Evaluation of Vesiculogenesis in Children With GH Deficiency Undergoing Hormone Replacement Therapy
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 10
- 试验地点
- 1
- 主要终点
- Derivational profile of extracellular vesicles
研究概览
简要总结
The primary objective of the study is to evaluate the size and derivational profile of the extracellular vescicles (EV) generated in children with GH deficiency, undergoing hormone replacement therapy with rhGH. Secondary objectives is to correlate vesiculogenesis with auxometric and biochemical parameters used in clinical-endocrine practice in the evaluation of short stature.
The results of the study will provide useful information to more rationally set up the clinical and biochemical follow-up of hormone replacement therapy with rhGH, as well as to understand the molecular and cellular mechanisms underlying the multi-systemic action of GH, the most important anabolic hormone of the human organism.
详细描述
Methods:
At the Regional Reference Center for Growth Disorders, Istituto Auxologico Italiano, IRCCS, Milan, 10 children of both sexes affected by isolated GH deficiency will be recruited according to the criteria set out in AIFA note 39 for this pathology (short stature: ≤ - 3 SD or ≤ -2 SD and growth velocity/year ≤ -1.0 SD for age and sex evaluated at least 6 months apart and peak GH at two different stimulating pharmacological tests < 8 ng/ml). The exclusion criterion from the present study (and from treatment with rhGH) will be the presence of organic pathologies at the hypothalamic-pituitary level (assessed by brain MRI).
In basal conditions (pre-treatment), we will proceed with the collection of clinical and anthropometric/auxometric data, including the evaluation of body composition with bioimpedance analysis. The same work-up will be repeated at the subsequent 6-month follow-up (see below).
Each subject will undergo hormone replacement therapy with rhGH at a dose of 0.025-0.035 mg/kg of body weight per day (or 0.7-1.0 mg/m2 of body surface area per day).
In conditions of fasting (at least 12 hours), blood samples will be taken at T0 (i.e., before the start of hormone replacement therapy with rhGH) and at T6 (i.e., after 6 months of rhGH, approximately 10-12 hours after administration of the previous day's dose).
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 5 Years 至 15 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •short stature: ≤ - 3 SD or ≤ -2 SD
- •growth velocity/year ≤ -1.0 SD for age and sex evaluated at least 6 months apart - peak GH at two different stimulus pharmacological tests < 8 ng/ml
排除标准
- •presence of organic pathologies at the hypothalamic-pituitary level (assessed by performing brain MRI).
研究组 & 干预措施
Hormone replacement therapy with rhGH
干预措施: rhGH (Drug)
结局指标
主要结局
Derivational profile of extracellular vesicles
时间窗: Baseline and after 6 months
Change in derivational profile of extracellular vesicles after rhGH treatment
Size of extracellular vesicles
时间窗: Baseline and after 6 months
Change in size of extracellular vesicles after rhGH treatment
次要结局
- Auxometric parameters - height(Baseline and after 6 months)
- Biochemical parameters - Insulin-like growth factor I (IGF-I)(Baseline and after 6 months)
- Auxometric parameters - weight(Baseline and after 6 months)
- Biochemical parameters - glycemia(Baseline and after 6 months)