NCT06455059进行中（未招募）3 期
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Vosoritide in Children With Hypochondroplasia
适应症
干预措施
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 80
- 试验地点
- 23
- 主要终点
- Change from baseline in annualized growth velocity (AGV) at Week 52 versus placebo
研究概览
简要总结
The intent and design of this Phase 3 study is to assess vosoritide as a therapeutic option for the treatment of children with hypochondroplasia (HCH).
详细描述
This is a Phase 3 randomized, stratified, placebo-controlled, double-blind multicenter study to evaluate the effect of 52 weeks of daily vosoritide administration on annualized growth velocity (AGV) in participants with HCH. Eligible participants with documented HCH confirmed by genetic testing will roll over from Study 111-902 and enter the 111-303 study. Participants will be randomly assigned to one of two treatment groups: Placebo or Vosoritide. The route of administration is subcutaneous injection, and the frequency of administration is daily.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 3 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants must be ≥ 3 to < 18 years of age at enrollment
- •A confirmed genetic diagnosis of HCH
- •A height Z score of ≤ - 2.0 standard deviations (SDs) in reference to the general population of the same age and sex, as calculated using the Center for Disease Control and Prevention (CDC) growth charts
- •Males and females are eligible to participate in this clinical study.
- •Females ≥ 10 years old or who have begun menses must have a negative pregnancy test at the Screening Visit and be willing to have additional pregnancy tests during the study.
- •If sexually active, participants must be willing to use a highly effective method of contraception while participating in the study.
排除标准
- •Short stature condition other than HCH
- •Have an unstable condition likely to require surgical intervention during the study.
- •Evidence of decreased growth velocity and/or growth plate closure
- •Taking any of the prohibited medications
- •Treated with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids
- •Planned or expected to have limb-lengthening surgery during the study period.
- •Planned or expected bone-related surgery (ie, surgery involving disruption of bone cortex, excluding tooth extraction), during the study period
- •Require any investigational agent prior to completion of study period.
- •Received vosoritide or another investigational product or investigational medical device in the past
- •Have used any investigational product or investigational medical device for the treatment of HCH or short stature at any time, including vosoritide
- •Have current malignancy, history of malignancy, or currently under work-up for suspected malignancy.
- •Have known hypersensitivity to vosoritide or its excipients.
- •Concurrent disease or condition that, in the view of the investigator, would interfere with study participation or safety evaluations, for any reason.
研究组 & 干预措施
vosoritide injection with vial and syringe
Experimental
干预措施: Vosoritide (Drug)
Placebo injection with vial and syringe
Placebo Comparator
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 versus placebo
时间窗: At week 52
次要结局
- Change from baseline in height Z-score at Week 52 versus placebo(At week 52)
- Change from baseline in standing height at Week 52 versus placebo(At week 52)
研究者
研究点 (23)
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