A Real-world Study of the Efficacy and Safety of PEGylated Recombinant Human Growth Hormone Injection in the Treatment of Short Stature in Chinese Children From the Global Registry for Novel Therapies For Rare Bone or Endocrine Conditions
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 2,600
- 试验地点
- 1
- 主要终点
- the change in height standard deviation score
研究概览
简要总结
In order to further observe the long-term safety and effectiveness of real-world polyethylene glycol-recombinant human growth hormone(PEG-rhGH) treatment of GHD, idiopathic short stature, and SGA in children, explore and analyze the factors affecting the efficacy of PEG-rhGH and the height prediction model after treatment, etc., collect and analyze more scientifically and rationally, and understand the situation of real-world PEG-GH treatment. A database registration study was developed.
详细描述
A database registration study was developed to retrospectively analyze the efficacy and safety of polyethylene glycol recombinant human growth hormone injection in the treatment of slow growth in children caused by endogenous growth hormone deficiency, idiopathic short stature, Turner syndrome, etc., and to further prospectively follow up and monitor. In order to participate in the global real-world use of long-acting growth hormone, all data from this study were uploaded to the Global Registry of New Therapies for Rare Endocrine Diseases and Bone Disorders (GloBE-Reg) platform.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Retrospective
入排标准
- 年龄范围
- — 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients with endogenous growth hormone deficiency, Turner Syndrome, SGA, idiopathic short stature, etc. treated with polyethylene glycol recombinant human growth hormone injection
排除标准
- •Patients with serious heart and lung, blood system, malignant tumors and other diseases or systemic infections, immune function is low and Persons with mental illness;
研究组 & 干预措施
growth hormone deficiency(GHD)
Growth hormone deficiency (GHD) is a rare and treatable condition that causes short height in children and metabolic issues in adults.
干预措施: Polyethylene glycol recombinant human growth hormone injection (Drug)
Idiopathic short stature (ISS)
Idiopathic short stature is a condition in which the height of the individual is more than 2 SD below the corresponding mean height for a given age, sex and population, in whom no identifiable disorder is present.
干预措施: Polyethylene glycol recombinant human growth hormone injection (Drug)
small for gestational age (SGA)
Small for gestational age (SGA) is defined as a birth weight of less than 10th percentile for gestational age.
干预措施: Polyethylene glycol recombinant human growth hormone injection (Drug)
Turner Syndrome (TS)
Turner syndrome, a condition that affects only females, results when one of the X chromosomes (sex chromosomes) is missing or partially missing.
干预措施: Polyethylene glycol recombinant human growth hormone injection (Drug)
others
others who with the height of the individual is more than 2 SD below the corresponding mean height for a given age, sex and population.
干预措施: Polyethylene glycol recombinant human growth hormone injection (Drug)
结局指标
主要结局
the change in height standard deviation score
时间窗: 3-10years
final adult height standard deviation score- height standard deviation score before treatment
次要结局
未报告次要终点
研究者
Chunxiu Gong
Principal Investigator
Beijing Children's Hospital