A Study of EPX-100 (Clemizole Hydrochloride) in Participants With Dravet Syndrome
- Registration Number
- NCT04462770
- Lead Sponsor
- Epygenix
- Brief Summary
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.
- Detailed Description
This is a global multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of clemizole HCl as adjunctive therapy in children and adult participants with Dravet syndrome. The study consists of a 4-week Observational Period, a 16-week Double-Blind (DB) Period and an Open-Label Extension (OLE) Period for up to 156 weeks.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 150
-
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, assent (if applicable) prior to initiation of any study related procedures.
-
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.
Key
- 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 (CNS) disease deemed progressive, metabolic illness, or progressive degenerative disease.
- Prior or concurrent use of lorcaserin.
- Concurrent use of fenfluramine.
- Epilepsy surgery planned during the study or epilepsy surgery within 6 months prior to Screening.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Double-blind clemizole HCl Clemizole HCl Participants will receive their first dose of study drug following randomization. Placebo Placebo Participants will receive their first dose of study drug following randomization. Open-label clemizole HCl 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. Open-label clemizole HCl Placebo 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.
- Primary Outcome Measures
Name Time Method Percent Change in CMS-28 From Baseline Period up to 16 weeks Percent change in CMS-28 from the Baseline Period through the end of the DB Period
- Secondary Outcome Measures
Name Time Method Number of Countable Motor Seizure-free Days From Baseline Period up to 16 weeks Number of countable motor seizure-free days from the Baseline Period through the end of the DB Period
Clinical Global Impression of Improvement - Clinician (CGII-C) Score Week 16 CGII-C score at the end of the DB Period
Clinical Global Impression of Improvement - Participant/Caregiver (CGII-P) Score Week 16 CGII-P score at the end of the DB Period
Proportion of Participants with ≥50% Reduction in CMS-28 From Baseline Period up to 12 weeks Proportion of participants with ≥50% reduction in CMS-28 from the Baseline Period through the end of the DB Maintenance Phase only
Percent Change in All Seizures Week 16 Percent change in all seizures at the end of the DB Period
Incidence of Rescue Anti-epileptic Drug (AED) From Baseline Period up to 16 weeks Incidence of rescue AED use as measured by the number of days on rescue AEDs from the Baseline Period through the end of the DB Period
Incidence of Treatment-Emergent Adverse Events (TEAEs) From the first dose administration of study drug up to end of the study, approximately up to 172 weeks Incidence of TEAEs will be compared among the treatment groups
Proportion of Participants with ≥50% reduction in CMS-28 From Baseline Period up to 16 weeks Proportion of participants with ≥50% reduction in CMS-28 from the Baseline Period through the end of the DB Period
Trial Locations
- Locations (44)
University of California Irvine
🇺🇸Orange, California, United States
UCSF Medical Center
🇺🇸San Francisco, California, 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
Norton Children's Research Institute
🇺🇸Louisville, Kentucky, United States
University of Michigan- Mott Children's Hospital
🇺🇸Ann Arbor, Michigan, United States
Children's Nebraska
🇺🇸Omaha, Nebraska, United States
Northeast Regional Epilepsy Group
🇺🇸Hackensack, New Jersey, United States
Neurology Center for Epilepsy and Seizures
🇺🇸Marlboro, New Jersey, United States
Weill Cornell Medical Center
🇺🇸New York, New York, United States
Duke University Health System
🇺🇸Durham, North Carolina, United States
Wake Forest Baptist Medical Center
🇺🇸Winston-Salem, North Carolina, United States
Cincinnati Children's Hospital Medical Center
🇺🇸Cincinnati, Ohio, United States
Cleveland Clinic
🇺🇸Cleveland, Ohio, United States
Nationwide Children's Hospital
🇺🇸Columbus, Ohio, United States
The Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
Le Bonheur Children's Hospital
🇺🇸Memphis, Tennessee, United States
Child Neurology Consultants of Austin
🇺🇸Austin, Texas, United States
UT Southwestern/Children's Health
🇺🇸Dallas, Texas, United States
University of Utah
🇺🇸Salt Lake City, Utah, United States
Seattle Children's Hospital
🇺🇸Seattle, Washington, United States
UBC Children's Hospital Research Institute
🇨🇦Vancouver, British Columbia, Canada
Children's Hospital of Eastern Ontario Research Institute Inc.
🇨🇦Ottawa, Ontario, Canada
The Hospital for Sick Children
🇨🇦Toronto, Ontario, Canada
Toronto Western Hospital, University Health Network
🇨🇦Toronto, Ontario, Canada
Tbilisi State Medical University, Givi Zhvania Academic, Clinic of Pediatry
🇬🇪Tbilisi, Georgia
Medi Club Georgia LLC
🇬🇪Tbilisi, Georgia
Institute of Neurology and Neuropsychology
🇬🇪Tbilisi, Georgia
Semmelweis University
🇭🇺Budapest, Hungary
University of Debrecen
🇭🇺Debrecen, Hungary
University Clinical Center in Gdansk, Division of Developmental Neurology
🇵🇱Gdańsk, Poland
Medical Centre Plejady
🇵🇱Kraków, Poland
Institute of Mother and Child
🇵🇱Warsaw, Poland
"Prof. Dr. Al. Obregia" Psychiatry Clinical Hospital
🇷🇴Bucharest, Romania
Hospital Infantil Universitario Niño Jesús
🇪🇸Madrid, Madrid provincia, Spain
Hospital de la Santa Creu i Sant Pau
🇪🇸Barcelona, Spain
Cardiff and Vale University Health Board
🇬🇧Cardiff, United Kingdom
Alder Hey Children's NHS Foundation Trust
🇬🇧Liverpool, United Kingdom
Great Ormond Street Hospital For Children
🇬🇧London, United Kingdom
Sheffield Children's Hospital
🇬🇧Sheffield, United Kingdom
Children's Hospital of Los Angeles
🇺🇸Los Angeles, California, United States