Miller Bradley S, Blair Joanne C, Rasmussen Michael Højby, Frystyk Jan, Lemminger Anders Krogh, Maniatis Aristides, Mori Jun, Böttcher Volker, Kim Ho-Seong, Polak Michel, Horikawa Reiko
Division of Endocrinology, Department of Pediatrics, University of Minnesota Medical School, M Health Fairview Masonic Children's Hospital, Minneapolis, MN 55454, United States.
Department of Endocrinology, Alder Hey Children's NHS Foundation Trust, Liverpool L14 5AB, United Kingdom.
Eur J Endocrinol. 2025 Apr 30;192(5):651-661. doi: 10.1093/ejendo/lvaf096.
Somapacitan is a long-acting GH approved for once-weekly treatment of GH deficiency (GHD). This study aims to evaluate the efficacy and tolerability of somapacitan after 3 years of treatment and 2 years after switch from daily GH in children with GHD.
Randomized, multi-national, open-labelled, active-controlled parallel-group phase 3 trial, with a 52-week main phase and 3-year safety extension (NCT03811535).
Treatment-naïve children with GHD were randomized (2:1) to continuous somapacitan (0.16 mg/kg/week; "soma/soma" group) or daily GH (Norditropin®; 0.034 mg/kg/day) followed by somapacitan (0.16 mg/kg/week; "switch" group).
Of 200 participants, 188 completed 3 years of treatment. Sustained growth was observed in both groups. At week 156, mean (SD) height velocity (HV) between weeks 104 and 156 was 7.4 (1.5) cm/year in the soma/soma group and 7.8 (1.4) cm/year in the switch group. At week 156, the soma/soma and switch groups had reached a mean (SD) height SD score (HSDS) of -0.95 (0.98) and -1.08 (0.93), respectively, and were approaching the mean mid-parental HSDS of -0.74 (for both groups). Mean total insulin-like growth factor I (IGF-I) SDS during year 3 was similar between groups and within normal range (-2.0 to +2.0). Bioactive IGF-I and bioactive IGF-I to IGF-I ratio were similar between groups. Somapacitan was well tolerated, with low proportions reporting injection-site reactions.
Sustained efficacy and tolerability were observed for continuous somapacitan treatment for 3 years, and for 2 years after the switching from daily GH treatment. HSDS in both groups was approaching mean mid-parental HSDS.
NCT03811535.
索马帕西坦是一种长效生长激素（GH），已获批用于每周一次治疗生长激素缺乏症（GHD）。本研究旨在评估索马帕西坦治疗3年以及从每日注射生长激素转换治疗2年后在GHD儿童中的疗效和耐受性。
随机、多国、开放标签、活性对照平行组3期试验，主要阶段为52周，安全性扩展期为3年（NCT03811535）。
初治的GHD儿童被随机分组（2:1），分别接受持续的索马帕西坦治疗（0.16 mg/kg/周；“索马/索马”组）或每日生长激素治疗（诺德人体生长激素®；0.034 mg/kg/天），之后改为索马帕西坦治疗（0.16 mg/kg/周；“转换”组）。
200名参与者中，188名完成了3年治疗。两组均观察到持续生长。在第156周时，“索马/索马”组在第104周至156周期间的平均（标准差）身高增长速度（HV）为7.4（1.5）厘米/年，“转换”组为7.8（1.4）厘米/年。在第156周时，“索马/索马”组和“转换”组的平均（标准差）身高标准差评分（HSDS）分别达到-0.95（0.98）和-1.08（0.93），均接近父母平均HSDS的-0.74（两组均如此）。第3年期间两组的平均总胰岛素样生长因子I（IGF-I）标准差评分相似且在正常范围内（-2.0至+2.0）。两组间生物活性IGF-I以及生物活性IGF-I与IGF-I的比值相似。索马帕西坦耐受性良好，报告注射部位反应的比例较低。
持续使用索马帕西坦治疗3年以及从每日生长激素治疗转换后使用2年，均观察到持续的疗效和耐受性。两组的HSDS均接近父母平均HSDS。
NCT03811535。
