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Clinical Trial
. 2023 Nov 17;108(12):3090-3099.
doi: 10.1210/clinem/dgad394.

Effective GH Replacement With Somapacitan in Children With GHD: REAL4 2-year Results and After Switch From Daily GH

Bradley S Miller  1 , Joanne C Blair  2 , Michael Højby Rasmussen  3 , Aristides Maniatis  4 , Jun Mori  5 , Volker Böttcher  6 , Ho-Seong Kim  7 , Rikke Beck Bang  8 , Michel Polak  9 , Reiko Horikawa  10
Affiliations
Clinical Trial

Effective GH Replacement With Somapacitan in Children With GHD: REAL4 2-year Results and After Switch From Daily GH

Bradley S Miller et al. J Clin Endocrinol Metab. .

Erratum in

Abstract

Context: Somapacitan is a long-acting GH derivative for treatment of GH deficiency (GHD).

Objective: Evaluate the efficacy and tolerability of somapacitan in children with GHD after 2 years of treatment and after the switch from daily GH.

Design: A randomized, multinational, open-labelled, controlled parallel group phase 3 trial, comprising a 52-week main phase and 3-year safety extension (NCT03811535).

Setting: Eighty-five sites across 20 countries.

Patients: A total of 200 treatment-naïve prepubertal patients were randomized and exposed; 194 completed the 2-year period.

Interventions: Patients were randomized 2:1 to somapacitan (0.16 mg/kg/wk) or daily GH (0.034 mg/kg/d) during the first year, after which all patients received somapacitan 0.16 mg/kg/wk.

Main outcome measures: Height velocity (HV; cm/year) at week 104. Additional assessments included HV SD score (SDS), height SDS, IGF-I SDS, and observer-reported outcomes.

Results: HV was sustained in both groups between 52 and 104 weeks. At week 104, mean (SD) for HV between weeks 52 and 104 was 8.4 (1.5) cm/year after continuous somapacitan treatment and 8.7 (1.8) cm/year after 1 year of somapacitan treatment following switch from daily GH. Secondary height-related endpoints also supported sustained growth. Mean IGF-I SDS during year 2 was similar between groups and within normal range (-2 to +2). Somapacitan was well tolerated, with no safety or tolerability issues identified. GH patient preference questionnaire results show that most patients and their caregivers (90%) who switched treatment at year 2 preferred once-weekly somapacitan over daily GH treatment.

Conclusions: Somapacitan in children with GHD showed sustained efficacy and tolerability for 2 years, and after switching from daily GH. Patients/caregivers switching from daily GH expressed a preference for somapacitan.

Clinical trial registration: NCT03811535.

Keywords: growth hormone; growth hormone deficiency; growth hormone replacement therapy; long-acting growth hormone; somapacitan.

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Figures

Figure 1.
Figure 1.
Trial overview and profile. (A) Design of the REAL4 trial and safety extension. Results from the main phase and first year of safety extension (104 weeks total) are reported in this study. Time axis is not to scale. (B) Population disposition of trial participants during the main trial period (weeks 0-52) and the first year of the extension period (weeks 52-104). The full analysis set (FAS) represents all randomly assigned children in the trial to either weekly somapacitan or daily GH (Norditropin). The safety analysis set (SAS) contains all randomly assigned children who received at least 1 dose of randomized treatment. A total of 127 and 67 children completed 104 weeks in soma/soma and switch groups, respectively; 132 and 68 were included in the FAS and SAS, respectively. *1 participant discontinued treatment in the main phase. Abbreviations: GHD, GH deficiency; SAS, safety analysis set.
Figure 2.
Figure 2.
Observed height velocity from baseline to week 104. Mean (SD) observed HV (cm/year) at baseline (week 0), week 52, and week 104 for the soma/soma and switch groups. Data are presented as mean with error bars representing SD. Abbreviations: HV, height velocity; soma, somapacitan.
Figure 3.
Figure 3.
Sustained increase in HSDS from baseline to week 104 for continued somapacitan treatment (soma/soma) and switch from daily GH to somapacitan treatment. (A) Observed mean HSDS at baseline (week 0), week 52, and week 104 for the soma/soma and switch groups. (B) Mean change in HSDS from baseline. Data are presented as mean with error bars representing SD. Abbreviations: HSDS, height SD score; soma, somapacitan.
Figure 4.
Figure 4.
IGF-I SDS remained in normal range in year 2. Model-derived means for weekly average IGF-I SDS in somapacitan-treated patients after 52 weeks of treatment (+0.52), between 52 and 104 weeks of treatment (+0.72) or following switch from daily GH to somapacitan in year 2 (+0.75) are compared with observed IGF­ – I SDS for daily GH at week 52 (+0.10). Data are presented as mean with error bars representing SD. Abbreviations: SDS, SD score; soma, somapacitan.
Figure 5.
Figure 5.
Patient preference for once-weekly somapacitan over daily GH. Observer-reported outcome assessments performed at week 56 using GH-PPQ for patients switching from daily GH to once-weekly somapacitan treatment at week 52. Abbreviations: PPQ, patient preference questionnaire.
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