Evolution Of Growth Rate In Children Suffering From A Disease Associated With Growth Retardation and Treated By Genotonorm. A Pilot Study.
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Pfizer
- 入组人数
- 46
- 试验地点
- 1
- 主要终点
- Change From Baseline in Annual Growth Rate Standard Deviation Score (SDS) for Chronological Age (CA) at Month 36 in Intent-to-Treat (ITT) Population
研究概览
简要总结
To show an increase in annual growth rate 3 years after Visit 2. Annual growth rate in standard deviation (SD) after 3 years will be compared to growth rate before the start of GH treatment.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 11 Years 至 13 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Bone age < 13 years for a boy and < 11 years for a girl
- •Naive child: Measured Height < -2.5 SD for CA
- •Child currently treated by GH
排除标准
- •Idiopathic short stature
- •Syndrome known to be associated with an increased risk of cancer e.g. family history of adenomatous polyposis
研究组 & 干预措施
Somatropin
干预措施: Somatropin (Drug)
结局指标
主要结局
Change From Baseline in Annual Growth Rate Standard Deviation Score (SDS) for Chronological Age (CA) at Month 36 in Intent-to-Treat (ITT) Population
时间窗: Baseline, Month 36
Change in annual growth rate (AGR) standard deviation score (SDS) for chronological age (CA) derived by subtracting AGR SDS CA at baseline from each time point (Yx) value. AGR at Yx= (height Yx-height Y\[x-1\])/(\[date of Yx-date of Y{x-1}\]/365.25). AGR as SDS calculated using Sempe reference means and standard deviations (SD) for growth rate. AGR SDS CA (for both baseline and Yx)= (AGR-reference mean for growth rate CA)/reference SD for growth rate CA. CA calculated as integer (Date of height measurement-Date of birth)/365.25\*12. SDS indicates how similar participant was to reference population.
Change From Baseline in Annual Growth Rate Standard Deviation Score (SDS) for Chronological Age (CA) at Month 36 in Per-Protocol (PP) Population
时间窗: Baseline, Month 36
Change in annual growth rate (AGR) standard deviation score (SDS) for chronological age (CA) derived by subtracting AGR SDS CA at baseline from each time point (Yx) value. AGR at Yx= (height Yx-height Y\[x-1\])/(\[date of Yx-date of Y{x-1}\]/365.25). AGR as SDS calculated using Sempe reference means and standard deviations (SD) for growth rate. AGR SDS CA (for both baseline and Yx)= (AGR-reference mean for growth rate CA)/reference SD for growth rate CA. CA calculated as integer (Date of height measurement-Date of birth)/365.25\*12. SDS indicates how similar participant was to reference population.
次要结局
- Change From Baseline in Annual Growth Rate at Month 12, Month 24 and Month 36 in ITT Population(Baseline, Month 12, Month 24, Month 36)
- Change From Baseline in Annual Growth Rate at Month 12, Month 24, Month 36 in PP Population(Baseline, Month 12, Month 24, Month 36)
- Height(Baseline, Month 12, Month 24, Month 36)
- Change From Baseline in Height at Month 12, Month 24 and Month 36(Baseline, Month 12, Month 24, Month 36)
- Mean Height Standard Deviation Score (SDS) for Chronological Age (CA)(Baseline, Month 12, Month 24, Month 36)
- Change From Baseline in Height Standard Deviation Score (SDS) for Chronological Age (CA) at Month 12, Month 24 and Month 36(Baseline, Month 12, Month 24, Month 36)
- Mean Height Standard Deviation Score (SDS) for Bone Age (BA)(Baseline, Month 12, Month 24, Month 36)
- Change From Baseline in Height Standard Deviation Score (SDS) for Bone Age (BA) at Month 12, Month 24 and Month 36(Baseline, Month 12, Month 24, Month 36)
- Change From Baseline in Annual Growth Rate Standard Deviation Score (SDS) for Chronological Age (CA) at Month 12 and Month 24 in ITT Population(Baseline, Month 12, Month 24)
- Change From Baseline in Annual Growth Rate Standard Deviation Score (SDS) for Chronological Age (CA) at Month 12 and Month 24 in PP Population(Baseline, Month 12, Month 24)
- Mean Growth Rate Standard Deviation Score (SDS) for Bone Age (BA)(Month 12, Month 24, Month 36)
- Body Mass Index (BMI)(Baseline, Month 12, Month 24, Month 36)
- Change From Baseline in Body Mass Index (BMI) at Month 12, Month 24 and Month 36(Baseline, Month 12, Month 24, Month 36)
- Change From Baseline in Bone Age (BA) at Month 12, Month 24 and Month 36(Baseline, Month 12, Month 24, Month 36)
- Ratio of Bone Age (BA) to Chronological Age (CA)(Baseline, Month 12, Month 24, Month 36)