Recombinant Human Insulin-Like Growth Factor (rhIGF-1) Treatment of Short Stature Associated With Primary IGF-1 Deficiency: A Multi-Center, Open Label, Concentration-Controlled Study
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- Ipsen
- 入组人数
- 45
- 试验地点
- 1
- 主要终点
- Height Velocity From Pretreatment (Week 0) to Week 34
研究概览
简要总结
This study is intended to assess the effects of once daily dosing of recombinant human insulin-like growth factor (rhIGF-1) in increasing height velocity.
详细描述
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 growth hormone (GH) action. In this protocol, primary IGFD is defined as short stature (<-2 standard deviations [SDs] below the mean for age and gender), and abnormal serum IGF-1 (<-2 SDs below the mean for age and gender).
The trial is an open-label, concentration-controlled trial conducted at up to 20 centers throughout the United States.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 3 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Chronological age ≥ 3
- •Chronological age or bone age ≤ 12 for boys and ≤ 11 for girls
- •Prepubertal at Visit 1
- •Height SD score of < -2
- •IGF-1 SD score of < -2
排除标准
- •Prior treatment with GH, IGF-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
rhIGF-1 QD
Subjects received subcutaneous injection (SC) injections of rhIGF-1 once a day.
干预措施: rhIGF-1 (mecasermin) for a period of 86 weeks (Drug)
结局指标
主要结局
Height Velocity From Pretreatment (Week 0) to Week 34
时间窗: Pretreatment to Week 34
Height was 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. The subject was repositioned between each measurement. Height velocity during an interval of time is defined as the change in height during the time interval divided by the duration of the time interval. Missing Week 34 heights were imputed using the last height SD score carried forward.
Height Velocity From Week 34 to 86
时间窗: Week 34 to 86
Height was 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. The subject was repositioned between each measurement. Height velocity during an interval of time is defined as the change in height during the time interval divided by the duration of the time interval. Missing Week 86 heights were imputed using the last height SD score carried forward.
次要结局
- Change in Height SD Score From Pretreatment to Week 34(Pretreatment and Week 34)
- Change in Height SD Score From Pretreatment to Week 86(Pretreatment and Week 86)
- Change in Bone Age From Pretreatment to Week 86 Minus Change in Chronological Age(Pretreatment to Week 86)
- Percent Change in Serum Concentration of IGFBP-1, IGFBP-2 and IGFBP-3 From Pretreatment to Week 86(Pretreatment and Week 86)
- Percent Change in Serum Concentration of ALS From Pretreatment to Week 86(Pretreatment and Week 86)