Multicenter, Randomized, Double-blind, Placebo-controlled Trial of Clemizole HCl as Adjunctive Therapy in Patients With Lennox-Gastaut Syndrome
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- 入组人数
- 260
- 试验地点
- 72
- 主要终点
- Percent Change in CMMS-28
研究概览
简要总结
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).
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 2 Years 至 55 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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.
研究组 & 干预措施
Placebo
Participants will receive their first dose of study drug following randomization.
干预措施: Placebo (Drug)
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.
干预措施: Clemizole HCl (Drug)
Double-blind clemizole HCl
Participants will receive their first dose of study drug following randomization.
干预措施: Clemizole HCl (Drug)
结局指标
主要结局
Percent Change in CMMS-28
时间窗: From Baseline Period up to 16 weeks
Percent change in CMMS-28 from the Baseline Period through the end of the DB Period
次要结局
- Proportion of Participants with ≥50% Reduction in CMMS-28(From Baseline Period up to 12 weeks)
- Percent Change in CMMS-28 Seizure-free Days(From Baseline Period up to 16 weeks)
- Clinical Global Impression of Change (CGI-C) Score(Week 16)
- Caregiver Global Impression of Change (CaGI-C) Score(Week 16)
- Caregiver Global Impression of Change in Seizure Intensity/Duration (CaGI-CSID) Score(Week 16)
- Change in Quality of Life Inventory (QI)-Disability Score(From Baseline Period up to 16 weeks)
- Percent Change per 28 Days in the Number of Seizure Free Days(From Baseline Period up to 16 weeks)
- Percent Change in CMMS-28(From Baseline Period up to 12 weeks)
- Incidence of Treatment-Emergent Adverse Events (TEAEs)(From the first dose administration of study drug up to end of the study, approximately up to 172 weeks)
- Proportion of Participants with ≥50% Reduction in CMMS-28(From Baseline Period up to 16 weeks)