A Phase 1b/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Clinical Activity of ABS-1230 Administered to Pediatric and Young Adult Participants With KCNT1-Related Epilepsy
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 55
- 试验地点
- 1
- 主要终点
- Part 1: Safety and tolerability of ABS-1230 (incidence and severity of adverse events)
研究概览
简要总结
This trial will evaluate the safety, tolerability, pharmacokinetics, and clinical activity of ABS-1230 compared with placebo in participants with KCNT1-related epilepsy
详细描述
This is a Phase 1b/2 study that consists of 3 parts.
In part 1, participants will receive ABS-1230 for 12 weeks, with a follow-up period of 2 weeks.
In part 2, participants will receive ABS-1230 or placebo for 12 weeks, with a follow-up period of 2 weeks.
All participants who complete part 1 or part 2 will have the option to continue receiving ABS-1230 in an open-label extension study (part 3).
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)
盲法说明
Part 1 will be open-label. Part 2 of the study will be conducted in a randomized double-blind manner. Part 3 will be an open-label extension.
入排标准
- 年龄范围
- 1 Month 至 21 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Aged 1 month to <22 years
- •Clinician-confirmed diagnosis of KCNT1-related epilepsy
- •Has an average of at least 4 countable motor seizures per week
- •Is taking no more than 6 antiseizure medications (ASM) and is able to keep stable doses of ASMs for the duration of Part 1 or Part 2
排除标准
- •Is currently taking phenytoin, carbamazepine, stiripentol, or quinidine
- •Has a medical condition that, in the opinion of the investigator, would limit the participant's ability to participate in the study or might compromise participant safety or interfere with evaluation of the study drug.
研究组 & 干预措施
Placebo (Part 2)
干预措施: Placebo (Drug)
ABS-1230 (Part 1)
干预措施: ABS-1230 (Drug)
ABS-1230 (Part 3)
干预措施: ABS-1230 (Drug)
ABS-1230 (Part 2)
干预措施: ABS-1230 (Drug)
结局指标
主要结局
Part 1: Safety and tolerability of ABS-1230 (incidence and severity of adverse events)
时间窗: Measured from Day 1 to End of Study or Early Termination (up to 12 weeks)
Safety and tolerability of ABS-1230 by determining the incidence and severity of treatment emergent adverse events
Part 2: Efficacy of ABS-1230
时间窗: Measured from Day 1 to End of Study or Early Termination (up to 12 weeks)
Percentage change from baseline in countable motor seizures (normalized per 28 days) as recorded in the seizure diary
Part 3: Safety and tolerability of ABS-1230 (incidence and severity of adverse events)
时间窗: Measured from Day 1 of Part 3 to End of Study or Early Termination (up to 1 year)
Safety and tolerability of ABS-1230 by determining the incidence and severity of treatment emergent adverse events
次要结局
- Maximum Plasma Concentration [Cmax] of ABS-1230(Measured from Day 1 to End of Study or Early Termination (up to 12 weeks))
- Total Exposure [AUCtau] ABS-1230(Measured from Day 1 to End of Study or Early Termination (up to 12 weeks))