Somapacitan in Children Born SGA: 52-week Efficacy, Safety, and IGF-I Response Results from the Phase 2 REAL5 Study.

Autor:	Juul A; Department of Growth and Reproduction, Copenhagen University Hospital - Rigshospitalet, Copenhagen 2100, Denmark.; Department of Clinical Medicine, University of Copenhagen, Copenhagen, Denmark., Backeljauw P; Division of Endocrinology, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, Ohio 45229, USA., Højby M; Novo Nordisk A/S, Søborg 2860, Denmark., Frystyk J; Department of Clinical Research, University of Southern Denmark, Odense 5230, Denmark.; Department of Endocrinology, Odense University Hospital, Odense, Denmark., Kawai M; Department of Gastroenterology, Nutrition and Endocrinology, Osaka Women's and Children's Hospital, Izumi, Osaka 594-1101, Japan., Kildemoes RJ; Novo Nordisk A/S, Søborg 2860, Denmark., Lemminger AK; Novo Nordisk A/S, Søborg 2860, Denmark., Linglart A; AP-HP, Université Paris Saclay, INSERM, Service d'Endocrinologie et Diabète de l'Enfant, Hôpital Bicêtre Paris Saclay, Le Kremlin-Bicêtre 94270, France., Zuckerman-Levin N; Institute of Diabetes, Endocrinology and Metabolism, Rambam Health Care Center, Bruce Rappaport Faculty of Medicine, Technion, Haifa 31096, Israel., Horikawa R; Division of Endocrinology and Metabolism, National Center for Child Health and Development, Tokyo 157-8535.
Jazyk:	angličtina
Zdroj:	The Journal of clinical endocrinology and metabolism [J Clin Endocrinol Metab] 2024 Sep 13. Date of Electronic Publication: 2024 Sep 13.
DOI:	10.1210/clinem/dgae616
Abstrakt:	Context: Somapacitan, a once-weekly reversible albumin-binding GH derivative, is evaluated in short children born small for gestational age (SGA). Objective: Evaluate efficacy, safety, tolerability as well as total and bioactive insulin-like growth factor-I (IGF-I) response of once-weekly somapacitan compared to daily GH in children born SGA. Methods: REAL5 is a randomized, multi-center, open-label, controlled phase 2 study comprising a 26-week main phase, 26-week extension, and an ongoing 4-year safety extension (NCT03878446). Setting: Thirty-eight sites across 12 countries. Patients: Sixty-two GH-treatment-naïve, prepubertal short children born SGA were randomized; 61 completed 52-weeks of treatment. Interventions: Patients randomized (1:1:1:1:1) to somapacitan (0.16, 0.20 or 0.24 mg/kg/week) or daily GH (0.035 or 0.067 mg/kg/day), all administered subcutaneously. Results: Estimated mean height velocity (HV; cm/year) at week 52 was 8.5, 10.4 and 10.7 cm/year for somapacitan 0.16, 0.20 and 0.24 mg/kg/week, respectively, and 9.3 and 11.2 cm/year for daily GH 0.035 and 0.067 mg/kg/day, respectively. Dose-dependent increases in total IGF-I as well as peak IGF-I bioactivity were observed for both treatments and were similar between comparator groups. For somapacitan, exposure-response modelling indicated highest efficacy with 0.24 mg/kg/week after 52 weeks of treatment. Similar safety and tolerability were demonstrated across all groups. Conclusions: A sustained dose-dependent growth response was demonstrated for somapacitan after 52 weeks of treatment. Overall, somapacitan 0.24 mg/kg/week provides similar efficacy, safety, and tolerability, as well as comparable bioactive and total IGF-I response, as daily GH (0.067 mg/kg/day) in children born SGA. (© The Author(s) 2024. Published by Oxford University Press on behalf of the Endocrine Society.)
Databáze:	MEDLINE
Externí odkaz:	Zobrazit plný text záznamu