RECRUITING

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

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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).

Official Title

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)

Quick Facts

Study Start:2020-09-15
Study Completion:2029-05-01
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT04462770

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.

Ages Eligible for Study:2 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. 1. Male and female participants 2 years and older at time of consent.
  2. 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. 3. Clinical diagnosis of DS. Participants must have seizures which are not completely controlled by AEDs with the following criteria:
  4. * Onset of seizures prior to 18 months of age,
  5. * Normal development at onset,
  6. * History of at least one type of countable motor seizure (CMS),
  7. * Brain MRI without cortical malformation (not including mild atrophy associated with the natural progression of DS),
  8. * Genetic mutation of the SCN1A gene must be documented.
  1. 1. Known sensitivity, allergy, or previous exposure to clemizole HCl.
  2. 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. 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. 4. Concurrent use of lorcaserin. Note: Prior use of lorcaserin is permitted if at least 30 days have passed since the last dose.
  5. 5. Concurrent use of fenfluramine.
  6. 6. Epilepsy surgery planned during the study or epilepsy surgery within 6 months prior to Screening.

Contacts and Locations

Study Contact

Krystle Rapchak
CONTACT
+1 (312) 847-1289
clinicaltrials@harmonybiosciences.com
Eric Bauer
CONTACT
clinicaltrials@harmonybiosciences.com

Principal Investigator

Amit Ray, MD
STUDY_DIRECTOR
Harmony Biosciences Management, Inc.

Study Locations (Sites)

Children's Hospital of Los Angeles
Los Angeles, California, 90027
United States
University of California Irvine
Orange, California, 92868
United States
UCSF Medical Center
San Francisco, California, 94158
United States
Yale University School of Medicine
New Haven, Connecticut, 06510
United States
The Nemours Foundation
Wilmington, Delaware, 19803
United States
Rare Disease Research FL
Kissimmee, Florida, 34746
United States
Pediatric Neurology and Epilepsy Specialists
Winter Park, Florida, 32789
United States
Clinical Integrative Research Center of Atlanta (CIRCA)
Atlanta, Georgia, 30328
United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
United States
Norton Children's Research Institute
Louisville, Kentucky, 40202
United States
University of Michigan- Mott Children's Hospital
Ann Arbor, Michigan, 48109
United States
Children's Nebraska
Omaha, Nebraska, 68114
United States
Northeast Regional Epilepsy Group
Hackensack, New Jersey, 07601
United States
Neurology Center for Epilepsy and Seizures
Marlboro, New Jersey, 07746
United States
Weill Cornell Medical Center
New York, New York, 10021
United States
Northwell Health - Lenox Hill Hospital
New York, New York, 10075
United States
Duke University Health System
Durham, North Carolina, 27710
United States
Wake Forest Baptist Medical Center
Winston-Salem, North Carolina, 27157
United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
United States
Cleveland Clinic
Cleveland, Ohio, 44195
United States
Nationwide Children's Hospital
Columbus, Ohio, 43205
United States
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
United States
Le Bonheur Children's Hospital
Memphis, Tennessee, 38103
United States
Child Neurology Consultants of Austin
Austin, Texas, 78757
United States
UT Southwestern/Children's Health
Dallas, Texas, 75207
United States
University of Utah
Salt Lake City, Utah, 84112
United States
Seattle Children's Hospital
Seattle, Washington, 98105
United States

Collaborators and Investigators

Sponsor: Epygenix

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

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2020-09-15
Study Completion Date2029-05-01

Study Record Updates

Study Start Date2020-09-15
Study Completion Date2029-05-01

Terms related to this study

Keywords Provided by Researchers

  • Clemizole hydrochloride
  • Convulsive seizure
  • Pediatric epilepsy
  • Dravet syndrome

Additional Relevant MeSH Terms

  • Dravet Syndrome