A Phase 3, Randomized, Multi-Center, Double-Blind, Placebo-Controlled Clinical Trial to Evaluate the Efficacy, Safety, Tolerability, and Pharmacokinetics of Relutrigine in Participants With DEE Followed by an Open-Label Extension
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 160
- 试验地点
- 23
- 主要终点
- To assess the effect of relutrigine on seizure frequency in participants with DEEs compared to placebo
研究概览
简要总结
A Phase 3, Randomized, Multi-Center, Double-Blind, Placebo-Controlled Clinical Trial to Evaluate the Efficacy, Safety, Tolerability, and Pharmacokinetics of Relutrigine in Participants with Developmental and Epileptic Encephalopathies Followed by an Open-Label Extension
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 2 Years 至 65 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Has a documented diagnosis of a developmental and epileptic encephalopathy.
- •Onset of seizures <12 years old.
- •Has a weight >7 kg at the time of signing consent/assent.
排除标准
- •Has a history of left bundle branch block, arrhythmias, Brugada syndrome, congenital heart disease, familial short QT syndrome, or family history of sudden death or ventricular arrhythmias, including idiopathic ventricular fibrillation.
- •Had 2 or more episodes of convulsive status epilepticus requiring hospitalization and intubation in the 6 months prior to Screening.
- •Has an abnormal ECG reading, including a QT interval corrected for heart rate using Bazett's method (QTcB) <350 and >450 ms (males), or <360 and >460 ms (females) at Screening and/or on Day
- •Any nerve stimulation must have been placed at least 3 months prior to Screening with at least 1 month of stable settings prior to Screening.
- •Has received any other experimental or investigational drug, device, or other therapy within 30 days or 5 half-lives (whichever is longer) prior to Screening, including any prior use of gene therapy.
- •Is currently pregnant or breastfeeding or is planning to become pregnant during the clinical trial or within 5 half-lives of the last study drug dose.
研究组 & 干预措施
Part A: Double-Blind Treatment Period (Placebo)
Eligible participants will be randomly assigned in a double-blind manner and a 1:1 ratio to receive 1.0mg-1.5mg/kg relutrigine or placebo once daily orally or gastronomy/jejunostomy for 16 weeks
干预措施: Placebo (Drug)
Part B: Open-Label Extension Treatment Period
Participants from Part A will have the option to rollover to Part B to receive 1.0mg-1.5mg/kg of relutrigine once daily orally or gastronomy/jejunostomy for 32 weeks
干预措施: 1.5mg/kg/day PRAX-562 (Drug)
Part A: Double-Blind Treatment Period
Eligible participants will be randomly assigned in a double-blind manner and a 1:1 ratio to receive 1.0mg-1.5mg/kg relutrigine or placebo once daily orally or gastronomy/jejunostomy for 16 weeks
干预措施: 1.0mg/kg/day PRAX-562 (Drug)
Part A: Double-Blind Treatment Period
Eligible participants will be randomly assigned in a double-blind manner and a 1:1 ratio to receive 1.0mg-1.5mg/kg relutrigine or placebo once daily orally or gastronomy/jejunostomy for 16 weeks
干预措施: 1.5mg/kg/day PRAX-562 (Drug)
Part B: Open-Label Extension Treatment Period
Participants from Part A will have the option to rollover to Part B to receive 1.0mg-1.5mg/kg of relutrigine once daily orally or gastronomy/jejunostomy for 32 weeks
干预措施: 1.0mg/kg/day PRAX-562 (Drug)
结局指标
主要结局
To assess the effect of relutrigine on seizure frequency in participants with DEEs compared to placebo
时间窗: 16 weeks
Change from baseline in monthly motor seizure frequency
次要结局
- Achieve >50% reduction in monthly seizure frequency from baseline(16 weeks)
- Change in seizure-free days(16 weeks)
- Clinical Global Impression-Severity questionnaire(16 weeks)
- Clinical Global Impression-Improvement questionnaire(16 weeks)
- Caregiver Global Impression-Severity questionnaire(16 weeks)
- Caregiver Global Impression-Improvement questionnaire(16 weeks)
- To evaluate the safety and tolerability of relutrigine in participants with DEEs(16 weeks)
- Columbia-Suicide Severity Rating Scale questionnaire(16 weeks)
- Evaluate the safety and tolerability of relutrigine in participants with DEEs(16 weeks)
- To evaluate the safety labs and tolerability of relutrigine in participants with DEEs(16 weeks)
- To evaluate the change in respiratory rate and tolerability of relutrigine in participants with DEEs(16 weeks)
- To evaluate the change in blood pressure and tolerability of relutrigine in participants with DEEs(16 weeks)
- To evaluate the change in pulse and tolerability of relutrigine in participants with DEEs(16 weeks)
- To evaluate the change in body temperature and tolerability of relutrigine in participants with DEEs(16 weeks)
- To evaluate the change in body temperature and tolerability of relutrigine in participants with DEEs(16 weeks)
- To evaluate the safety labs and tolerability of relutrigine in participants with DEEs(16 weeks)
- To evaluate the change in respiratory rate and tolerability of relutrigine in participants with DEEs(16 weeks)