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

Description

This is a multicenter, Phase 3, randomized, double-blind, placebo-controlled study designed to evaluate the efficacy and safety of clemizole HCL (EPX-100) as adjunctive therapy in children and adult participants with Dravet syndrome.

Conditions

Dravet Syndrome

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Study Overview

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Study Details

Study overview

This is a multicenter, Phase 3, randomized, double-blind, placebo-controlled study designed to evaluate the efficacy and safety of clemizole HCL (EPX-100) as adjunctive therapy in children and adult participants with Dravet syndrome.

A 20-Week Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial of EPX-100 (Clemizole Hydrochloride) as Adjunctive Therapy in Children and Adult Participants With Dravet Syndrome (ARGUS Trial)

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

Condition
Dravet Syndrome
Intervention / Treatment

-

Contacts and Locations

Los Angeles

Children's Hospital of Los Angeles, Los Angeles, California, United States, 90027

Orange

University of California Irvine, Orange, California, United States, 92868

San Francisco

UCSF Medical Center, San Francisco, California, United States, 94158

Wilmington

The Nemours Foundation, Wilmington, Delaware, United States, 19803

Kissimmee

Rare Disease Research FL, Kissimmee, Florida, United States, 34746

Winter Park

Pediatric Neurology and Epilepsy Specialists, Winter Park, Florida, United States, 32789

Atlanta

Clinical Integrative Research Center of Atlanta (CIRCA), Atlanta, Georgia, United States, 30328

Chicago

Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States, 60611

Louisville

Norton Children's Research Institute, Louisville, Kentucky, United States, 40202

Ann Arbor

University of Michigan- Mott Children's Hospital, Ann Arbor, Michigan, United States, 48109

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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, assent (if applicable) prior to initiation of any study related procedures.
  • 3. Clinical diagnosis of Dravet syndrome. 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 Dravet syndrome),
  • * Genetic mutation of the SCN1A gene must be documented.
  • 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 (CNS) disease deemed progressive, metabolic illness, or progressive degenerative disease.
  • 4. Prior or concurrent use of lorcaserin.
  • 5. Concurrent use of fenfluramine.
  • 6. Epilepsy surgery planned during the study or epilepsy surgery within 6 months prior to Screening.

Ages Eligible for Study

2 Years to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Epygenix,

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

Study Record Dates

2029-05-01