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 endpoint
was AHV 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 for ≤ 130 weeks. The mean daily somatropin dose was
0.29 mg/kg/week upon entering the trial. 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 daily somatropin
from the heiGHt trial, and 140 patients on SKYTROFA from the
fliGHt trial. The mean age at baseline was 9.5 years for
SKYTROFA-treated patients from the heiGHt trial, 9.5 years
for daily somatropin-treated patients from the heiGHt trial,
and 11.1 years for patients from the fliGHt trial. Long-term
safety was the primary endpoint.4