A Phase IV Open-label Study of Predictive Markers in Growth Hormone Deficient Pre-pubertal Children Treated With Saizen®
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 入组人数
- 214
- 主要终点
- Change From Baseline in Serum Insulin Like Growth Factor-1 Standard Deviation Score (IGF-1 SDS) Levels at Week 4
研究概览
简要总结
This is an open-label, prospective, multicentric, non-comparative, non-randomized Phase IV interventional study in which subjects pre-diagnosed with Growth Hormone Deficiency (GHD) were treated for 4 weeks with Saizen to compare the response between GHD children born appropriate for gestational age (AGA) and those born small for gestation age (SGA) after 4 weeks of Saizen therapy.
详细描述
The response to growth hormone (GH) treatment, short-term as well as long-term, displays considerable inter individual variability. This is particularly evident for the endpoint of paediatric GH administration, that is (i.e.) the growth response, which is pronounced in children who are affected by GHD. This is an open-label, multicentric study in which subjects pre-diagnosed with GHD were treated for 4 weeks with Saizen. Two hundred fourteen GHD evaluable pre-pubertal subjects were planned to be recruited in approximately 9 sites in China. Demographic data, medical history, tanner stage, physical examination, body weight, height, bone age measurement, body mass index, review of baseline medications and procedures and blood sampling were performed at baseline visit, end of treatment visit (week 4) and at 4 week follow-up visit.
OBJECTIVES
Primary objective:
- To compare the response between GHD children born AGA and those born SGA after 4 weeks of Saizen therapy
Secondary Objectives:
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
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male and female subjects with documented pre-established diagnosis of GHD with a GH peak response of <10 microgram/liter (mcg/L) with 2 GH stimulation tests, without priming with estradiol
- •Subjects with SGA defined as birth weight and/or length at least 2 standard deviations (SDs) below the mean for gestational age
- •Subjects with prepubertal status according to Tanner
- •Subjects with pre-established history of normal thyroid function or adequate substitution for at least 3 months
- •Subjects with weight for stature within the population specific normal range (>5th and <95th percentiles) for gender
- •Subjects with willingness and ability to comply with the protocol for the duration of the study
- •Subjects whose parents or guardians written informed consent given before any study-related procedure that was not part of the subjects normal medical care, with the understanding that the subject or parent/guardian might withdraw consent at any time without prejudice to future medical care. If the child was old enough to read and write, a separate assent form was given
排除标准
- •Subjects who acquired GHD due to central nervous system tumor, trauma, infection, infiltration (documented by imaging), and history of irradiation or cranial surgery
- •Subjects with previous treatment with GH, growth hormone releasing hormone (GHRH), anabolic steroids or any treatment affecting growth
- •Subjects who had previous treatment with corticosteroids, except in case of topical or inhaled corticosteroid administration for atopic disease. Corticosteroids for hormonal substitution were also allowed if the condition and the treatment regimen had been stable for at least 3 months
- •Subjects with severe associated pathology affecting growth such as malnutrition, malabsorption, or bone dysplasia
- •Subjects with chronic severe kidney disease
- •Subjects with chronic severe liver disease
- •Subjects with chronic infectious disease
- •Subjects with acute or severe illness during the previous 6 months
- •Subjects with significant concomitant illness that would interfere with participation or assessment in this study
- •Subjects who had active malignancy (except non-melanomatous skin malignancies that had undergone surgical excision and/or biopsy, diagnosis and treatment to resolution)
- •Subjects with history or active idiopathic intra-cranial hypertension (benign intracranial hypertension or pseudo-tumor cerebri)
- •Subjects with diabetes mellitus type I & II
- •Subjects with any autoimmune disease
- •Subjects who had previous screening failure in this study
- •Subjects who had used an investigational drug or participated in another clinical study within the last 3 months
结局指标
主要结局
Change From Baseline in Serum Insulin Like Growth Factor-1 Standard Deviation Score (IGF-1 SDS) Levels at Week 4
时间窗: Baseline and Week 4
Insulin Like Growth Factor-1 Standard Deviation Score (IGF-1 SDS) was calculated as logarithm (log) 10 actual value of IGF-1 - log 10 (mean reference value of IGF-1) divided by log10 reference standard deviation of IGF-1.
次要结局
- Change From Baseline in Insulin Like Growth Factor Binding Protein-3 (IGFBP-3) Levels at Week 4(Baseline and Week 4)
- Change From Baseline in Fasting Glucose at Week 4(Baseline and Week 4)
- Change From Baseline in Fasting Insulin at Week 4(Baseline and Week 4)
- Change From Baseline in Homeostasis Model Assessment of Insulin Resistance (HOMA-IR) Test at Week 4(Baseline and Week 4)
- Change From Baseline in Lipid Profile at Week 4(Baseline and Week 4)