Somapacitan in children born small for gestational age: a multi-centre, open-label, controlled phase 2 study
An erratum to this article was published (Eur J Endocrinol, 10 Jan 2023 ↗)
Authors' conclusion
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.
Study context
- Population
- Growth hormone (GH) treatment-naïve, prepubertal short children born small for gestational age (SGA); 38 sites across 12 countries61 completed main phase
- Participants
- 62 participants
- Comparison
- Once-weekly somapacitan vs daily GH
- Somapacitan 0.16n = 12
- Somapacitan 0.20n = 13
- Somapacitan 0.24n = 12
- Daily GH 0.035n = 12reference
- Daily GH 0.067n = 13reference
Methodological detailsmulti-centre, open-label, phase 2, 26-week main phase and 4-year extension
- Multi-centre
- Open-label
- Phase 2
- 26-week main phase and 4-year extension
Primary result
As reported in the abstract
Estimated mean annualised height velocity · 26 weeks
Further findings
Changes from baseline in height velocity (HV) standard deviation score (SDS), height SDS and IGF-I SDS · 26 weeks
- Similar dose-dependent responses
Exposure-response modelling
- Greatest efficacy correlated with the highest somapacitan exposure
Safety
- Similar safety and tolerability for all weekly somapacitan and daily GH doses
Original publication
Somapacitan in children born small for gestational age: a multi-centre, open-label, controlled phase 2 study. Eur J Endocrinol. Published 10 January 2023. doi:10.
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