Recombinant Human Insulin-Like Growth Factor-1 (IGF-1) Treatment of Children With Growth Failure Associated With Primary IGF-1 Deficiency: An Open-Label, Multi-Center, Extension Study
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 发起方
- Ipsen
- 入组人数
- 114
- 试验地点
- 1
- 主要终点
- Height Velocity During BID Dosing Period
研究概览
简要总结
This is an extension study to Tercica study MS301 (NCT00125164) and is intended to collect long term safety and efficacy data on the continued use of recombinant human insulin-like growth factor-1 (rh IGF-1) in children and adolescents treated for primary IGF-1 deficiency (IGFD). The secondary objective is to use the data collected to learn more about the relationship of IGF-1 exposure to the promotion of normal growth and pubertal development.
详细描述
Primary IGFD is a term that has been used to describe patients with intrinsic cellular defects in GH action. In this protocol, subjects that have completed one year of mecasermin treatment on Tercica protocol MS301 (NCT00125164) will be allowed to enroll in this extension study. All subjects were planned to receive treatment.
This is a Phase IIIb open-label, multi-center, parallel dose, extension study conducted in approximately 40 centers across 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
- 4 Years 至 15 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Parents or legally authorized representatives must give signed informed consent before any trial related activities are conducted
- •Where required, assent of the subject will be appropriately documented prior to any study related activities
- •Completion of assessments at Visit 9 (Month 120 of Study MS301 [NCT00125164])
排除标准
- •Incomplete participation in MS301 (NCT00125164)
- •Known or suspected allergy to the trial product (mecasermin, recombinant human IGF-1 injection) or its formulation
- •Development or presence of a chronic condition except as approved by the Medical Monitor
- •Pregnancy
- •Any social or medical condition that, in the opinion of the investigator, would be detrimental to either the subject or the study
研究组 & 干预措施
All rhIGF-1 Subjects
All subjects entering MS306 began recombinant human insulin-like growth factor-1 (rhIGF-1) twice a day (BID) treatment. Each subject treated in MS301 had an MS306 starting dose that was based on their dose at the completion of MS301 (i.e. subcutaneous injections of rhIGF-1 at 40, 80, or 120 micrograms [μg]/ kilogram [kg] BID).
MS301 untreated control subjects were randomised in MS306 in a 1:1 ratio to a dose of either 80 or 120 μg/kg rhIGF-1 BID.
Following Protocol Amendment 1, all subjects received either 80 or 120 μg/kg rhIGF-1 BID until the implementation of Protocol Amendment 2.
Following Protocol Amendment 2, all subjects were first switched to receive subcutaneous injections of 160 μg/kg rhIGF-1 once a day (QD), followed by individual dose-escalation first to 200 μg/kg rhIGF-1 QD and subsequently to a targeted maximum dose of 240 μg/kg rhIGF-1 QD. Subjects were treated QD until the early termination of the study.
干预措施: rh IGF-1 (mecasermin) (Drug)
结局指标
主要结局
Height Velocity During BID Dosing Period
时间窗: At Years 1, 2 and 3 in BID dosing period.
Height was measured standing, without shoes, as the average of 3 measurements by the same observer identical technique with a Harpenden or other wall-mounted stadiometer at baseline and each study visit up to 3 years. Height velocity (during any interval of time (annualised) is computed as (height on date 2 - height on date 1)/(age on date 2 - age on date 1) where height is expressed as centimetres so that height velocity is expressed as centimetres per year (cm/yr). Height velocity is presented for subjects completing each year of BID treatment (i.e. Year 1 \[0-1 years\], Year 2 \[1-2 years\], Year 3 \[2-3 years\]).
次要结局
- Mean Change From Baseline in Height Standard Deviation (SD) Score During BID Dosing Period(At baseline and Years 1, 2 and 3 in BID dosing period.)
- Mean Change From Baseline in Body Mass Index (BMI) SD Score During BID Dosing Period(At baseline and Years 1, 2 and 3 in BID dosing period.)
- Mean Change From Baseline in Predicted Adult Height During BID Dosing Period(At baseline and Years 1, 2 and 3 in BID dosing period.)
- Mean Change From Baseline in Bone Age During BID Dosing Period(At baseline and Years 1, 2 and 3 in BID dosing period.)