Effective growth hormone replacement with once-weekly somapacitan in Japanese children with growth hormone deficiency: Results from REAL4, a phase 3 clinical trial.

Autor: Mori J; Division of Pediatric Endocrinology and Metabolism, Children's Medical Center, Osaka City General Hospital, Osaka, Japan., Ohata Y; Department of Pediatrics, Osaka University Graduate School of Medicine, Suita, Japan., Fujisawa Y; Department of Pediatrics, Hamamatsu University School of Medicine, Hamamatsu, Japan., Sato Y; Rare Disease Group, Novo Nordisk Pharma Ltd., Tokyo, Japan., Röhrich S; Global Medical Affairs, Novo Nordisk Health Care AG, Zürich, Switzerland., Rasmussen MH; Medical and Science, Rare Disease and Advanced Therapies, Clinical Drug Development, Novo Nordisk A/S, Søborg, Denmark., Bang RB; Biostatistics, Rare Disease and Advanced Therapies, Data Science, Novo Nordisk A/S, Aalborg, Denmark., Horikawa R; Division of Endocrinology and Metabolism, National Center for Child Health and Development, Tokyo, Japan.
Jazyk: angličtina
Zdroj: Clinical endocrinology [Clin Endocrinol (Oxf)] 2024 Apr; Vol. 100 (4), pp. 389-398. Date of Electronic Publication: 2024 Feb 18.
DOI: 10.1111/cen.15025
Abstrakt: Objective: Somapacitan is a long-acting growth hormone (GH) derivative developed for the treatment of GH deficiency (GHD). This study evaluates the efficacy and tolerability of somapacitan in Japanese children with GHD after 104 weeks of treatment and after switch from daily GH.
Design: Subanalysis on Japanese patients from a randomised, open-labelled, controlled parallel-group phase 3 trial (REAL4, NCT03811535).
Patients and Measurements: Thirty treatment-naïve patients were randomised 2:1 to somapacitan (0.16 mg/kg/week) or daily GH (0.034 mg/kg/day) up to Week 52, after which all patients received somapacitan. Height velocity (HV; cm/year) at Weeks 52 and 104 were the primary measurements. Additional assessments included HV SD score (SDS), height SDS, bone age, insulin-like growth factor-I (IGF-I) SDS, and observer-reported outcomes.
Results: At Week 52, observed mean HV was similar between treatment groups (10.3 vs. 9.8 cm/year for somapacitan and daily GH, respectively). Similar HVs between groups were also observed at Week 104: 7.4 cm/year after continuous somapacitan treatment (soma/soma) and 7.9 cm/year after 1-year somapacitan treatment following switch from daily GH (switch). Other height-related endpoints supported continuous growth. IGF-I SDS increased in both groups with mean IGF-I SDS within -2 and +2 during the study. Somapacitan was well tolerated, one mild injection site reaction was reported, with no reports of injection site pain. Patient preference questionnaires showed that most patients and their caregivers (90.9%) who switched treatment at Week 52 preferred once-weekly somapacitan over daily GH treatment.
Conclusions: Somapacitan showed sustained efficacy in Japanese children with GHD over 104 weeks and for 52 weeks after switching from daily GH. Somapacitan was well tolerated and preferred over daily GH.
(© 2024 Novo Nordisk A/S. Clinical Endocrinology published by John Wiley & Sons Ltd.)
Databáze: MEDLINE
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