Evaluating clemizole hydrochloride for treating Dravet syndrome

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This multicenter, Phase 3, randomized, double-blind, placebo-controlled study aims to assess the safety and efficacy of clemizole hydrochloride (EPX-100) as an adjunctive therapy for individuals with Dravet syndrome. Participants will undergo a 4-week observational period followed by a 16-week double-blind phase, and an open-label extension lasting up to 156 weeks. The study includes both children and adults who experience seizures that are not fully controlled by existing antiepileptic drugs.

Why it matters

Who can join: the full rules

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 it's running

• University of Alabama at Birmingham
  Birmingham, Alabama, United States Recruiting
• Arkansas Children's Hospital
  Little Rock, Arkansas, United States Recruiting
• University of California Irvine
  Orange, California, United States Recruiting
• Yale School of Medicine - Yale-New Haven Hospital
  New Haven, Connecticut, United States Recruiting
• Pediatric Neurology and Epilepsy Specialists
  Winter Park, Florida, United States Recruiting
• Norton Children's Research Institute
  Louisville, Kentucky, United States Recruiting

+36 more sites. See ClinicalTrials.gov for the full list.

Study contacts

• Study coordinator: Krystle Rapchak
• Email: clinicaltrials@harmonybiosciences.com
• Phone: +1 (312) 847-1289

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• Measuring GABA Levels in Children with Dravet Syndrome

  This study is trying to measure GABA levels in the brains of children with Dravet Syndrome to see how they compare to kids without the condition, hoping to find new ways to understand and treat the syndrome.

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.