A Comparative Study of the Therapeutic Effect of PEGylated Recombinant Human Growth Hormone(PEG-rhGH) Injection on Turner Syndrome in a Clinical Trial and Short-acting Recombinant Human Growth Hormone in a Real World Study
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 743
- 试验地点
- 1
- 主要终点
- Change in height standard deviation scores (HT SDS) after 1 year (52 weeks) of treatment compared to before (i.e., baseline) (ΔHT SDS).
研究概览
简要总结
This study is divided into two parts:
Part A: A multicenter, retrospective, observational study to evaluate the effectiveness of PEG-rhGH in the treatment of Turner syndrome with domestic real-world data on rhGH as external controls. The collection and arrangement of real world data is the content of the real world study (RWS); Part B: Meta analysis (MA) was conducted based on the previous literatures on rhGH in the treatment of Turner syndrome, and MA results were used as external controls to evaluate the effectiveness of PEG-rhGH.
Real-world data on rhGH treatment of Turner syndrome in China were obtained from the HIS system and/or paper medical records of participating domestic centers and/or photocopied/printed medical records of other hospitals. Data on the efficacy of PEG-rhGH injection in the treatment of Turner syndrome were obtained from the 0.2mg /kg/ week group in the Phase II clinical trial of PEG-rhGH injection in the treatment of Turner syndrome (multicenter, randomized, blank control, superior efficacy) conducted by Changchun GeneScience Pharmaceutical Co., Ltd (Protocol number: GenSci032-02, version date: April 27, 2015). The MA study data of rhGH in the treatment of Turner syndrome in foreign countries came from the relevant literature retrieved from the approved drug database of FDA, PubMed and Web of Science database.
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Retrospective
入排标准
- 年龄范围
- 0 Years 至 18 Years（Child, Adult）
- 性别
- Female
- 接受健康志愿者
- 否
入选标准
- •Patients diagnosed with Turner syndrome by a clinician;
- •The first treatment was short-acting rhGH, and the first administration date was not earlier than July 1, 2008;
- •Follow-up data are available at the center before the first dosing and after at least one dosing.
排除标准
- 未提供
结局指标
主要结局
Change in height standard deviation scores (HT SDS) after 1 year (52 weeks) of treatment compared to before (i.e., baseline) (ΔHT SDS).
时间窗: Week 52
Change in height standard deviation scores (HT SDS) after 1 year (52 weeks) of treatment compared to before (i.e., baseline) (ΔHT SDS).
次要结局
- The annualized height velocity (AHV, cm/ year) after 1 year (52 weeks) of treatment.(Week 52)