndrome (DS).
View original scientific description
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).
Interventions
DRUG
Clemizole HCl
Clemizole HCl will be administered as an oral solution.
DRUG
Placebo
Placebo will be administered as an oral solution.
Primary outcome measures
Percent Change in Countable Motor Seizures Per 28 Days (CMS-28) in the Titration Plus Maintenance Periods Relative to Baseline
Time frame: From Baseline Period (Day 1) up to 16 weeks
Percent change in CMS-28 from the Baseline Period through the end of the DB period.
European Union: Percent Change in Countable Motor Seizures Per 28 Days in the Maintenance Period Relative to Baseline
Time frame: From maintenance period Baseline (Day 29) up to Day 85
Percent change in CMS-28 from the Baseline Period through the end of the maintenance period.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- 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, 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
- 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
Where
- Birmingham, Alabama
- Little Rock, Arkansas
- Los Angeles, California
- Orange, California
- San Francisco, California
- New Haven, Connecticut
- Wilmington, Delaware
- Kissimmee, Florida
- Winter Park, Florida
- Atlanta, Georgia
- Chicago, Illinois
- Louisville, Kentucky
And 18 more locations — see the full list below.
Collaborators
Harmony Biosciences Management, Inc.
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Jun 30, 2026 · Source of record for eligibility and locations