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)
Who is this study for? Patients with Dravet Syndrome
What treatments are being studied? EPX-100 (Clemizole HCl)
Status: Recruiting
Location: See all (69) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 3
SUMMARY
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).
Eligibility
Participation Requirements
Sex: All
Minimum Age: 2
Healthy Volunteers: f
View:
• 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.
Locations
United States
Alabama
University of Alabama at Birmingham
RECRUITING
Birmingham
Arkansas
Arkansas Children's Hospital
RECRUITING
Little Rock
California
Children's Hospital of Los Angeles
TERMINATED
Los Angeles
University of California Irvine
RECRUITING
Orange
UCSF Medical Center
RECRUITING
San Francisco
Connecticut
Yale School of Medicine - Yale-New Haven Hospital
RECRUITING
New Haven
Yale University School of Medicine
NOT_YET_RECRUITING
New Haven
Delaware
The Nemours Foundation
TERMINATED
Wilmington
Florida
Rare Disease Research FL
RECRUITING
Kissimmee
Pediatric Neurology and Epilepsy Specialists
RECRUITING
Winter Park
Georgia
Clinical Integrative Research Center of Atlanta (CIRCA)
WITHDRAWN
Atlanta
Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago
TERMINATED
Chicago
Kentucky
Norton Children's Research Institute
RECRUITING
Louisville
Michigan
University of Michigan- Mott Children's Hospital
TERMINATED
Ann Arbor
North Carolina
Duke University Health System
RECRUITING
Durham
Science 37
ACTIVE_NOT_RECRUITING
Morrisville
Wake Forest Baptist Medical Center
RECRUITING
Winston-salem
Nebraska
Children's Nebraska
RECRUITING
Omaha
New Jersey
Northeast Regional Epilepsy Group
TERMINATED
Hackensack
Neurology Center for Epilepsy and Seizures
ACTIVE_NOT_RECRUITING
Marlboro
New York
Northwell Health - Lenox Hill Hospital
NOT_YET_RECRUITING
New York
Weill Cornell Medical Center
RECRUITING
New York
Ohio
Cincinnati Children's Hospital Medical Center
RECRUITING
Cincinnati
Cleveland Clinic
RECRUITING
Cleveland
Nationwide Children's Hospital
WITHDRAWN
Columbus
Pennsylvania
The Children's Hospital of Philadelphia
RECRUITING
Philadelphia
Tennessee
Le Bonheur Children's Hospital
TERMINATED
Memphis
Texas
Child Neurology Consultants of Austin
RECRUITING
Austin
UT Southwestern/Children's Health
WITHDRAWN
Dallas
Utah
University of Utah
RECRUITING
Salt Lake City
Washington
Seattle Children's Hospital
RECRUITING
Seattle
Wisconsin
Medical College of Wisconsin, Children's Wisconsin
NOT_YET_RECRUITING
Milwaukee
Other Locations
Argentina
Fundación Para La Lucha Contra Las Enfermedades Neurológicas de La Infancia - FLENI
NOT_YET_RECRUITING
Buenos Aires
Conci Carpinella - Santa Rosa N° 748
NOT_YET_RECRUITING
Córdoba
Instituto Privado Kremer
NOT_YET_RECRUITING
Córdoba
Instituto Médico Rivarola (GIEN SRL)
NOT_YET_RECRUITING
San Luis
Brazil
Hospital Universitário da Universidade Estadual de Campinas - UNICAMP
NOT_YET_RECRUITING
Campinas
Fundacao Faculdade Regional de Medicina de Sao Jose Do Rio Preto Hospital de Base - PPDS
NOT_YET_RECRUITING
São José Do Rio Preto
Hospital São Paulo da Universidade Federal de Sao Paulo - Escola Paulista de Medicina UNIFESP-EPM
NOT_YET_RECRUITING
São Paulo
Instituto de Psiquiatria HC FMUSP
NOT_YET_RECRUITING
São Paulo
Canada
Children's Hospital of Eastern Ontario Research Institute Inc.
RECRUITING
Ottawa
The Hospital for Sick Children
RECRUITING
Toronto
Toronto Western Hospital, University Health Network
RECRUITING
Toronto
UBC Children's Hospital Research Institute
RECRUITING
Vancouver
Georgia
Institute of Neurology and Neuropsychology LTD
RECRUITING
Tbilisi
Medi Club Georgia LLC
RECRUITING
Tbilisi
Tbilisi State Medical University, Givi Zhvania Academic, Clinic of Pediatry
RECRUITING
Tbilisi
Hungary
Semmelweis University
RECRUITING
Budapest
University of Debrecen
RECRUITING
Debrecen
India
KLES Dr Prabhakar Kore Hospital and Medical Research Centre
NOT_YET_RECRUITING
Belagavi
National Institute of Mental Health and Neurosciences (NIMHANS)
NOT_YET_RECRUITING
Bengaluru
Rainbow Children's Hospital
NOT_YET_RECRUITING
Hyderabad
Yashoda Hospitals Hitec City
NOT_YET_RECRUITING
Hyderabad
Amrita Institute of Medical Sciences & Research Center
RECRUITING
Kochi
SRCC Children's Hospital
RECRUITING
Mahālakshmi
Jaslok Hospital & Research Centre
RECRUITING
Mumbai
Central India Cardiology Hospital and Research Institute
RECRUITING
Nagpur
All India Institute of Medical Sciences (AIIMS)
RECRUITING
New Delhi
KEM Hospital Research Centre
NOT_YET_RECRUITING
Pune
Saachi Children's Hospital
RECRUITING
Surat
Christian Medical College
RECRUITING
Vellore
Poland
University Clinical Center in Gdansk, Division of Developmental Neurology
RECRUITING
Gdansk
Medical Centre Plejady
RECRUITING
Krakow
Institute of Mother and Child
RECRUITING
Warsaw
Spain
Hospital de la Santa Creu i Sant Pau
RECRUITING
Barcelona
Hospital Infantil Universitario Niño Jesús
RECRUITING
Madrid
United Kingdom
Cardiff and Vale University Health Board
ACTIVE_NOT_RECRUITING
Cardiff
Great Ormond Street Hospital For Children
ACTIVE_NOT_RECRUITING
London
Sheffield Children's Hospital
ACTIVE_NOT_RECRUITING
Sheffield
Contact Information
Primary
Krystle Rapchak
clinicaltrials@harmonybiosciences.com
+1 (312) 847-1289
Backup
Eric Bauer
clinicaltrials@harmonybiosciences.com
Time Frame
Start Date: 2020-09-15
Estimated Completion Date: 2029-05
Participants
Target number of participants: 150
Treatments
Experimental: Double-blind clemizole HCl
Participants will receive their first dose of study drug following randomization.
Placebo_comparator: Placebo
Participants will receive their first dose of study drug following randomization.
Experimental: Open-label clemizole HCl
Eligible participants who complete the DB Period will have the option to continue in the OLE Period, during which they will receive clemizole HCL for up to 3 years.
Authors
Gautam Popli, Julie Ziobro, Linda C Laux, James Wheless, Eric Segal, Robert Nahouraii, Aaron Cardon, Sharon Whiting
Related Therapeutic Areas
Sponsors
Leads: Epygenix
Collaborators: Harmony Biosciences Management, Inc.