Status:
RECRUITING
A Study of EPX-100 (Clemizole Hydrochloride) in Participants With Dravet Syndrome
Lead Sponsor:
Epygenix
Collaborating Sponsors:
Harmony Biosciences Management, Inc.
Conditions:
Dravet Syndrome
Eligibility:
All Genders
2+ years
Phase:
PHASE3
Brief 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 a...
Detailed Description
This is a global, multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of clemizole hydrochloride as adjunctive therapy in children and adult participant...
Eligibility Criteria
Inclusion
- Key
- 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 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
- 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.
Key Trial Info
Start Date :
September 15 2020
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
May 1 2029
Estimated Enrollment :
150 Patients enrolled
Trial Details
Trial ID
NCT04462770
Start Date
September 15 2020
End Date
May 1 2029
Last Update
August 28 2025
Active Locations (46)
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1
Children's Hospital of Los Angeles
Los Angeles, California, United States, 90027
2
University of California Irvine
Orange, California, United States, 92868
3
UCSF Medical Center
San Francisco, California, United States, 94158
4
Yale University School of Medicine
New Haven, Connecticut, United States, 06510