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Looking for participantsPhase3

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

Sponsor: Epygenix

NCT ID: NCT04462770

View on ClinicalTrials.gov ↗

At a glance

What the study gets you
Access to the study treatment being tested
Type of study
Interventional (receives a drug or procedure)
Time in hospital
In-person visits at study sites — visit count not specified by the sponsor
Drug or intervention
Clemizole HCl (drug), Placebo (drug)
How long the study runs
Study runs about 104 months (dates as stated)
About the drug or intervention
Clemizole HCl — drug: Clemizole HCl will be administered as an oral solution. · Placebo — drug: Placebo will be administered as an oral solution.
Patient visit burden
Not specified by the sponsor
Type of study
Testing a treatment
Ages
2 Years and over
Who
All
Number of participants
150
Started
2020-09-15
Last checked
2026-09

Plain English Summary

What is this study?

  • • Testing a new treatment for dravet syndrome
  • • Phase3 - 150 participants
  • • 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)

Who can take part?

  • • Ages 2 Years and over
  • • Diagnosed with dravet syndrome

Where?

  • • Cardiff - Cardiff and Vale University Health Board
  • • London - Great Ormond Street Hospital For Children
  • • Sheffield - Sheffield Children's Hospital

This is a simplified summary. Always discuss with your doctor before making any decisions.

About This 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).

More detail

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.

Dravet Syndrome

How this trial compares with your answers

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What we know so far

Condition· Matched your search
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Still need:

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Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.

Eligibility at a Glance

Key info

  • Age: 2 Years and over
  • Who can join: All genders

Treatment history

Treatments you must have had:

  • ✓ seizures which are not completely controlled by AEDs with the following criteria:

What the study is looking for

  • ✓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 agreement to take part...
  • ✓Clinical diagnosis of DS. Participants must have seizures which are not completely controlled by AEDs with the...
  • ✓Onset of seizures prior to 18 months of age,
  • ✓Normal development at onset,

Who cannot take part

  • ✗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...
  • ✗Concurrent use of lorcaserin. Note: Prior use of lorcaserin is permitted if at least 30 days have passed since the...
  • ✗Concurrent use of fenfluramine.
  • ✗Epilepsy surgery planned during the study or epilepsy surgery within 6 months prior to Screening.
See the full criteria
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.

Where Is This Study? (3 UK sites)

Cardiff and Vale University Health Board

Cardiff CF14 4XW, United Kingdom

ACTIVE_NOT_RECRUITING
Hospital R&D contact (matched)

Sarah Martin

research.development@wales.nhs.uk029 2074 6986

Great Ormond Street Hospital For Children

London WC1N 1EH, United Kingdom

ACTIVE_NOT_RECRUITING
Hospital R&D contact (matched)

Main Email: Research.Governance@gosh.nhs.uk

Research.Governance@gosh.nhs.uk0207 905 2700

Sheffield Children's Hospital

Sheffield S102TH, United Kingdom

ACTIVE_NOT_RECRUITING
Hospital R&D contact (matched)

Alessia Dunn

STH.ResearchAdministration@nhs.net0114 2712550

How to Get in Touch

Krystle Rapchak

Sponsor contact

CONTACT

+1 (312) 847-1289 clinicaltrials@harmonybiosciences.com

Eric Bauer

Sponsor contact

CONTACT

clinicaltrials@harmonybiosciences.com
Data sourced from ClinicalTrials.gov · Last verified: 2026-09