A Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Phase 3 Study to Evaluate the Efficacy and Safety of OV101 in Pediatric Individuals With Angelman Syndrome
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 104
- 试验地点
- 1
- 主要终点
- Clinical Global Impressions- Improvement in Angelman Syndrome (CGI-I-AS) Rating at 12 Weeks
研究概览
简要总结
The purpose of this study is to assess the efficacy and safety of oral OV101 (gaboxadol) in pediatric subjects with Angelman syndrome.
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
- Double (Participant, Investigator)
盲法说明
Double (Participant/Care Giver and Investigator/Outcomes Assessor)
入排标准
- 年龄范围
- 2 Years 至 12 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female and 2 to 12 years old (inclusive) at the time of informed consent
- •Confirmed molecular diagnosis of AS
- •Has a CGI-S-AS score of 3 or more at baseline.
- •Meets the following age-appropriate body weight criterion:
- •Subjects 2 to 3 years old must have a minimum body weight of 9 kg.
- •Subjects 4 years and older must be between 17 kg and 64 kg (inclusive).
- •Stable concomitant mediations for at least 4 weeks before study start
排除标准
- •Any condition that would limit study participation
- •Clinically significant lab or vital sign abnormalities at the time of screening
- •Poorly controlled seizures (weekly seizures of any frequency with a duration more than 3 minutes, weekly seizures occurring more than 3 times per week, each with a duration of less than 3 minutes, or as defined by investigator assessment)
- •Use of prescription medications for sleep, minocycline, or levodopa within the 4 weeks prior to Day 1 or during the study. Benzodiazepines chronically administered for seizure control are permitted.
- •Cannot comply with protocol study assessments during screening or caregiver unable to comply with study requirements.
- •Enrolled in any clinical trial or used any investigational agent within the 30 days before screening or concurrently with this study.
研究组 & 干预措施
OV101 once daily (weight-based dosing) Other Name:Gaboxadol
OV101 (gaboxadol), oral, provided once daily at bedtime for 12 week duration
干预措施: Gaboxadol (Drug)
Placebo once daily
Matching placebo,oral, provided once daily at bedtime for 12 week duration
干预措施: Placebo (Drug)
结局指标
主要结局
Clinical Global Impressions- Improvement in Angelman Syndrome (CGI-I-AS) Rating at 12 Weeks
时间窗: 12 weeks
To evaluate the efficacy of OV101 versus placebo as assessed by the Clinical Global Impressions-Improvement-Angelman syndrome (CGI-I-AS) score at Week 12. CGIA-I-AS scores range from 1 (very much improved), to 4 (no change), to 7 (very much worse).
次要结局
未报告次要终点