A Phase 1, Double-blind, Placebo-controlled, Multiple Ascending Dose Study to Determine the Safety, Tolerability, and Pharmacokinetics of SAGE-718 Oral Solution in Healthy Adults With an Open-label Cohort of Patients With Huntington's Disease
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 6
- 试验地点
- 1
- 主要终点
- Number of Participants with the Incidence of Adverse Events and Serious Adverse Events.
研究概览
简要总结
This study is a phase 1, double-blind, placebo-controlled, multiple ascending dose study to determine the safety, tolerability, and pharmacokinetics of SAGE-718 oral solution in healthy adults (Part A) with an open-label cohort of patients with Huntington's disease (Part B)
详细描述
This posting addresses Part B
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
- 18 Years 至 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subject is positive for mutant HTT (documented CAG repeats ≥ 40 units).
- •Subject has a body weight ≥50 kg and body mass index ≥18.0 and ≤30.0 kg/m2 at screening.
排除标准
- •Subject has any clinically significant abnormal finding on the physical exam at screening or admission.
- •Subject has a history or presence of a neurologic disease or condition (other than Huntington's disease), including but not limited to severe chorea, epilepsy, closed head trauma with clinically significant sequelae, or a prior seizure.
- •Subject has a family history of epilepsy.
- •Subject has a positive screening test for alcohol or drugs of abuse (including marijuana) at screening or admission.
研究组 & 干预措施
SAGE-718
干预措施: SAGE-718 (Drug)
结局指标
主要结局
Number of Participants with the Incidence of Adverse Events and Serious Adverse Events.
时间窗: 21 Days
Percentage of participants with change from baseline in vital signs.
时间窗: 21 Days
Percentage of participants with change from baseline in clinical laboratory parameters.
时间窗: 21 Days
Change from baseline in electrocardiograms (ECGs) including PR interval, QT interval, QTc interval, QTcF, and rhythm abnormalities
时间窗: 21 Days
Change from baseline in Columbia-Suicide Severity Rating Scale (C-SSRS).
时间窗: 21 Days
次要结局
- PK profile of SAGE-718 following administration of multiple doses of SAGE-718 Oral Solution as assessed by area under the curve [AUC].(17 Days)
- PK profile of SAGE-718 following administration of multiple doses of SAGE-718 Oral Solution as assessed by maximum observed concentration [Cmax].(17 Days)
- PK profile of SAGE-718 following administration of multiple doses of SAGE-718 Oral Solution as assessed by time of occurrence of Cmax [tmax].(17 Days)