Somapacitan in children born small for gestational age: a multi-centre, open-label, controlled phase 2 study

Author:

Juul Anders12ORCID,Backeljauw Philippe3,Højby Michael4,Kawai Masanobu5ORCID,Kildemoes Rasmus Juul4,Linglart Agnès6ORCID,Zuckerman-Levin Nehama7,Horikawa Reiko8ORCID

Affiliation:

1. Department of Growth and Reproduction, Copenhagen University Hospital - Rigshospitalet , Copenhagen 2100 , Denmark

2. Department of Clinical Medicine, University of Copenhagen , Copenhagen 2200 , Denmark

3. Division of Endocrinology, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine , Cincinnati, OH 45229 , United States

4. Clinical Drug Development, Novo Nordisk A/S , Søborg 2860 , Denmark

5. Department of Gastroenterology, Nutrition and Endocrinology, Research Institute, Osaka Women's and Children's Hospital , Izumi, Osaka 594-1101 , Japan

6. AP-HP, Université Paris Saclay, INSERM, Service d'Endocrinologie et Diabète de l'Enfant, Hôpital Bicêtre Paris Saclay , Le Kremlin-Bicêtre 94270 , France

7. Institute of Diabetes, Endocrinology and Metabolism, Rambam Health Care Center, Bruce Rappaport Faculty of Medicine , Technion, Haifa 31096 , Israel

8. Division of Endocrinology and Metabolism, National Center for Child Health and Development , Tokyo 157-8535 , Japan

Abstract

Abstract 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.

Funder

Novo Nordisk

Publisher

Oxford University Press (OUP)

Subject

Endocrinology,General Medicine,Endocrinology, Diabetes and Metabolism

Reference39 articles.

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