Somapacitan in children born small for gestational age: a multi-centre, open-label, controlled phase 2 study.
Eur J Endocrinol
; 188(1)2023 Jan 10.
Article
en En
| MEDLINE
| ID: mdl-36651161
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 11.3â cm/year for somapacitan 0.16, 0.20, and 0.24â mg/kg/week, respectively, compared to 10.3 and 11.9â cm/year for daily GH 0.035 and 0.067â 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 0.24â mg/kg/week appears most efficacious, providing similar efficacy, safety, and tolerability as daily GH 0.067â mg/kg/day in short children born SGA after 26 weeks of treatment.Palabras clave
Texto completo:
1
Colección:
01-internacional
Base de datos:
MEDLINE
Asunto principal:
Estatura
/
Recién Nacido Pequeño para la Edad Gestacional
/
Hormona de Crecimiento Humana
Tipo de estudio:
Clinical_trials
/
Prognostic_studies
Límite:
Child
/
Female
/
Humans
/
Newborn
Idioma:
En
Revista:
Eur J Endocrinol
Asunto de la revista:
ENDOCRINOLOGIA
Año:
2023
Tipo del documento:
Article
País de afiliación:
Dinamarca