Weekly Somapacitan is Effective and Well Tolerated in Children With GH Deficiency: The Randomized Phase 3 REAL4 Trial

Bradley S. Miller, Joanne C. Blair, Michael Højby Rasmussen, Aristides Maniatis, Rasmus Juul Kildemoes, Jun Mori, Michel Polak, Rikke Beck Bang, Volker Böttcher, Stefano Stagi, Reiko Horikawa

Research output: Contribution to journalArticlepeer-review

17 Scopus citations

Abstract

Context: Somapacitan, a once-weekly reversible albumin-binding GH derivative, is evaluated in children with GH deficiency (GHD). Objective: To demonstrate efficacy and safety of somapacitan vs daily GH. Methods: REAL4 is a randomised, multinational, open-labeled, active-controlled parallel group phase 3 trial, comprising a 52-week main trial and 3-year extension (NCT03811535). Setting: Eighty-six sites across 20 countries. Patients: 200 treatment-naïve patients were randomized and exposed. Interventions: Patients were randomized 2:1 to somapacitan (0.16 mg/kg/wk) or daily GH (Norditropin; 0.034 mg/kg/d), administered subcutaneously. Main outcome measures: The primary endpoint was annualized height velocity (HV; cm/y) at week 52. Additional assessments included HV SD score (SDS), height SDS, bone age, IGF-I SDS, patient-reported outcomes, and safety measures. Results: Estimated mean HV at week 52 was 11.2 and 11.7 cm/y for somapacitan and daily GH, respectively. Noninferiority was confirmed. Changes in HV SDS, height SDS, bone age, and IGF-I SDS from baseline to week 52 were similar between treatment groups. At week 52, mean IGF-I SDS values were similar between treatment groups and within normal range (-2 to +2). Safety of somapacitan was consistent with the well-known daily GH profile. Low proportions of injection-site reactions were reported for somapacitan (5.3%) and daily GH (5.9%). Both treatments similarly reduced disease burden from baseline to week 52, whereas a greater treatment burden reduction was observed for somapacitan. Conclusions: Similar efficacy for somapacitan compared to daily GH was demonstrated over 52 weeks of treatment with comparable safety and mean IGF-I SDS levels in treatment-naïve children with GHD.

Original languageEnglish (US)
Pages (from-to)3378-3388
Number of pages11
JournalJournal of Clinical Endocrinology and Metabolism
Volume107
Issue number12
DOIs
StatePublished - Dec 1 2022

Bibliographical note

Publisher Copyright:
© The Author(s) 2022. Published by Oxford University Press on behalf of the Endocrine Society.

Keywords

  • growth hormone
  • growth hormone deficiency
  • growth hormone replacement therapy
  • long-acting growth hormone
  • somapacitan
  • treatment burden

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