{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,9]],"date-time":"2026-09-09T21:45:35Z","timestamp":1788990335579,"version":"build-2803163510"},"reference-count":39,"publisher":"Oxford University Press (OUP)","issue":"1","license":[{"start":{"date-parts":[[2023,1,10]],"date-time":"2023-01-10T00:00:00Z","timestamp":1673308800000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100004191","name":"Novo Nordisk","doi-asserted-by":"crossref","id":[{"id":"10.13039\/501100004191","id-type":"DOI","asserted-by":"crossref"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2023,1,10]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Objective<\/jats:title>\n                    <jats:p>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).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Design<\/jats:title>\n                    <jats:p>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.<\/jats:p>\n                    <jats:p>Sixty-two GH treatment-na\u00efve, 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.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>After 26 weeks of treatment, the estimated mean annualised height velocity (HV) was 8.9, 11.0, and 11.3\u2005cm\/year for somapacitan 0.16, 0.20, and 0.24\u2005mg\/kg\/week, respectively, compared to 10.3 and 11.9\u2005cm\/year for daily GH 0.035 and 0.067\u2005mg\/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.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>Based on the totality of data on improvements in height-based parameters combined with exposure-response analyses, somapacitan 0.24\u2005mg\/kg\/week appears most efficacious, providing similar efficacy, safety, and tolerability as daily GH 0.067\u2005mg\/kg\/day in short children born SGA after 26 weeks of treatment.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1093\/ejendo\/lvac008","type":"journal-article","created":{"date-parts":[[2023,1,3]],"date-time":"2023-01-03T16:59:54Z","timestamp":1672765194000},"page":"19-30","source":"Crossref","is-referenced-by-count":22,"title":["Somapacitan in children born small for gestational age: a multi-centre, open-label, controlled phase 2 study"],"prefix":"10.1093","volume":"188","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-0534-4350","authenticated-orcid":false,"given":"Anders","family":"Juul","sequence":"first","affiliation":[{"name":"Department of Growth and Reproduction, Copenhagen University Hospital - Rigshospitalet , Copenhagen 2100 , Denmark"},{"name":"Department of Clinical Medicine, University of Copenhagen , Copenhagen 2200 , Denmark"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Philippe","family":"Backeljauw","sequence":"additional","affiliation":[{"name":"Division of Endocrinology, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine , Cincinnati, OH 45229 , United States"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael","family":"H\u00f8jby","sequence":"additional","affiliation":[{"name":"Clinical Drug Development, Novo Nordisk A\/S , S\u00f8borg 2860 , Denmark"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4466-1559","authenticated-orcid":false,"given":"Masanobu","family":"Kawai","sequence":"additional","affiliation":[{"name":"Department of Gastroenterology, Nutrition and Endocrinology, Research Institute, Osaka Women's and Children's Hospital , Izumi, Osaka 594-1101 , Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rasmus Juul","family":"Kildemoes","sequence":"additional","affiliation":[{"name":"Clinical Drug Development, Novo Nordisk A\/S , S\u00f8borg 2860 , Denmark"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3455-002X","authenticated-orcid":false,"given":"Agn\u00e8s","family":"Linglart","sequence":"additional","affiliation":[{"name":"AP-HP, Universit\u00e9 Paris Saclay, INSERM, Service d'Endocrinologie et Diab\u00e8te de l'Enfant, H\u00f4pital Bic\u00eatre Paris Saclay , Le Kremlin-Bic\u00eatre 94270 , France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nehama","family":"Zuckerman-Levin","sequence":"additional","affiliation":[{"name":"Institute of Diabetes, Endocrinology and Metabolism, Rambam Health Care Center, Bruce Rappaport Faculty of Medicine , Technion, Haifa 31096 , Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3429-3246","authenticated-orcid":false,"given":"Reiko","family":"Horikawa","sequence":"additional","affiliation":[{"name":"Division of Endocrinology and Metabolism, National Center for Child Health and Development , Tokyo 157-8535 , Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"286","published-online":{"date-parts":[[2023,1,10]]},"reference":[{"issue":"2","key":"2023072118421039000_lvac008-B1","doi-asserted-by":"crossref","first-page":"283","DOI":"10.1016\/j.ecl.2016.01.008","article-title":"Growth and metabolism in children born small for gestational 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