A Phase 2 Adult and Adolescent Angelman Syndrome Clinical Trial: A Randomized, Double-Blind, Safety and Efficacy Study of Gaboxadol
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- Healx AI
- 入组人数
- 88
- 试验地点
- 1
- 主要终点
- Incidence of Adverse Events in Placebo and Active Treatment Groups
研究概览
简要总结
The purpose of the study is to assess the safety and tolerability of oral OV101 (gaboxadol) in adult and adolescent subjects with Angelman syndrome. In addition, several exploratory efficacy outcome measures will be investigated.
详细描述
Two dosing schedules of OV101 (gaboxadol) giving as once daily or twice daily dose will be assessed against placebo.
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 13 Years 至 49 Years（Child, Adult）
- 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
OV101 regimen 1
OV101 once daily
干预措施: OV101 Regimen 1 (Drug)
OV101 regimen 2
OV101 twice daily
干预措施: OV101 regimen 2 (Drug)
Placebo
Twice daily
干预措施: Placebo (Other)
结局指标
主要结局
Incidence of Adverse Events in Placebo and Active Treatment Groups
时间窗: Baseline and Week 12
Treatment-related TEAEs (Treatment Emergent Adverse Event) in ≥ 2 Subjects in OV101 Combined, Safety Set. The incidence of TEAEs assessed as treatment-related (at least possibly related to study drug, by the Investigator). Preferred Term in the table below.
次要结局
未报告次要终点