A 52-week, randomized, open-label, active-controlled, parallel-group phase 3 study of 161 treatment-naïve, prepubertal (Tanner stage 1) pediatric patients with GHD aged 3 years and older. Patients were randomized in a 2:1 ratio to receive either SKYTROFA® 0.24 mg/kg/week (n = 105) or daily somatropin 0.24 mg/kg/week (n = 56). The primary objective was to evaluate and compare the LS mean AHV of prepubertal children with growth failure due to GHD treated with weekly SKYTROFA with that of somatropin at 52 weeks.1,3
Once-weekly SKYTROFA® was studied in a multicenter, phase 3, open-label, 26-week trial investigating the safety, tolerability, and efficacy of SKYTROFA administered once weekly in children with GHD. The trial included 3 treatment-naïve and 143 treatment-experienced patients previously treated with daily hGH (≥ 0.20 mg hGH/kg/week) for ≤ 130 weeks. Children with open epiphyses aged 3 to 17 years must have been treated with daily hGH (≥ 0.20 mg hGH/kg/week) for 13 to 130 weeks. Safety and tolerability were the primary endpoints.3
SKYTROFA® is being studied in an ongoing open-label extension study of pediatric patients with GHD who previously participated in phase 3 SKYTROFA trials. The study enrolled a total of 298 patients: 103 patients on SKYTROFA and 55 patients on a daily somatropin from the heiGHt trial, and 140 patients on SKYTROFA from the fliGHt trial. The mean age at baseline was 9.54 years (range: 4.4 to 14.1 years) for SKYTROFA-treated patients from the heiGHt trial, 9.56 years (range: 4.2 to 13.9 years) for daily somatropin-treated patients from the heiGHt trial, and 11.11 years (range: 1.7 to 17.8 years) for patients from the fliGHt trial.4