An Open-label Pilot Study With an Extension Phase to Evaluate the Pharmacokinetics, and to Generate Preliminary Safety, Tolerability, and Efficacy of Perampanel (E2007) Oral Suspension When Given as an Adjunctive Therapy in Pediatric Subjects From 2 to Less Than 12 Years of Age With Epilepsy
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- Eisai Inc.
- 入组人数
- 63
- 主要终点
- Apparent Clearance (CL/F) of Perampanel [Core Study]
研究概览
简要总结
This study is designed to evaluate the pharmacokinetics, efficacy, and safety of perampanel oral suspension on seizure frequency in pediatric participants maintained on one to three stable antiepileptic drugs
详细描述
This is a multicenter, multiple ascending dose, open-label study (Core Study) with an Extension Phase. The Core Study consisted of 2 phases, the Pretreatment Phase and the Treatment Phase. The Pretreatment Phase lasted up to 2 weeks in duration, during which participants were assessed for their eligibility to participate in the study. The Treatment Phase consisted of 3 periods: Titration (7 weeks), Maintenance (4 weeks), and Follow-up (4 weeks; only for those participants not rolling over into the Extension Phase after completing the Treatment Phase and for those participants who discontinued from the study). All subjects who completed all scheduled visits up to and including the final visit of the Treatment Phase (Core Study) were eligible to participate in the Extension Phase of the study. The Extension Phase consisted of 2 periods: Maintenance (41 weeks) and Follow-up (4 weeks).
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 11 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
perampanel
Age Cohort 1 ( greater than or equal to 7 to less than 12 years of age at time of consent/assent) and age Cohort 2 ( greater than or equal to 2 to less than 7 years of age).
干预措施: perampanel (Drug)
结局指标
主要结局
Apparent Clearance (CL/F) of Perampanel [Core Study]
时间窗: From Day 8 up to Day 78
CL/F was defined as the volume of plasma cleared of the drug per unit time. Blood samples were collected at day 8, Day 36, Day 64 , and Day 78. The CL/F values were calculated for each visit and averaged to derive the total CL/F value per arm. Data was analyzed for 2 categories: CYP3A4/5 inducers (carbamazepine, oxcarbazepine and phenytoin) and non-inducers. Data is presented as mean Liter per hour +/-standard deviation.
Steady-state Average Concentration (C av,ss) of Perampanel [Core Study]
时间窗: From Day 8 up to Day 78
C av,ss was calculated as 'Dose (mg)/Dosing Interval (24 h)/(CL/F \[L/h\]) x 1000'. C av,ss during a dosing interval was dose-normalized to 0.12 mg/kg in participants aged ≥ 2 to less than 12 years (intended to correspond to 8 mg/70 kg in adults/adolescents). Blood samples were collected at day 8, Day 36, Day 64 , and Day 78. C av,ss values were calculated for each visit and averaged to derive the total C av,ss value per arm. Data was analysed for 2 categories: CYP3A4/5 inducers (carbamazepine, oxcarbazepine and phenytoin) and non-inducers. Data is presented as mean Liter per hour +/- standard deviation.
次要结局
- Palatability Questionnaire Assessment - Would You/Your Child Have Preferred This Medicine to Have Been Flavored, e.g. Fruity [Core Study](Week 5 or at the time of early discontinuation)
- Percent Change From Baseline in Seizure Frequency Per 28 Days in Treatment Phase [Core Study](Baseline [2 weeks Pretreatment Phase (Visit 1) plus 4 weeks Prior to Pretreatment Phase], Week 0 to Week 15)
- Palatability Questionnaire Assessment - How Does This Medicine Smell [Core Study](Week 5 or at the time of early discontinuation)
- Seizure-free Rate During the Maintenance Period [Core Study](Week 9 to Week 11)
- The Clinical Global Impression of Change at the End of Treatment (EOT) [Core Study](Week 0 (Baseline), Week 11 or EOT)
- The Clinical Global Impression of Change During the Overall Treatment Duration by Visit and at EOT [Extension Phase](Week 0 (Baseline), Week 11, Week 28, Week 52 or EOT)
- 50% Responder Rate During the Maintenance Period-LOCF [Core Study](Baseline [2 weeks Pretreatment Phase (Visit 1) plus 4 weeks Prior to Pretreatment Phase], Week 9 to 11)
- Number of Participants With Treatment Emergent Non-Serious Adverse Events (AEs) and Treatment Emergent Serious Adverse Events (SAEs) as a Measure of Safety and Tolerability of Perampanel(For each participant, from the first treatment dose till 30 days after the last dose or up to Week 15 for Core Study and Week 56 for the Extension Phase)
- Palatability Questionnaire Assessment - How Does This Medicine Taste [Core Study](Week 5 or at the time of early discontinuation)
- Palatability Questionnaire Assessment - Based on Its Taste, Smell, and How it Felt in the Mouth, How Easy or Difficult Was it for You / Your Child to Take This Medicine Every Day [Core Study](Week 5 or at the time of early discontinuation)
- Percentage Change From Baseline in Seizure Frequency Per 28 Days During the Overall Treatment Duration by 13-week Intervals [Extension Phase](Baseline [2 weeks Pretreatment Phase (Visit 1) plus 4 weeks Prior to Pretreatment Phase], Weeks 1-13, Weeks 14-26, Weeks 27-39, and Weeks 40-52)
- 50 % Responder Rate During the Overall Treatment Duration by 13-week Intervals [Extension Phase](Baseline [2 weeks Pretreatment Phase (Visit 1) plus 4 weeks Prior to Pretreatment Phase], Weeks 1-13, Weeks 14-26, Weeks 27-39, and Weeks 40-52)
- Seizure-free Rate During the Overall Treatment Duration [Extension Phase](Baseline [2 weeks Pretreatment Phase (Visit 1) plus 4 weeks Prior to Pretreatment Phase], Weeks 1-13, Weeks 14-26, Weeks 27-39, and Weeks 40-52)