A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 121
- 试验地点
- 24
- 主要终点
- Change From Baseline in Annualized Growth Velocity (AGV) at Week 52
研究概览
简要总结
The intent and design of this Phase 3 study is to assess BMN 111 as a therapeutic option for the treatment of children with Achondroplasia.
详细描述
This is a Phase 3 randomized, placebo-controlled, double-blind multicenter study with approximately 110 subjects, aged 5 to < 18 years old. Subjects with documented Achondroplasia confirmed by genetic testing will have been enrolled in Study 111-901 for at least a 6-month period immediately before entering into the 111-301 study. Eligible subjects will be randomly assigned to one of two treatment groups: placebo or BMN 111 at 15 μg/kg. The route of administration is subcutaneous injection and the frequency is daily.
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)
入排标准
- 年龄范围
- 5 Years 至 18 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
Active BMN 111
Daily subcutaneous injection of 15 micrograms per kilogram BMN111
干预措施: BMN 111 (Drug)
Placebo
Daily subcutaneous injection of placebo
干预措施: Placebo (Drug)
结局指标
主要结局
Change From Baseline in Annualized Growth Velocity (AGV) at Week 52
时间窗: At Baseline and Week 52
AGV at a Post-baseline Visit is defined as \[(Height at Post-baseline Visit - Height at Baseline)/(Date of Post-baseline Visit - Date of Baseline Assessment)\] x 365.25 AGV at Baseline is defined as \[(Height at Baseline - last height measurement in Study 111-901 at least 6 months prior to Baseline)/(Date of Baseline Assessment - Date of last height measurement in Study 111-901 at least 6 months prior to Baseline)\] x 365.25
次要结局
- Change From Baseline in Height Z-score at Week 52(At baseline and Week 52)
- Change From Baseline in Upper to Lower Segment Body Ratio at Week 52(At baseline and Week 52)
- Summary of Subjects Experiencing Adverse Events (AEs) During Treatment(Up to Week 56)