A Study of EPX-100 (Clemizole Hydrochloride) in Participants With Dravet Syndrome
Recruiting · NCT04462770 · Interventional (participants receive a specific treatment) · Lead sponsor: Epygenix
View the official record on ClinicalTrials.gov →Interventions studied
Clemizole HClPlacebo
What this trial 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 take part
Age range
2 Years and older
Sex
All (male and female)
Healthy volunteers
No - a diagnosis or condition is required
Phase
Phase 3
Study type
Interventional (participants receive a specific treatment)
Inclusion criteria
- 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.
- Key
Exclusion criteria
- 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 it is running
60 locations listed across 21 US states.
- 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 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
- 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
- Northwell Health - Lenox Hill Hospital - New York, New York, United States
- Duke University Health System - Durham, North Carolina, United States
- Science 37 - Morrisville, 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
- Medical College of Wisconsin, Children's Wisconsin - Milwaukee, Wisconsin, United States
- Hospital de la Santa Creu i Sant Pau - Barcelona, Spain
- Semmelweis University - Budapest, Hungary
- Fundación Para La Lucha Contra Las Enfermedades Neurológicas de La Infancia - FLENI - Buenos Aires, Argentina
- Hospital Universitário da Universidade Estadual de Campinas - UNICAMP - Campinas, Brazil
- Cardiff and Vale University Health Board - Cardiff, United Kingdom
- Conci Carpinella - Santa Rosa N° 748 - Córdoba, Argentina
- Instituto Privado Kremer - Córdoba, Argentina
- University of Debrecen - Debrecen, Hungary
- University Clinical Center in Gdansk, Division of Developmental Neurology - Gdansk, Poland
- Medical Centre Plejady - Krakow, Poland
- Great Ormond Street Hospital For Children - London, United Kingdom
- Instituto Médico Rivarola (GIEN SRL) - San Luis, Argentina
- Sheffield Children's Hospital - Sheffield, United Kingdom
- Fundacao Faculdade Regional de Medicina de Sao Jose Do Rio Preto Hospital de Base - PPDS - São José do Rio Preto, Brazil
- Hospital São Paulo da Universidade Federal de Sao Paulo - Escola Paulista de Medicina UNIFESP-EPM - São Paulo, Brazil
- Instituto de Psiquiatria HC FMUSP - São Paulo, Brazil
- Tbilisi State Medical University, Givi Zhvania Academic, Clinic of Pediatry - Tbilisi, Georgia
- Medi Club Georgia LLC - Tbilisi, Georgia
- Institute of Neurology and Neuropsychology LTD - Tbilisi, Georgia
- Institute of Mother and Child - Warsaw, Poland
- Rainbow Children's Hospital - Hyderabad, Andhra Pradesh, India
- Yashoda Hospitals Hitec City - Hyderabad, Andhra Pradesh, India
- UBC Children's Hospital Research Institute - Vancouver, British Columbia, Canada
- Saachi Children's Hospital - Surat, Gujarat, India
- KLES Dr Prabhakar Kore Hospital and Medical Research Centre - Belagavi, Karnataka, India
- National Institute of Mental Health and Neurosciences (NIMHANS) - Bengaluru, Karnataka, India
- Amrita Institute of Medical Sciences & Research Center - Kochi, Kerala, India
- Hospital Infantil Universitario Niño Jesús - Madrid, Madrid Provincia, Spain
- KEM Hospital Research Centre - Pune, Maharashta, India
Showing 60 of 68 listed sites. See the official record for the complete list.
Read the full protocol, contacts and eligibility on ClinicalTrials.gov →