Efficacy and Safety Evaluation of Human Growth Hormone Therapy in Patients with Idiopathic Short Stature in Korea - A Randomised Controlled Trial

Eur Endocrinol. 2020 Apr;16(1):54-59. doi: 10.17925/EE.2020.16.1.54. Epub 2019 Oct 15.

Abstract

Background: This trial evaluated the efficacy and safety of growth hormone (GH) therapy (Norditropin®; Novo Nordisk, Bagsværd, Denmark) in paediatric patients with idiopathic short stature (ISS) in Korea.

Methods: This was an open-label, parallel-group, multicentre, interventional trial (ClinicalTrials.gov identifier: NCT01778023). Pre-pubertal patients (mean age 6.2 years; height, 107.1 cm) were randomised 2:1 to 12 months' GH treatment (0.469 mg/kg/week; group A, n=36) or 6 months untreated followed by 6 months' GH treatment (group B, n=18). Safety analysis was based on adverse events (AEs) in all GH-treated patients.

Results: After 6 months, height velocity (Ht-V), change in both height standard deviation score (Ht-SDS) and insulin-like growth factor 1 (mean difference [95% confidence interval {CI}]: 5.15 cm/year [4.09, 6.21]; 0.57 [0.43, 0.71]; 164.56 ng/mL [112.04, 217.08], respectively; all p<0.0001) were greater in group A than in group B. Mean difference in Ht-V for 0-6 months versus 6-12 months was 2.80 cm/year (95% CI 1.55, 4.04) for group A and -4.60 cm/year (95% CI -6.12, -3.09; both p<0.0001) for group B. No unexpected AEs were reported.

Conclusions: During the first 6 months, height was significantly increased in GH-treated patients versus untreated patients with ISS. Safety of GH was consistent with the known safety profile.

Keywords: Human growth hormone; clinical trial; idiopathic; short stature.

Associated data

  • ClinicalTrials.gov/NCT01778023

Grants and funding

Support: This trial was sponsored by Novo Nordisk Pharma Korea, Ltd. Novo Nordisk was responsible for preparing the protocol, providing clinical trial supplies, monitoring the trial, supervising activities of the clinical services vendor of electronic data capture system, central lab, statistical analysis, data management and medical writing, ensuring smooth running of the trial and appropriate safety surveillance. No funding was received in the publication of this article.