目的 评价注射用重组人生长激素（rhGH）治疗中国青春期前特发性矮小症（ISS）的有效性和安全性。方法 该研究为2016年12月27日至2020年07月30日，招募青春期前ISS患儿开展的一项随机、开放、空白对照、多中心的Ⅲ期临床试验。纳入受试者以3:3:1的比例随机分配至高剂量组、低剂量组和空白对照组，rhGH的给药周期为52周。观察治疗前后实际年龄的身高标准差积分变化（ΔHT SDS)，治疗结束时受试者的年生长速率（HV）和骨成熟情况，同时考察治疗期间药物安全性。结果 共有150例受试者完成了试验。基线时各组受试者的年龄、骨龄、性别、身高、体重、体质指数及HT-SDS、年HV、胰岛素样生长因子1水平差异均无统计学意义（P>0.05）。全数据集分析（FAS）和符合方案集（PPS）分析均发现，第26、39、52周，高剂量组、低剂量组和对照组之间ΔHT SDS的差异均有统计学意义（P<0.001）；两两比较发现，高剂量和低剂量组的ΔHT SDS均高于对照组，差异均有统计学意义（P<0.05）。FAS分析发现，第13、26、39、52周，高剂量组、低剂量组和对照组之间HV的差异均有统计学意义（P<0.001）；两两比较发现，高剂量和低剂量组的HV均高于对照组，差异均有统计学意义（P<0.05）。第52周，各组之间骨成熟差异无统计学意义（P>0.05）。研究过程中，无严重不良反应发生。结论 注射用rhGH治疗ISS疗效确切，患儿用药52周后有明显的身高增长，但对终身高的作用和安全性尚需更多的观察与评估。

Objective To evaluate the efficacy and safety of recombinant human growth hormone (rhGH) for injection in the treatment of prepubertal children with idiopathic short stature (ISS) in China. Methods This study was a randomized, open-label, controlled, multicenter, phase Ⅲ clinical trial of prepubertal ISS from December 27, 2016 to July 30, 2020. Participants were randomly assigned to the high-dose group, low-dose group and control group in a ratio of 3:3:1, and the administration cycle of rhGH was 52 weeks. Changes in height standard deviation scores (ΔHT-SDS) of actual age before and after treatment, annual height velocity (HV) and bone maturity at the end of treatment were observed. At the same time, the rhGH safety during treatment was investigated. Results A total of 150 participants completed the trial. At baseline, there were no significant differences in age, bone age, sex, height, weight, body mass index, HT-SDS, annual HV and insulin-like growth factor 1 levels among all groups (P>0.05). Both full analysis set (FAS) and per protocol set (PPS) analysis found statistically significant differences in ΔHT SDS among the high-dose, low-dose and control groups at week 26, 39 and 52 (P<0.001). Pairwise comparison showed that ΔHT SDS in the high-dose and low-dose groups were higher than those in the control group, and the differences were statistically significant (P<0.05). FAS analysis showed statistically significant differences in HV among the high-dose, low-dose and control groups at week 13, 26, 39, and 52 (P<0.001). Pairwise comparison showed that HV in the high-dose and low-dose groups was higher than that in the control group, and the differences were statistically significant (P<0.05). At week 52, there was no significant difference in bone maturity among all groups (P>0.05). During the study, no serious adverse reaction occurred in each group. Conclusions The rhGH injection is effective in treating ISS, and there is a significant growth in stature after 52 weeks of treatment, but more observation and evaluation of the effect and safety of lifetime height are needed.