One Arm, Open Study to Assess Biochemical Markers of Growth Response to Growth Hormone Treatment in Children With Idiopathic Short Stature
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 入组人数
- 21
- 试验地点
- 1
- 主要终点
- Height
研究概览
简要总结
One arm, open, prospective, intervention study to assess biochemical markers of growth response to Growth Hormone treatment in 20 Children, aged 3-9 years old, with idiopathic short stature. All participants will be treated with Growth Hormone during the first year of the study (and then in accordance with the local ethic requirement, to supply drug which is not approved for the indication used in the study, for additional 3 years) and then will be followed up for the next 3 years. The impact of Growth Hormone therapy on clinical laboratory parameters that are indicative of the growth response will be assessed by collecting blood and urine samples during the 4 years study period. The primary endpoints are measurements of height and growth velocity during the year of Growth Hormone treatment, the height at the beginning of puberty and final height. Secondary endpoints are psychological parameters, assessed by questionnaires.
详细描述
One arm, open prospective intervention study to assess biochemical markers of growth response to Growth Hormone treatment in 20 children, aged 3-9 years old, with idiopathic short stature.
Objectives:
- To determine axiological and biochemical markers for growth response
- To assess the period of time necessary to determine the parameters which will differentiate between responders and non-responders
Inclusion criteria:
- Ages 3 to <9 years
- Short stature with height >2.25 Standard Deviation below the mean
- Prepubertal (Tanner stage I) at commencement of trial
- Peak Growth Hormone above 10ng/ml in at least one provocative test for Growth Hormone secretion
- Signing Informed consent forms
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
- 3 Years 至 9 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Ages 3 to <9 years
- •Short stature with height >2.25 Standard Deviation below the mean
- •Prepubertal (Tanner stage I) at commencement of trial
- •Peak Growth Hormone above 10ng/ml in at least one provocative test for Growth Hormone secretion
- •Signing informed consent forms
排除标准
- •Intra Uterine Growth Retardation
- •Growth retardation associated with malignancy, severe chronic disease, genetic syndromes and endocrine disorders
- •Treatment with any medical product which may interfere with Growth Hormone
研究组 & 干预措施
single arm
干预措施: Somatotropin growth hormone recombinant human (Drug)
结局指标
主要结局
Height
时间窗: every 4 months
Growth velocity
时间窗: every 4 months
Height at beginning of puberty
时间窗: At the biginning of puberty
Final height
时间窗: When acheiving final height
次要结局
- Psychological parameters(once a year)
- HbA1c and IGF-1(at baseline. after 3 months and than every 6 months)