TY - JOUR
T1 - Weekly Somapacitan is Effective and Well Tolerated in Children With GH Deficiency
T2 - The Randomized Phase 3 REAL4 Trial
AU - Miller, Bradley S.
AU - Blair, Joanne C.
AU - Rasmussen, Michael Højby
AU - Maniatis, Aristides
AU - Kildemoes, Rasmus Juul
AU - Mori, Jun
AU - Polak, Michel
AU - Bang, Rikke Beck
AU - Böttcher, Volker
AU - Stagi, Stefano
AU - Horikawa, Reiko
N1 - Publisher Copyright:
© 2022 The Author(s). Published by Oxford University Press on behalf of the Endocrine Society.
PY - 2022/12/1
Y1 - 2022/12/1
N2 - 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.
AB - 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.
KW - growth hormone
KW - growth hormone deficiency
KW - growth hormone replacement therapy
KW - long-acting growth hormone
KW - somapacitan
KW - treatment burden
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U2 - 10.1210/clinem/dgac513
DO - 10.1210/clinem/dgac513
M3 - Article
C2 - 36062966
AN - SCOPUS:85142939346
SN - 0021-972X
VL - 107
SP - 3378
EP - 3388
JO - Journal of Clinical Endocrinology and Metabolism
JF - Journal of Clinical Endocrinology and Metabolism
IS - 12
ER -