Fu Junfen, Cheng Xinran, Højby Michael, Gong Chunxiu, Leunbach Tina Lund, Li Yanhong, Wei Haiyan, Zhu Yu, Zhang Yining, Zhong Yan
Endocrinology Department, Children's Hospital of Zhejiang University School of Medicine, National Clinical Research Center for Child Health, Hangzhou, China.
Department of Pediatric Genetics, Endocrinology and Metabolism, Chengdu Women and Children's Center Hospital, Chengdu, China.
Horm Res Paediatr. 2025 May 5:1-12. doi: 10.1159/000545786.
REAL6 is a randomized, multi-centre, open-labelled, active-controlled parallel group phase 3 trial (NCT04970654) investigating once-weekly somapacitan, a reversible albumin-binding growth hormone (GH) derivative, in Chinese children with GH deficiency (GHD).
110 treatment-naïve patients were randomized 2:1 to somapacitan (0.16 mg/kg/week) or daily GH (Norditropin®; 0.034 mg/kg/day) for 52 weeks. Annualized height velocity (HV; cm/year) at week 52 was the primary measurement. Additional assessments included HV standard deviation score (SDS), height SDS (HSDS), bone age, insulin-like growth factor-I (IGF-I) SDS, and observer-reported outcomes.
Estimated mean HV at week 52 was 11.0 and 10.4 cm/year for somapacitan and daily GH, respectively. Non-inferiority of somapacitan compared to daily GH was confirmed. Changes in HVSDS, HSDS, bone age, and IGF-I SDS from baseline to week 52 were similar between groups. At week 52, mean (SD) IGF-I SDS was within intended reference range (-2.0 to +2.0) and comparable between groups: +0.5 (1.4) for somapacitan versus +0.1 (1.2) for daily GH. Somapacitan was well tolerated with a safety profile consistent with the well-known safety profile of daily GH. A low proportion of injection site reactions were reported for somapacitan (2.7%), with no reports of injection site pain during the 52-week treatment period. Disease burden was reduced from baseline to week 52 for both treatments. Somapacitan reduced treatment burden compared to daily GH.
Efficacy and safety profiles were comparable for Chinese children with GHD treated with somapacitan or daily GH. Both treatments similarly reduced disease burden, while treatment burden was reduced with somapacitan.
REAL6是一项随机、多中心、开放标签、活性对照平行组3期试验（NCT04970654），研究每周一次的索马比星（一种可逆的白蛋白结合生长激素（GH）衍生物）用于中国生长激素缺乏症（GHD）儿童的情况。
110名未接受过治疗的患者按2:1随机分组，接受索马比星（0.16毫克/千克/周）或每日生长激素（诺德人体生长激素；0.034毫克/千克/天）治疗52周。第52周时的年化身高增长速度（HV；厘米/年）是主要测量指标。其他评估包括HV标准差评分（SDS）、身高SDS（HSDS）、骨龄、胰岛素样生长因子-I（IGF-I）SDS以及观察者报告的结果。
第52周时，索马比星组和每日生长激素组的估计平均HV分别为11.0厘米/年和10.4厘米/年。证实了索马比星相对于每日生长激素的非劣效性。两组从基线到第52周的HVSDS、HSDS、骨龄和IGF-I SDS变化相似。在第52周时，平均（标准差）IGF-I SDS在预期参考范围内（-2.0至+2.0），且两组之间具有可比性：索马比星组为+0.5（1.4），每日生长激素组为+0.1（1.2）。索马比星耐受性良好，安全性与每日生长激素的已知安全性特征一致。索马比星报告的注射部位反应比例较低（2.7%），在52周治疗期间未报告注射部位疼痛。两种治疗方法从基线到第52周疾病负担均有所减轻。与每日生长激素相比，索马比星减轻了治疗负担。
对于接受索马比星或每日生长激素治疗的中国GHD儿童，其疗效和安全性相当。两种治疗方法同样减轻了疾病负担，而索马比星减轻了治疗负担。
