Effective growth hormone replacement with once‐weekly somapacitan in Japanese children with growth hormone deficiency: Results from REAL4, a phase 3 clinical trial

Author:

Mori Jun1,Ohata Yasuhisa2,Fujisawa Yasuko3,Sato Yukihito4,Röhrich Sebastian5,Rasmussen Michael Højby6ORCID,Bang Rikke Beck7,Horikawa Reiko8

Affiliation:

1. Division of Pediatric Endocrinology and Metabolism Children's Medical Center, Osaka City General Hospital Osaka Japan

2. Department of Pediatrics Osaka University Graduate School of Medicine Suita Japan

3. Department of Pediatrics Hamamatsu University School of Medicine Hamamatsu Japan

4. Rare Disease Group Novo Nordisk Pharma Ltd. Tokyo Japan

5. Global Medical Affairs Novo Nordisk Health Care AG Zürich Switzerland

6. Medical and Science, Rare Disease and Advanced Therapies Clinical Drug Development, Novo Nordisk A/S Søborg Denmark

7. Biostatistics, Rare Disease and Advanced Therapies Data Science, Novo Nordisk A/S Aalborg Denmark

8. Division of Endocrinology and Metabolism National Center for Child Health and Development Tokyo Japan

Abstract

AbstractObjectiveSomapacitan is a long‐acting growth hormone (GH) derivative developed for the treatment of GH deficiency (GHD). This study evaluates the efficacy and tolerability of somapacitan in Japanese children with GHD after 104 weeks of treatment and after switch from daily GH.DesignSubanalysis on Japanese patients from a randomised, open‐labelled, controlled parallel‐group phase 3 trial (REAL4, NCT03811535).Patients and MeasurementsThirty treatment‐naïve patients were randomised 2:1 to somapacitan (0.16 mg/kg/week) or daily GH (0.034 mg/kg/day) up to Week 52, after which all patients received somapacitan. Height velocity (HV; cm/year) at Weeks 52 and 104 were the primary measurements. Additional assessments included HV SD score (SDS), height SDS, bone age, insulin‐like growth factor‐I (IGF‐I) SDS, and observer‐reported outcomes.ResultsAt Week 52, observed mean HV was similar between treatment groups (10.3 vs. 9.8 cm/year for somapacitan and daily GH, respectively). Similar HVs between groups were also observed at Week 104: 7.4 cm/year after continuous somapacitan treatment (soma/soma) and 7.9 cm/year after 1‐year somapacitan treatment following switch from daily GH (switch). Other height‐related endpoints supported continuous growth. IGF‐I SDS increased in both groups with mean IGF‐I SDS within −2 and +2 during the study. Somapacitan was well tolerated, one mild injection site reaction was reported, with no reports of injection site pain. Patient preference questionnaires showed that most patients and their caregivers (90.9%) who switched treatment at Week 52 preferred once‐weekly somapacitan over daily GH treatment.ConclusionsSomapacitan showed sustained efficacy in Japanese children with GHD over 104 weeks and for 52 weeks after switching from daily GH. Somapacitan was well tolerated and preferred over daily GH.

Funder

Novo Nordisk

Publisher

Wiley

Cited by 3 articles. 订阅此论文施引文献 订阅此论文施引文献,注册后可以免费订阅5篇论文的施引文献,订阅后可以查看论文全部施引文献

同舟云学术

1.学者识别学者识别

2.学术分析学术分析

3.人才评估人才评估

"同舟云学术"是以全球学者为主线,采集、加工和组织学术论文而形成的新型学术文献查询和分析系统,可以对全球学者进行文献检索和人才价值评估。用户可以通过关注某些学科领域的顶尖人物而持续追踪该领域的学科进展和研究前沿。经过近期的数据扩容,当前同舟云学术共收录了国内外主流学术期刊6万余种,收集的期刊论文及会议论文总量共计约1.5亿篇,并以每天添加12000余篇中外论文的速度递增。我们也可以为用户提供个性化、定制化的学者数据。欢迎来电咨询!咨询电话:010-8811{复制后删除}0370

www.globalauthorid.com

TOP

Copyright © 2019-2024 北京同舟云网络信息技术有限公司
京公网安备11010802033243号  京ICP备18003416号-3