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

What this study is about

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 join (things the study team will check)

✅ You may be able to join if…

• 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 informed consent and assent (if applicable) prior to initiation of any study related procedures.
• 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.

🚫 You may not be able to join if…

• 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 or device trial at any time during the study.
• 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.
• Concurrent use of lorcaserin. Note: Prior use of lorcaserin is permitted if at least 30 days have passed since the last dose.
• Concurrent use of fenfluramine.
• Epilepsy surgery planned during the study or epilepsy surgery within 6 months prior to Screening.

Where this trial is running

• University of Alabama at Birmingham, Birmingham, Alabama, United States
• Arkansas Children's Hospital, Little Rock, Arkansas, United States
• Children's Hospital of Los Angeles, Los Angeles, California, United States
• University of California Irvine, Orange, California, United States
• UCSF Medical Center, San Francisco, California, United States
• Yale University School of Medicine, New Haven, Connecticut, United States
• Yale School of Medicine - Yale-New Haven Hospital, New Haven, Connecticut, United States
• The Nemours Foundation, Wilmington, Delaware, United States
• Rare Disease Research FL, Kissimmee, Florida, United States
• Pediatric Neurology and Epilepsy Specialists, Winter Park, Florida, United States
• Clinical Integrative Research Center of Atlanta (CIRCA), Atlanta, Georgia, United States
• Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States

+ 48 more sites.

Who to contact

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

It's completely normal to call and ask questions before deciding anything. Mention the study ID: NCT04462770.