Efficacy and Safety of Weekly Somatrogon vs Daily Somatropin in Children With Growth Hormone Deficiency: A Phase 3 Study.

IF 2.2 4区生物学 Q2 PLANT SCIENCES

Journal of Plant Biology Pub Date : 2022-06-16 DOI:10.1210/clinem/dgac220

Cheri L Deal, Joel Steelman, Elpis Vlachopapadopoulou, Renata Stawerska, Lawrence A Silverman, Moshe Phillip, Ho-Seong Kim, CheolWoo Ko, Oleg Malievskiy, Jose F Cara, Carl L Roland, Carrie Turich Taylor, Srinivas Rao Valluri, Michael P Wajnrajch, Aleksandra Pastrak, Bradley S Miller

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引用次数: 0

Abstract

Context: Somatrogon is a long-acting recombinant human growth hormone (rhGH) in development for once-weekly treatment of children with growth hormone deficiency (GHD).

Objective: We aimed to compare the efficacy and safety of once-weekly somatrogon with once-daily somatropin in prepubertal children with GHD.

Methods: In this 12-month, open-label, randomized, active-controlled, parallel-group, phase 3 study, participants were randomized 1:1 to receive once-weekly somatrogon (0.66 mg/kg/week) or once-daily somatropin (0.24 mg/kg/week) for 12 months. A total of 228 prepubertal children (boys aged 3-11 years, girls aged 3-10 years) with GHD, impaired height and height velocity (HV), and no prior rhGH treatment were randomized and 224 received ≥1 dose of study treatment (somatrogon: 109; somatropin: 115). The primary endpoint was annualized HV at month 12.

Results: HV at month 12 was 10.10 cm/year for somatrogon-treated subjects and 9.78 cm/year for somatropin-treated subjects, with a treatment difference (somatrogon-somatropin) of 0.33 (95% CI: -0.24, 0.89). The lower bound of the 2-sided 95% CI was higher than the prespecified noninferiority margin (-1.8 cm/year), demonstrating noninferiority of once-weekly somatrogon vs daily somatropin. HV at month 6 and change in height standard deviation score at months 6 and 12 were similar between both treatment groups. Both treatments were well tolerated, with a similar percentage of subjects experiencing mild to moderate treatment-emergent adverse events in both groups (somatrogon: 78.9%, somatropin: 79.1%).

Conclusion: The efficacy of once-weekly somatrogon was noninferior to once-daily somatropin, with similar safety and tolerability profiles. (ClinicalTrials.gov no. NCT02968004).

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生长激素缺乏症儿童每周使用索马特罗贡与每天使用索马特罗宾的疗效和安全性对比：3期研究

背景：Somatrogon是一种正在开发的长效重组人生长激素（rhGH），用于生长激素缺乏症（GHD）儿童的每周一次治疗：我们的目的是比较每周一次的索玛曲贡（somatrogon）和每天一次的索玛托品（somatropin）对青春期前生长激素缺乏症儿童的疗效和安全性：在这项为期12个月的开放标签、随机、主动对照、平行组的3期研究中，参与者按1:1的比例随机接受每周一次的索马曲贡（0.66毫克/公斤/周）或每日一次的索马托品（0.24毫克/公斤/周），为期12个月。共有228名青春期前儿童（男孩3-11岁，女孩3-10岁）患有GHD，身高和身高速度（HV）受损，且之前未接受过rhGH治疗，他们被随机分配，其中224人接受了≥一个剂量的研究治疗（索马特罗贡：109人；索马特罗肽：115人）。主要终点是第12个月时的年化HV：第 12 个月时，经索马曲贡治疗的受试者的 HV 为 10.10 厘米/年，经索马托品治疗的受试者的 HV 为 9.78 厘米/年，治疗差异（索马曲贡-索马托品）为 0.33（95% CI：-0.24，0.89）。双侧 95% CI 的下限高于预设的非劣效性边际（-1.8 厘米/年），表明每周一次索马特罗贡与每天一次索马特罗滨相比具有非劣效性。两个治疗组在第 6 个月时的身高体重以及第 6 个月和第 12 个月时的身高标准偏差评分变化相似。两种疗法的耐受性都很好，两组中出现轻度至中度治疗突发不良事件的受试者比例相似（索马曲康：78.9%；索马托品：79.1%）：结论：每周一次的索马曲贡疗效不劣于每日一次的索马托品，安全性和耐受性相似。(结论：每周一次索马曲贡的疗效并不亚于每日一次的索马托品，其安全性和耐受性相似。）

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Journal of Plant Biology 生物-植物科学

CiteScore

5.40

自引率

3.40%

发文量

审稿时长

1 months

期刊介绍： Journal of Plant Biology, an official publication of the Botanical Society of Korea, is an international journal devoted to basic researches in biochemistry, cellular biology, development, ecology, genetics, molecular biology, physiology, and systematics of plants. The Journal publishes the following categories of paper: Original articles -- For publication in Journal of Plant Biology the manuscript must provide a significant new contribution to our understanding of plants. All areas of plant biology are welcome. No limit on the length, but a concise presentation is encouraged. Reviews -- Invited by the EiC. Brief Communications -- Concise but independent report representing significant contribution to plant science. The Botanical Society of Korea was founded on November 30, 1957 to promote studies, disseminate and exchange information on the field of plant biology. The first issue of The Korean Journal of Botany, the official publication of the society, was published on April 1, 1958. It was published twice a year, but quarterly from 5th volume in 1962. In 1994, it was renamed to Journal of Plant Biology and published in English since 1996. The journal entered its 50th year of publication in 2007.