New Potential Biomarkers for Diagnosis of Growth Hormone (GH) Deficiency and Early Assessment of Response to GH Treatment for Appropriate Personalization of Therapy
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 400
- 试验地点
- 1
- 主要终点
- Assessing new biomarkers for the diagnosis of GHD
研究概览
简要总结
This study aims at improving knowledge about the diagnosis of growth hormone deficiency (GHD) and treatment with growth hormone (GH), with the goal of providing information on the presence of new biomarkers, such as miRNAs, for diagnostic and therapeutic purposes, with the goal of establishing a personalized GH treatment scheme, optimizing resources, reducing costs, and improving outcomes.
详细描述
In 200 prepubertal and pubertal subjects with the suspicion of GHD, the levels of three specific circulating miRNas will be measured to establish whether they can be used for the diagnosis of GH deficiency. In subjects with isolated idiopathic GHD (IIGHD), short statured patients born SGA, Noonan and Turner syndromes, and SHOX deficiency, we will also assess the changes of the identified miRNAs before and after 3 months on GH therapy to verify an early growth prediction model for growth response at 12 months on treatment. Finally, we plan to identify any differences in these miRNAs at 3 months on treatment using daily GH versus weekly (long-acting) GH.
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
- 2 Years 至 15 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
结局指标
主要结局
Assessing new biomarkers for the diagnosis of GHD
时间窗: 3 years
The 1st objective will consist in the enrolment of 200 subjects, nationwide, undergoing standard routine work-up for the diagnosis of GHD. The specific miRNAs will be measured at the time of both stimulation tests in order to include subjects with both confirmed and unconfirmed GHD and guarantee reproducibility; in addition, subjects with organic GH deficiency will be included.
Measuring miRNA changes after 3 months on GH therapy
时间窗: 3 years
The 2nd objective will consist in measuring these specific miRNAs before and at 3 months on treatment in 200 subjects with isolated idiopathic GHD(IIGHD), and in 60 short statured patients born SGA, in 60 having Noonan and Turner syndromes, and in 30 with SHOX deficiency, according to current indications to verify whether changes are confirmed in pubertal versus prepubertal subjects and in conditions other than IIGHD.
Multiple linear regression models
时间窗: 3 years
The 3rd objective will consist in investigating the major determinants of height variations between 0 and 6 months, 0 and 12 months and the variance of growth rate variation between 0 and 6 months including the levels and/or changes in the specific miRNAs at baseline and at 3 months on treatment by using multiple linear regression models to establish early prediction models of growth response..
Measuring miRNA changes between daily and long-acting GH therapy
时间窗: 3 years
The 4th objective will consist in measuring these specific miRNAs before and at 3 months on treatment in children with IIGHD being treated with daily versus longacting GH. Findings will be compared with those in the IIGHD group receiving daily GH treatment as an exploratory task, thus, only a small number of subjects will be enrolled, and anyway as many as possible.
次要结局
- Sex-specific miRNA changes after 3 months on long-acting GH therapy(3 years)