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

Purpose

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).

Condition

  • Lennox Gastaut Syndrome

Eligibility

Eligible Ages
Between 2 Years and 55 Years
Eligible Sex
All
Accepts Healthy Volunteers
No

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.

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.

Study Design

Phase
Phase 3
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Intervention Model Description
Patients are randomized 1:1 to clemizole HCl (EPX-100) or placebo.
Primary Purpose
Treatment
Masking
Triple (Participant, Care Provider, Investigator)

Arm Groups

ArmDescriptionAssigned Intervention
Placebo Comparator
Placebo 		Participants will receive their first dose of study drug following randomization.
  • Drug: Placebo
    Placebo will be administered as an oral solution.
Experimental
Double-blind clemizole HCl 		Participants will receive their first dose of study drug following randomization.
  • Drug: Clemizole HCl
    Clemizole HCl will be administered as an oral solution.
    Other names:
    • EPX-100
Experimental
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.
  • Drug: Clemizole HCl
    Clemizole HCl will be administered as an oral solution.
    Other names:
    • EPX-100

Recruiting Locations

UCI Center for Innovative Health Therapies
Orange, California 92868
Contact:
David King-Stephens, MD
714-456-7720

University of Miami Miller School of Medicine
Miami, Florida 33136
Contact:
Brett Alonso Cardenas
305-243-3100
bac301@med.miami.edu

AdventHealth Innovation Tower
Orlando, Florida 32804

Re:Cognition Health
Chicago, Illinois 60611
Contact:
Antonio Iglesias, MD
312-971-3318
aiglesias@re-cognitionhealth.com

Neurology Center for Epilepsy and Seizures
Marlboro, New Jersey 07746
Contact:
732-433-3431

Tekton Research
Marlboro, New Jersey 07746
Contact:
Aimee Kinlan
626-218-8294
aimee.kinlan@tektonresearch.com

NYU Comprehensive Epilepsy Center - BRANY - PPDS
New York, New York 10016-4852

Weill Cornell Medicine/New York Presbyterian Hospital
New York, New York 10021
Contact:
Lundy Santil
800-785-3150
ludny.saintil@thermofisher.com

Duke University Medical Center
Durham, North Carolina 27705
Contact:
Muhammad Zafar, MD
muhammad.zafar@duke.edu

Children's Hospital of Philadelphia
Philadelphia, Pennsylvania 19104

Clinical Neurosciences Center
Salt Lake City, Utah 84132

More Details

Status
Recruiting
Sponsor
Epygenix

Study Contact

Juby Philip
(302) 559-4320
clinicaltrials@harmonybiosciences.com

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.