Somapacitan Is Effective and Well Tolerated in Chinese Children with Growth Hormone Deficiency: A Randomized Controlled Phase 3 Study.

INTRODUCTION

REAL6 is a randomized, multi-centre, open-labelled, active-controlled parallel group phase 3 trial (NCT04970654) investigating once-weekly somapacitan, a reversible albumin-binding growth hormone (GH) derivative, in Chinese children with GH deficiency (GHD).

METHODS

110 treatment-naïve patients were randomized 2:1 to somapacitan (0.16 mg/kg/week) or daily GH (Norditropin®; 0.034 mg/kg/day) for 52 weeks. Annualized height velocity (HV; cm/year) at week 52 was the primary measurement. Additional assessments included HV standard deviation score (SDS), height SDS (HSDS), bone age, insulin-like growth factor-I (IGF-I) SDS, and observer-reported outcomes.

RESULTS

Estimated mean HV at week 52 was 11.0 and 10.4 cm/year for somapacitan and daily GH, respectively. Non-inferiority of somapacitan compared to daily GH was confirmed. Changes in HVSDS, HSDS, bone age, and IGF-I SDS from baseline to week 52 were similar between groups. At week 52, mean (SD) IGF-I SDS was within intended reference range (-2.0 to +2.0) and comparable between groups: +0.5 (1.4) for somapacitan versus +0.1 (1.2) for daily GH. Somapacitan was well tolerated with a safety profile consistent with the well-known safety profile of daily GH. A low proportion of injection site reactions were reported for somapacitan (2.7%), with no reports of injection site pain during the 52-week treatment period. Disease burden was reduced from baseline to week 52 for both treatments. Somapacitan reduced treatment burden compared to daily GH.

CONCLUSIONS

Efficacy and safety profiles were comparable for Chinese children with GHD treated with somapacitan or daily GH. Both treatments similarly reduced disease burden, while treatment burden was reduced with somapacitan.