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A Study of Clemizole Hydrochloride for Dravet Syndrome

This study is testing a drug called clemizole hydrochloride (EPX-100) to see if it can help people with Dravet syndrome, a severe form of epilepsy. Researchers want to know if clemizole hydrochloride can reduce the number of seizures when added to a person's current medications. You might be able to join if you are 2 years or older and have a clinical diagnosis of Dravet syndrome. The main way researchers will know if the treatment is successful is by looking at how much the number of motor seizures (seizures that involve movement) changes over 16 weeks compared to before starting the study drug. The study is currently recruiting about 150 participants.

Study design
This is a Phase 3 study where participants are randomly assigned to receive either clemizole hydrochloride or a placebo (an inactive substance) without knowing which they are getting. It is a global, multicenter, double-blind study.
What's involved
You would participate in a 4-week observation period, followed by a 16-week period where you receive either clemizole hydrochloride or placebo. There is also an optional Open-Label Extension Period after the initial 16 weeks.
Compensation
Not stated in the trial record.
Follow-up
The primary outcomes are measured up to 16 weeks from the start of the study, and up to Day 85 for the European Union primary endpoint.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT04462770

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

Recruiting
PHASE3Ages 2+InterventionalTreatment
Epygenix
~150 participants
Updated 2026-08-13 on ClinicalTrials.gov
What's tested:Clemizole HClPlacebo

At a glance

Recruiting sites
39 of 69 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Percent Change in Countable Motor Seizures Per 28 Days (CMS-28) in the Titration Plus Maintenance Periods Relative to Baseline
Measured over From Baseline Period (Day 1) up to 16 weeks
+1 more outcome measured

Conditions

Dravet Syndrome

Explore related trials

Developmental and Epileptic Encephalopathy 6ASCN1A Gene

Where it's being run

69 sites across 39 states
Georgia4
Argentina4
Brazil4
California3
North Carolina3
Ohio3
Ontario3
Poland3

Study leadership

  • Amit Ray, MD · STUDY_DIRECTOR · Harmony Biosciences Management, Inc.

Who to contact

Krystle Rapchak
[email protected]
+1 (312) 847-1289
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

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.

What this trial measures

  • Percent Change in Countable Motor Seizures Per 28 Days (CMS-28) in the Titration Plus Maintenance Periods Relative to BaselineFrom 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 BaselineFrom 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.