Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Clinical Trial
. 2024 Apr;100(4):389-398.
doi: 10.1111/cen.15025. Epub 2024 Feb 18.

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

Jun Mori  1 , Yasuhisa Ohata  2 , Yasuko Fujisawa  3 , Yukihito Sato  4 , Sebastian Röhrich  5 , Michael Højby Rasmussen  6 , Rikke Beck Bang  7 , Reiko Horikawa  8
Affiliations
Clinical Trial

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

Jun Mori et al. Clin Endocrinol (Oxf). 2024 Apr.

Abstract

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.

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

PubMed Disclaimer

Similar articles

See all similar articles

References

REFERENCES

    1. Rothermel J, Reinehr T. Metabolic alterations in paediatric GH deficiency. Best Pract Res Clin Endocrinol Metab. 2016;30:757-770. doi:10.1016/j.beem.2016.11.004
    1. Yuen KCJ, Miller BS, Biller BMK. The current state of long-acting growth hormone preparations for growth hormone therapy. Curr Opin Endocrinol Diabetes Obes. 2018;25:267-273. doi:10.1097/MED.0000000000000416
    1. Acerini CL, Segal D, Criseno S, et al. Shared decision-making in growth hormone therapy-implications for patient care. Front Endocrinol. 2018;9:688. doi:10.3389/fendo.2018.00688
    1. Loftus J, Miller BS, Parzynski CS, et al. Association of daily growth hormone injection adherence and height among children with growth hormone deficiency. Endocr Pract. 2022;28:565-571. doi:10.1016/j.eprac.2022.02.013
    1. Tanaka T, Sato T, Yuasa A, Akiyama T, Tawseef A. Patient preferences for growth hormone treatment in Japanese children. Pediatr Int. 2021;63:1185-1191. doi:10.1111/ped.14760

Publication types

Associated data

Grants and funding

LinkOut - more resources