A Four Year Open Label Multi Center Randomized Two Arm Study Of Genotropin In Idiopathic Short Stature Patients: Comparing An Individualized, Target Driven Treatment Regimen To Standard Dosing Of Genotropin
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Pfizer
- 入组人数
- 316
- 试验地点
- 42
- 主要终点
- Absolute On-target Difference (AOTD) at 24 Months
研究概览
简要总结
To demonstrate that an individualized, formula-based Genotropin regimen for children with Idiopathic Short Stature will lead to a targeted height gain (to reach the target of 10th percentile (%), or -1.3 SDS) during 24 months of treatment. The endpoint at 4 years is to explore treatment efficiency over four years of two formula-based dose regimens (sub-arms) compared to standard treatment
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
- None
入排标准
- 年龄范围
- 3 Years 至 10 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Prepubertal children with bone ages between 3 and 10 years of age for males and 3 and 9 years of age for females
- •Naive to Growth Hormone treatment
排除标准
- •Abnormal karyotype. Small Gestational Age and Skeletal dysplasia.
研究组 & 干预措施
Standard
Standard daily HGH treatment
干预措施: Genotropin (Drug)
Formula-based
Formula-based dose regimen
干预措施: Genotropin (Drug)
结局指标
主要结局
Absolute On-target Difference (AOTD) at 24 Months
时间窗: 2 years
This was defined as an absolute difference between the 24-month height standard deviation score (SDS) and targeted 24-month height SDS (10th percentile (%), or -1.3 SDS). SDS indicates how similar the participant was to the reference population. These were calculated using 2000 Center for the Disease Control (CDC) growth reference tables (by age and gender).
次要结局
- Time Cost (Months Until >= -2 SDS)(2 years)
- Estimated Cost of Height Gain Estimated Until Full Adult Height (FAH) at 48 Months(4 years)
- Variability of Height SDS at 24 Months(2 years)
- Computed Cost of Height Gain at 48 Months(4 years)
- Change From Baseline in Height SDS at 48 Months.(4 years)