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Recruiting NCT05066217

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

Phase III Interventional Lennox Gastaut Syndrome

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Clemizole HCl, Placebo.
Who it may be relevant to
Registry conditions: Lennox Gastaut Syndrome. Basic parameters: 2 years — 55 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States, Italy, Poland
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Official title

Multicenter, Randomized, Double-blind, Placebo-controlled Trial of Clemizole HCl as Adjunctive Therapy in Patients With Lennox-Gastaut Syndrome

Overview

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.

Interventions

  • Drug Clemizole HCl
    Clemizole HCl will be administered as an oral solution.
  • Drug Placebo
    Placebo will be administered as an oral solution.

Primary outcome measures

  • Percent Change in CMMS-28 [Time frame: From Baseline Period up to 16 weeks]
Secondary outcome measures (10)
  • Proportion of Participants with ≥50% Reduction in CMMS-28 [Time frame: From Baseline Period up to 16 weeks]
  • Percent Change in CMMS-28 Seizure-free Days [Time frame: From Baseline Period up to 16 weeks]
  • Clinical Global Impression of Change (CGI-C) Score [Time frame: Week 16]
  • Caregiver Global Impression of Change (CaGI-C) Score [Time frame: Week 16]
  • Caregiver Global Impression of Change in Seizure Intensity/Duration (CaGI-CSID) Score [Time frame: Week 16]
  • Change in Quality of Life Inventory (QI)-Disability Score [Time frame: From Baseline Period up to 16 weeks]
  • Percent Change per 28 Days in the Number of Seizure Free Days [Time frame: From Baseline Period up to 16 weeks]
  • Percent Change in CMMS-28 [Time frame: From Baseline Period up to 12 weeks]
  • Proportion of Participants with ≥50% Reduction in CMMS-28 [Time frame: From Baseline Period up to 12 weeks]
  • Incidence of Treatment-Emergent Adverse Events (TEAEs) [Time frame: From the first dose administration of study drug up to end of the study, approximately up to 172 weeks]

Eligibility criteria

Inclusion criteria

  • Males or females, ages ≥2 to ≤55 years, at the time of Screening.
  • Participant/parent/legal authorized representative (LAR) willing and able to give written informed consent/assent.
  • 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

  • Known sensitivity, allergy, or previous exposure to clemizole HCl.
  • 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).
  • Family history of sudden cardiac death, unexplained death, or death from a primary dysrhythmia potentially associated with QT prolongation in any family member.
  • 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.
  • Epilepsy surgery planned during the study or epilepsy surgery within 6 months prior to Screening.
  • Concomitant use of fenfluramine.
  • Prior or concomitant use of lorcaserin.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Randomized
Model
Parallel assignment
Masking
Triple blind
Primary purpose
Treatment

Study locations

United States · 24 centers
  • Arkansas Children's Hospital — Little Rock
  • UC Irvine Medical Center — Orange
  • UCI Center for Innovative Health Therapies — Orange
  • Nemours Children's Health — Wilmington
  • Rare Disease Research — Kissimmee
  • University of Miami Miller School of Medicine — Miami
  • AdventHealth Innovation Tower — Orlando
  • Pediatric Neurology and Epilepsy Specialists — Winter Park
  • … and 16 more centers
Italy · 1 center
  • IRCCS Istituto Neurologico Mediterraneo Neuromed — Pozzilli
Poland · 1 center
  • Neurosphera — Warsaw

Identifiers

NCT: NCT05066217 · EPX-100-003

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗