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临床试验/NCT05070234
NCT05070234Unknown不适用

Genetic Diagnosis and the Response to Recombinant Human Growth Hormone Treatment in Small for Gestational Age Children With Short Stature

Chunxiu Gong0 个研究点目标入组 150 人开始时间: 2021年10月11日最近更新:
适应症

试验速览

阶段
不适用
发起方
入组人数
150
主要终点
Genetic testing

研究概览

简要总结

This study is a multi-center, retrospective and non-interventional research. In this study, a total of 150 short children who were small for gestational age and had been treated with recombinant human growth hormone (rhGH) are selected for genetic testing. The aims of this study are to analyze the genetic etiology of SGA children with short stature, and to compare the efficacy and safety of rhGH treatment in subjects with different etiologies.

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
2 Years 至 20 Years(Child, Adult)
性别
All
接受健康志愿者

入选标准

  • Single birth, clinical diagnosis of SGA;
  • rhGH treatment beginned before puberty (Tanner stage I), regardless of gender;
  • Before starting rhGH treatment, height was lower than -2 SDS compared with normal children of the same age and sex;
  • All the subjects and their guardians signed the informed consent and the informed consent for genetic testing.

排除标准

  • No efficacy and safety data were recorded after treatment with rhGH;
  • A history of blood transfusion within 3 months before the collection of the genetic blood samples, or a history of bone marrow transplantation between rhGH treatment and the enrollment in this study;
  • Other conditions that the investigator considered unsuitable for inclusion in this study.

结局指标

主要结局

Genetic testing

时间窗: At baseline

To detect the disease-causing genes of SGA children with short stature

次要结局

未报告次要终点

研究者

发起方
Chunxiu Gong
申办方类型
Other
责任方
Sponsor Investigator
主要研究者

Chunxiu Gong

Department of Endocrinology, Genetics and Metabolism

Beijing Children's Hospital

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