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

Официальное название

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

Обзор

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).

Подробное описание

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.

Вмешательства

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

Первичные конечные точки

• Percent Change in CMMS-28 [Срок оценки: From Baseline Period up to 16 weeks]

Вторичные конечные точки (10)

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

Критерии участия

Критерии включения

• 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.

Критерии исключения

• 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Параллельные группы

Маскирование

Тройное слепое

Основная цель

Лечение

Центры проведения

США · 24 центра

• 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
• … и ещё 16 центров

Италия · 1 центр

• IRCCS Istituto Neurologico Mediterraneo Neuromed — Pozzilli

Польша · 1 центр

• Neurosphera — Warsaw

Идентификаторы

NCT: NCT05066217 · EPX-100-003

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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