{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,23]],"date-time":"2026-09-23T04:33:10Z","timestamp":1790137990395,"version":"4.0.1"},"reference-count":42,"publisher":"Oxford University Press (OUP)","issue":"3","license":[{"start":{"date-parts":[[2026,3,3]],"date-time":"2026-03-03T00:00:00Z","timestamp":1772496000000},"content-version":"vor","delay-in-days":2,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100004191","name":"Novo Nordisk","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100004191","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2026,3,4]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Objective<\/jats:title>\n                    <jats:p>Daily growth hormone (GH) injections are indicated for the treatment of short stature in children with Noonan syndrome, which presents a treatment burden for the child and their parents\/caregivers. Somapacitan is a long-acting, reversible albumin-binding GH, developed for once-weekly administration. The objective of this study is to evaluate efficacy, safety, and tolerability of somapacitan versus daily GH in children living with Noonan syndrome.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Design<\/jats:title>\n                    <jats:p>REAL8 (NCT05330325) is a multinational, multicenter, randomized, open-labeled, active comparator, phase 3 basket study including four non-GH deficiency indications comprising a 52-week main phase and 104-week extension. Here, we present 52-week results from the REAL8 Noonan syndrome substudy.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>Seventy-seven GH-treatment-na\u00efve, prepubertal boys (aged 2.5\u201311 years) and girls (aged 2.5\u201310 years) with Noonan syndrome were randomized 2:1 to somapacitan 0.24\u2005mg\/kg\/week or daily GH 0.050\u2005mg\/kg\/day, administered subcutaneously.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>The primary endpoint, estimated mean annualized height velocity at week 52, was 10.4\u2005cm\/year for somapacitan versus 9.2\u2005cm\/year for daily GH (estimated treatment difference [ETD]: 1.2, 95% CI [0.32; 2.03]), confirming noninferiority and demonstrating superiority of somapacitan compared to daily GH (P &amp;lt; .01). The estimated change from baseline to week 52 in height standard deviation score was 1.07 and 0.75 for somapacitan and daily GH, respectively (ETD: 0.32, 95% CI [0.16; 0.48]). Somapacitan was well tolerated and had a similar safety profile to daily GH.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>Once-weekly somapacitan was confirmed as noninferior and demonstrated superiority to daily GH in HV after 52 weeks of treatment in treatment-na\u00efve children living with Noonan syndrome. Similar safety profiles and tolerability were observed for both groups.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Clinical Trial Registration<\/jats:title>\n                    <jats:p>NCT05330325.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1093\/ejendo\/lvag041","type":"journal-article","created":{"date-parts":[[2026,2,27]],"date-time":"2026-02-27T12:39:18Z","timestamp":1772195958000},"page":"393-403","source":"Crossref","is-referenced-by-count":3,"title":["Once-weekly somapacitan in children with Noonan syndrome: randomized controlled phase 3 trial"],"prefix":"10.1093","volume":"194","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-2567-7360","authenticated-orcid":false,"given":"Alexander A L","family":"Jorge","sequence":"first","affiliation":[{"name":"Genetic Endocrinology Unit (LIM25), Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo (FMUSP) , Sao Paulo 05403-010 ,","place":["Brazil"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4051-6661","authenticated-orcid":false,"given":"Assunta","family":"Albanese","sequence":"additional","affiliation":[{"name":"Endocrine Paediatric Unit, St George's University Hospitals, NHS Foundation Trust , London SW17 0QT ,","place":["United Kingdom"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2210-0366","authenticated-orcid":false,"given":"Michael","family":"H\u00f8jby","sequence":"additional","affiliation":[{"name":"Novo Nordisk A\/S , S\u00f8borg 2860 ,","place":["Denmark"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kamil","family":"Soltysik","sequence":"additional","affiliation":[{"name":"Novo Nordisk A\/S , S\u00f8borg 2860 ,","place":["Denmark"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9222-5009","authenticated-orcid":false,"given":"Thomas","family":"Edouard","sequence":"additional","affiliation":[{"name":"Endocrine, Bone Diseases, and Genetics Unit, Reference Center for Endocrine Diseases of Growth and Development, FIRENDO Network, Children's Hospital, Toulouse University Hospital, INSERM UMR 1301 RESTORE Institute , Toulouse 31300 ,","place":["France"]},{"name":"Endocrine, Bone Diseases and Genetics Unit, Reference Center for Rare Growth Disorders, CRESCENDO Network, Endo-ERN, Paediatric Research Unit CIC 1436 , Children\u2019s Hospital, Toulouse University Hospital, Toulouse 31300 ,","place":["France"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anna","family":"Grandone","sequence":"additional","affiliation":[{"name":"Department of Woman, Child and General and Specialized Surgery, University of Campania \u201cLuigi 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