دورية أكاديمية
Somapacitan in children born small for gestational age:a multi-centre, open-label, controlled phase 2 study
| العنوان: | Somapacitan in children born small for gestational age:a multi-centre, open-label, controlled phase 2 study |
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| المؤلفون: | Juul, Anders, Backeljauw, Philippe, Højby, Michael, Kawai, Masanobu, Kildemoes, Rasmus Juul, Linglart, Agnès, Zuckerman-Levin, Nehama, Horikawa, Reiko |
| المصدر: | Juul , A , Backeljauw , P , Højby , M , Kawai , M , Kildemoes , R J , Linglart , A , Zuckerman-Levin , N & Horikawa , R 2023 , ' Somapacitan in children born small for gestational age : a multi-centre, open-label, controlled phase 2 study ' , European Journal of Endocrinology , vol. 188 , no. 1 . https://doi.org/10.1093/ejendo/lvac008Test |
| سنة النشر: | 2023 |
| المجموعة: | University of Copenhagen: Research / Forskning ved Københavns Universitet |
| مصطلحات موضوعية: | growth hormone, long-acting growth hormone, small for gestational age, somapacitan |
| الوصف: | OBJECTIVE: Investigate efficacy, safety, and tolerability of 3 once-weekly somapacitan doses compared with daily growth hormone (GH) administration in short children born small for gestational age (SGA). DESIGN: Randomised, multi-centre, open-label, controlled phase 2 study comprising a 26-week main phase and a 4-year extension (NCT03878446). The study was conducted at 38 sites across 12 countries. 26-week main phase results are presented here.Sixty-two GH treatment-naïve, prepubertal short children born SGA were randomised and exposed; 61 completed the main phase. Three somapacitan doses (0.16 [n = 12], 0.20 [n = 13], 0.24 [n = 12] mg/kg/week) and 2 daily GH doses (0.035 [n = 12], 0.067 [n = 13] mg/kg/day) were administered subcutaneously. RESULTS: After 26 weeks of treatment, the estimated mean annualised height velocity (HV) was 8.9, 11.0, and 11.3 cm/year for somapacitan 0.16, 0.20, and 0.24 mg/kg/week, respectively, compared to 10.3 and 11.9 cm/year for daily GH 0.035 and 0.067 mg/kg/day. Changes from baseline in HV standard deviation score (SDS), height SDS, and insulin-like growth factor I (IGF-I) SDS showed similar dose-dependent responses. Exposure-response modelling indicated the greatest efficacy correlated with the highest somapacitan exposure. Similar safety and tolerability were demonstrated for all weekly somapacitan and daily GH doses. CONCLUSIONS: Based on the totality of data on improvements in height-based parameters combined with exposure-response analyses, somapacitan 0.24 mg/kg/week appears most efficacious, providing similar efficacy, safety, and tolerability as daily GH 0.067 mg/kg/day in short children born SGA after 26 weeks of treatment. |
| نوع الوثيقة: | article in journal/newspaper |
| وصف الملف: | application/pdf |
| اللغة: | English |
| DOI: | 10.1093/ejendo/lvac008 |
| الإتاحة: | https://doi.org/10.1093/ejendo/lvac008Test https://curis.ku.dk/portal/da/publications/somapacitan-in-children-born-small-for-gestational-ageTest(46cc067f-ccef-406e-b1a8-b0b6105014af).html https://curis.ku.dk/ws/files/335296287/lvac008.pdfTest |
| حقوق: | info:eu-repo/semantics/openAccess |
| رقم الانضمام: | edsbas.146ADE49 |
| قاعدة البيانات: | BASE |
| DOI: | 10.1093/ejendo/lvac008 |
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