A Study of EPX-100 (Clemizole Hydrochloride) in Participants With Dravet Syndrome
NCT ID: NCT04462770
Last Updated: 2025-08-28
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE3
150 participants
INTERVENTIONAL
2020-09-15
2029-05-01
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
TRIPLE
Study Groups
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Double-blind clemizole HCl
Participants will receive their first dose of study drug following randomization.
Clemizole HCl
Clemizole HCl will be administered as an oral solution.
Placebo
Participants will receive their first dose of study drug following randomization.
Placebo
Placebo will be administered as an oral solution.
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.
Clemizole HCl
Clemizole HCl will be administered as an oral solution.
Placebo
Placebo will be administered as an oral solution.
Interventions
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Clemizole HCl
Clemizole HCl will be administered as an oral solution.
Placebo
Placebo will be administered as an oral solution.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
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 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.
Exclusion Criteria
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 disease deemed progressive, metabolic illness, or progressive degenerative disease.
4. Concurrent use of lorcaserin. Note: Prior use of lorcaserin is permitted if at least 30 days have passed since the last dose.
5. Concurrent use of fenfluramine.
6. Epilepsy surgery planned during the study or epilepsy surgery within 6 months prior to Screening.
2 Years
ALL
No
Sponsors
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Harmony Biosciences Management, Inc.
INDUSTRY
Epygenix
INDUSTRY
Responsible Party
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Principal Investigators
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Amit Ray, MD
Role: STUDY_DIRECTOR
Harmony Biosciences Management, Inc.
Locations
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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 University School of Medicine
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
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 LTD
Tbilisi, , Georgia
Semmelweis University
Budapest, , Hungary
University of Debrecen
Debrecen, , Hungary
University Clinical Center in Gdansk, Division of Developmental Neurology
Gdansk, , Poland
Medical Centre Plejady
Krakow, , 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
Countries
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Central Contacts
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Facility Contacts
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Other Identifiers
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2024-518628-57-00
Identifier Type: CTIS
Identifier Source: secondary_id
EPX-100-001
Identifier Type: -
Identifier Source: org_study_id
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