Recombinant Human Insulin-Like Growth Factor (rhIGF-1) Treatment of Prepubertal Children With Growth Failure Associated With Primary IGF-1 Deficiency: A Phase 3, Randomized, Open Label, Observation-Controlled, Multicenter, Parallel-Dose Comparison Trial
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Ipsen
- 入组人数
- 137
- 试验地点
- 1
- 主要终点
- Height Velocity During the First Year - Intent to Treat (ITT)Population
研究概览
简要总结
This study is intended to determine whether twice daily weight based dosing with recombinant human insulin-like growth factor (rhIGF-1) will safely and effectively increase the growth of prepubertal children with short stature associated with low IGF-1 levels but who produce sufficient growth hormone (GH). Subjects will be randomized to either an observation arm or to active treatment.
详细描述
Prepubertal growth failure associated with primary IGF-1 deficiency (IGFD). Primary IGFD is a term that has been used to describe patients with intrinsic cellular defects in GH action. In this protocol, primary IGFD is defined as short stature (height standard deviation score[SDS]<-2 below the mean for age and gender), low serum IGF-1 (IGF-1 SDS <-2 below the mean for age and gender), and levels of growth hormone (GH) that are normal (≥7ng/mL) after a GH stimulation test. Primary IGFD is believed to result from a lower than normal ability to produce IGF-1 when exposed to normal levels of GH, i.e., a type of GH insensitivity or GH resistance.
This trial is one year, randomized, open label, observation-controlled, parallel-dose comparison efficacy and safety study conducted in approximately 40 centers across the United States.
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 12 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Chronological age ≥ 3 and chronological or bone age less than or equal to 11 years inclusive in girls;
- •Chronological age ≥ 3 and chronological or bone age less than or equal to 12 years inclusive for boys
- •Prepubertal
- •Height SD score of < -2
- •IGF-1 SD score of < -2
排除标准
- •Prior treatment with rhGH, rhIGF-1, or other growth-influencing medications
- •Growth failure associated with other identifiable causes (e.g., syndromes, chromosomal abnormality)
- •Chronic illness such as diabetes, cystic fibrosis, etc.
研究组 & 干预措施
40 μg/kg BID (twice daily dosing)
Injection of rhIGF-1 40 μg/kg BID. Per protocol amendment these subjects were reassigned to receive 120 μg/kg BID. Due to the dose change, the efficacy results for these subjects were analysed in a separate subanalysis. For all outcome measures, mean and standard deviations were not calculated for this arm.
干预措施: rhIGF-1 (mecasermin, Tercica, Inc.) (Drug)
80 μg/kg BID (twice daily dosing)
Injection of rhIGF-1 80 μg/kg BID
干预措施: rhIGF-1 (mecasermin, Tercica, Inc.) (Drug)
120 μg/kg BID (twice daily dosing)
Injection of rhIGF-1 120 μg/kg BID
干预措施: rhIGF-1 (mecasermin, Tercica, Inc.) (Drug)
结局指标
主要结局
Height Velocity During the First Year - Intent to Treat (ITT)Population
时间窗: Measured at baseline and at one year
Height to be measured standing without shoes as the average of three measurements by the same observer using identical technique with a Harpenden or other wall mounted stadiometer. Reposition subject between each measurement.
次要结局
- Change From Baseline in Height Standard Deviation (SD) Score at One Year - ITT Population(Measured at baseline and at one year)
- Changes in Bone Age From Baseline to One Year(Measured at baseline and at one year)
- Percent Changes From Baseline in Serum Concentrations of IGF-1 at One Year(Measured at baseline and at one year)
- Percent Changes From Baseline in Serum Concentrations of IGF-2 at One Year(Measured at baseline and at one year)
- Percent Changes From Baseline in Serum Concentrations of Insulin-like Growth Factor Binding Protein-2 (IGFBP-2) at One Year(Measured at baseline and at one year)
- Percent Changes From Baseline in Serum Concentrations of Insulin-like Growth Factor Binding Protein-3 (IGFBP-3) at One Year(Measured at baseline and at one year)
- IGF Generation Test: Change of Serum IGF-1 After 7 Days Exposure to Recombinant Human Growth Hormone (rhGH)(Study Day 1 and Day 7)
- IGF Generation Test: Change of Serum IGFBP-3 After 7 Days Exposure to Recombinant Human Growth Hormone (rhGH)(Study Day 1 and Day 7)