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NCT05066217PHASE3Recruiting

An Efficacy and Safety Study of Clemizole HCl in Patients With Lennox-Gastaut Syndrome

Epygenix

Start Date

4/9/2025

Completion Date

11/1/2029

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 Lennox-Gastaut syndrome (LGS).

Detailed Description

This is a multicenter, Phase 3, randomized, double-blind, placebo-controlled study designed to evaluate the efficacy and safety of clemizole HCl as adjunctive therapy in children and adult participants with LGS. The study will consist of an Observational Period, a Double-Blind (DB) Period, and an optional Open-Label Extension (OLE) Period.

Eligibility Criteria

Age Range: 2 years to 55 years

Key Inclusion Criteria: 1. Males or females, ages ≥2 to ≤55 years, at the time of Screening. 2. Participant/parent/legal authorized representative (LAR) willing and able to give written informed consent/assent. 3. Diagnosis of LGS, including: * Evidence of at least one type of countable major motor seizure. * History of electroencephalogram (EEG) consistent with LGS (abnormal background activity, and one of the following: 1) slow spike-wave discharges \[\<2.5 Hz\], or 2) paroxysmal fast activity during sleep). * Abnormal cognitive development. * Onset of seizures at 11 years of age or younger. Key Exclusion Criteria: 1. Known sensitivity, allergy, or previous exposure to clemizole HCl. 2. Known history of long QT syndrome or any significant history of a serious abnormality of the electrocardiogram (ECG) (e.g., recent myocardial infarction, clinically significant arrhythmia). 3. Family history of sudden cardiac death, unexplained death, or death from a primary dysrhythmia potentially associated with QT prolongation in any family member. 4. Seizures secondary to illicit drug or alcohol use, infection, neoplasm, demyelinating disease, degenerative neurological disease, or progressive central nervous system disease, metabolic illness, recent anoxic episode within the last 6 months requiring resuscitation, or progressive degenerative disease or any other condition, which in the opinion of the investigator, could affect seizure control. 5. Epilepsy surgery planned during the study or epilepsy surgery within 6 months prior to Screening. 6. Concomitant use of fenfluramine. 7. Prior or concomitant use of lorcaserin.

Interventions

DRUG

Clemizole HCl

DRUG

Placebo

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Conditions

Lennox Gastaut Syndrome

Locations

Arkansas Children's Hospital

Little Rock, Arkansas 72202

United States

UC Irvine Medical Center

Orange, California 92868

United States

UCI Center for Innovative Health Therapies

Orange, California 92868

United States

Nemours Children's Health

Wilmington, Delaware 19803

United States

Rare Disease Research

Kissimmee, Florida 34746

United States

University of Miami Miller School of Medicine

Miami, Florida 33136

United States

AdventHealth Innovation Tower

Orlando, Florida 32804

United States

Pediatric Neurology and Epilepsy Specialists

Winter Park, Florida 32789

United States

Re:Cognition Health

Chicago, Illinois 60611

United States

Norton Children's Medical Center

Louisville, Kentucky 40202

United States

Henry Ford Hospital

Detroit, Michigan 48202

United States

Minnesota Epilepsy Group, P.A.

Roseville, Minnesota 55102

United States

Children's Nebraska

Omaha, Nebraska 68114

United States

Neurology Center for Epilepsy and Seizures

Marlboro, New Jersey 07746

United States

Tekton Research

Marlboro, New Jersey 07746

United States

NYU Comprehensive Epilepsy Center - BRANY - PPDS

New York, New York 10016-4852

United States

Weill Cornell Medicine/New York Presbyterian Hospital

New York, New York 10021

United States

Atrium Health STRIVE Research

Charlotte, North Carolina 28207

United States

On-Site Clinical Solution

Charlotte, North Carolina 28211

United States

Duke University Medical Center

Durham, North Carolina 27705

United States

PPD Virtual-Science 37, Inc.

Morrisville, North Carolina 27560

United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania 19104

United States

UTHealth Houston

Houston, Texas 77030

United States

Clinical Neurosciences Center

Salt Lake City, Utah 84132

United States

IRCCS Istituto Neurologico Mediterraneo Neuromed

Pozzilli, Molise 86077

Italy

Neurosphera

Warsaw, Masovian Voivodeship 02-829

Poland