Phase 3 Clinical Trial Evaluating the Efficacy and Safety of Navepegritide and Lonapegsomatropin in Achondroplasia (cAtCH)
A Phase 3, Randomized, Double-blind, Active Controlled, Multi-center Trial Investigating the Efficacy and Safety of Dual Therapy With Once-weekly Navepegritide and Once-weekly Lonapegsomatropin Compared to Navepegritide Monotherapy and Lonapegsomatropin Monotherapy in Children and Adolescents With Achondroplasia
This pivotal trial is being conducted to evaluate the efficacy, safety and tolerability of dual therapy with navepegritide and lonapegsomatropin administered as separate subcutaneous (SC) injections once weekly in children with achondroplasia (ACH) aged 2 to 17 years.
研究概览
地位
条件
研究类型
注册 (估计的)
阶段
- 第三阶段
联系人和位置
学习联系方式
- 姓名：Ascendis Registry Inquiries
- 电话号码：+45 61242484
- 邮箱：asnd_registryinquiries@ascendispharma.com
参与标准
资格标准
适合学习的年龄
接受健康志愿者
有资格学习的性别
描述
Inclusion Criteria:
- 1. Age 2 to <18 years at randomization
- 2. Clinical diagnosis of ACH with historical genetic confirmation
- 3. At least 1 height measurement available from medical records
Exclusion Criteria:
- 1. Treatment with growth hormone within 1 year prior to randomization
- 2. Prior treatment with certain medication(s) which potentially could have affected growth (prior treatment with vosoritide is not exclusive)
- 3. History of any surgery intended to affect stature, growth, or body proportionality except procedures for leg bowing
- 4. Unable to stand without assistance
- 5. Known or suspected medical conditions other than achondroplasia that could affect growth
- 6. Known history or presence of injury of the growth plates that affects growth potential of long bones
- 7. Known history or presence of malignant disease
学习计划
研究是如何设计的？
设计细节
- 主要用途：治疗
- 分配：随机化
- 介入模型：并行分配
- 屏蔽：四人间
武器和干预
参与者组/臂 |
干预/治疗 |
|---|---|
|
实验性的：Once weekly navepegritide SC and lonapegsomatropin SC
|
Administered weekly as two separate SC injections. Treatment duration of up to 104 weeks |
|
实验性的：Once weekly navepegritide SC and placebo for lonapegsomatropin SC
|
Administered as two separate SC injections. Treatment duration of up to 52 weeks of navepegritide and placebo for lonapegsomatropin followed by 52 weeks of navepegritide and lonapegsomatropin. |
|
实验性的：Once weekly lonapegsomatropin SC and placebo for navepegritide SC
|
Administered as two separate SC injections. Treatment duration of up to 52 weeks of lonapegsomatropin and placebo for navepegritide followed by 52 weeks of navepegritide and lonapegsomatropin. |
研究衡量的是什么？
主要结果指标
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Annualized Growth Velocity (AGV)
大体时间：52 weeks
|
To evaluate the AGV, cm per year, of dual therapy with navepegritide and lonapegsomatropin compared to navepegritide alone |
52 weeks
|
次要结果测量
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Treatment-emergent Adverse Events (AE)
大体时间：Throughout trial duration 104 weeks
|
Number of events |
Throughout trial duration 104 weeks
|
|
Annualized Growth Velocity (AGV)
大体时间：52 weeks
|
To evaluate the AGV, cm per year, of dual therapy with navepegritide and lonapegsomatropin compared to lonapegsomatroptin alone |
52 weeks
|
|
Annualized Growth Velocity (AGV) change from baseline
大体时间：52 weeks
|
To evaluate the change from baseline in AGV, cm per year |
52 weeks
|
|
ACH-specific height Z-score
大体时间：52 weeks
|
To evaluate the change from baseline in ACH-specific height Z-score |
52 weeks
|
|
CDC-based height Z-score
大体时间：52 weeks
|
To evaluate the change from baseline in CDC-based height Z-score |
52 weeks
|
|
Bone safety
大体时间：Throughout trial duration 104 weeks
|
Qualitative assessment with radiology |
Throughout trial duration 104 weeks
|
|
Arm span Z-score
大体时间：52 weeks
|
Change from baseline in arm span Z-score. |
52 weeks
|
|
Upper to lower body segment ratio
大体时间：52 weeks
|
Change from baseline in upper to lower body segment ratio. |
52 weeks
|
|
Arm span
大体时间：52 weeks
|
Change from baseline in arm span, cm. |
52 weeks
|
|
Interpedicular distance (IDP)
大体时间：52 weeks
|
Change from baseline in IDP (L1-L4 average), mm |
52 weeks
|
合作者和调查者
调查人员
- 研究主任：Medical Director, MD、Ascendis Pharma A/S
研究记录日期
研究主要日期
学习开始 (估计的)
初级完成 (估计的)
研究完成 (估计的)
研究注册日期
首次提交
首先提交符合 QC 标准的
首次发布 (实际的)
研究记录更新
最后更新发布 (实际的)
上次提交的符合 QC 标准的更新
最后验证
更多信息
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