Abstract 102: Once-weekly somapacitan versus daily growth hormone in children born small for gestational age: 1-year results from a randomised phase 2 trial
Abstract
Treatment of short stature in children born small for gestational age (SGA) requires daily growth hormone (GH) injections that can be burdensome for patients and caregivers. Once-weekly somapacitan is a long-acting GH in phase 3 development for replacement therapy in children with GH deficiency. We report the 52-week results of the first phase 2, multinational, randomised, open-label, controlled, dose-finding trial (NCT03878446) investigating efficacy and safety of somapacitan as treatment for short stature in children born SGA compared with daily GH (Norditropin®, Novo Nordisk A/S). Sixty-two (62; 35.5% female) GH-treatment-naïve, prepubertal children received 0.16, 0.20 or 0.24 mg/kg/week subcutaneous (s.c.) somapacitan, or 0.035 or 0.067 mg/kg/day s.c. GH. All children were subsequently included in a long-term safety extension trial. At week 52, the observed mean height velocity (HV) was 8.5, 10.3 and 10.6 cm/year for 0.16 (n = 12), 0.20 (n = 13) and 0.24 mg/kg/week (n = 12) somapacitan, respectively, versus 9.4 and 11.2 cm/year for 0.035 (n = 11) and 0.067 mg/kg/day (n = 13) GH, respectively. The effect of the three somapacitan doses vs. GH on HV was not statistically significantly different. A similar pattern as seen for HV was observed for changes from baseline in height standard deviation score (SDS) and HV SDS. Dose-dependent increases in IGF-I SDS were observed with somapacitan and GH, and significant exposure-response relationships to HV were established for…
How strong is the evidence?
Evidence Hub reads this as a randomized trial, done in people. Its evidence level is 3 of 5 (Moderate · trial / cohort / secondary analysis). Trials without a large randomized design, cohorts and secondary analyses give moderate evidence. Reported size: 13 participants or records.
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