Introduction Somatrogon is a long-acting recombinant human growth hormone being developed as a once weekly treatment for children with growth hormone deficiency (GHD). The objective of this Phase 3 study (NCT03874013) was to compare the efficacy and safety of once-weekly somatrogon with once-daily Genotropin in Japanese children with GHD. Methods In this open-label, randomized, active-controlled study, 44 prepubertal Japanese children with GHD (boys: 3 to <11 years; girls: 3 to <10 years) were randomized 1:1 to receive once weekly somatrogon or once daily Genotropin (0.025 mg/kg/day) for 12 months. Dose escalation for somatrogon-treated subjects occurred in the first 6 weeks (0.25, 0.48, and 0.66 mg/kg/week; 2 weeks each) with the remaining 46 weeks at a dose of 0.66 mg/kg/week. The study’s primary endpoint was annualized height velocity (HV) at 12 months. Results Baseline characteristics were similar between treatment groups. Compared with Genotropin-treated subjects, somatrogon-treated subjects had higher least-squares (LS) mean HV at 12 months (9.65 cm/year vs 7.87 cm/year). Once weekly somatrogon was concluded as being comparable to once daily Genotropin as the mean treatment difference (somatrogon-Genotropin) in HV was +1.79 cm/year (95% CI, 0.97–2.60), which was greater than the pre-established margin (–1.8 cm/year). For both treatment groups, most adverse events were mild to moderate in severity and a similar proportion of subjects reported injection-site pain, although the somatrogon group reported more painful injections. Conclusion In prepubertal Japanese children with GHD, once-weekly somatrogon was comparable to once-daily Genotropin in terms of annualized (12-month) HV. Both treatments had similar safety and tolerability profiles. ClinicalTrials.gov:NCT03874013


中文翻译：

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每周一次 Somatrogon 与每日一次 Somatropin (Genotropin®) 在日本儿童生长激素缺乏症儿童中的疗效和安全性：随机 3 期研究的结果

简介 Somatrogon 是一种长效重组人类生长激素，被开发为每周一次的治疗生长激素缺乏症 (GHD) 的儿童。这项 3 期研究 (NCT03874013) 的目的是比较每周一次的 somatrogon 和每天一次的 Genotropin 在日本患有 GHD 的儿童中的疗效和安全性。方法 在这项开放标签、随机、主动对照研究中，44 名患有 GHD 的青春期前日本儿童（男孩：3 至 <11 岁；女孩：3 至 <10 岁）以 1:1 的比例随机接受每周一次的 somatrogon 或每天一次的治疗。 Genotropin (0.025 mg/kg/day) 12 个月。在前 6 周（0.25、0.48 和 0.66 mg/kg/周；各 2 周），somatrogon 治疗受试者的剂量增加，其余 46 周剂量为 0.66 mg/kg/周。该研究的主要终点是 12 个月时的年化身高增长速度 (HV)。结果 治疗组之间的基线特征相似。与 Genotropin 治疗的受试者相比，somatrogon 治疗的受试者在 12 个月时具有更高的最小二乘 (LS) 平均 HV（9.65 厘米/年对 7.87 厘米/年）。每周一次的 somatrogon 与每天一次的 Genotropin 相当，因为 HV 的平均治疗差异（somatrogon-Genotropin）为 +1.79 厘米/年（95% CI，0.97-2.60），大于预先确定的界限（ –1.8 厘米/年）。对于两个治疗组，大多数不良事件的严重程度为轻度至中度，相似比例的受试者报告了注射部位疼痛，尽管 somatrogon 组报告了更痛苦的注射。结论 在患有 GHD 的青春期前日本儿童中，就年化（12 个月）HV 而言，每周一次的 somatrogon 与每天一次的 Genotropin 相当。两种治疗具有相似的安全性和耐受性。ClinicalTrials.gov：NCT03874013