A novel Y-shaped pegylated recombinant human growth hormone for children with growth hormone deficiency: a phase 3 study.

Context: Pegpesen® is a novel Y-shape pegylated recombinant human growth hormone (rhGH) for once-weekly treatment of children with growth hormone deficiency (GHD).

Objective: To evaluate the efficacy and safety of Pegpesen® in children with GHD vs daily rhGH.

Design: A multicenter, randomized, controlled phase 3 clinical trial with a duration of 52 weeks treatment.

Setting: 23 centers in China.

Participants: 391 pediatric participants diagnosed with GHD.

Interventions: Participants were randomized 2:1 to the weekly Pegpesen® group (140 μg/kg/week) or the daily rhGH group (245 μg/kg/week) for 52 weeks.

Main outcome measures: The primary endpoint was the growth velocity (GV) at 52 weeks, and the secondary endpoints mainly involved changes from baseline in height standard deviation scores for chronological age and bone age (ΔHt SDS CA and ΔHt SDS BA).

Results: At 52 weeks, the least squares mean (LS Means) of GV was 9.910 cm/year in the Pegpesen® group and 10.037 cm/year in the daily rhGH group. The LS Means difference between groups was -0.127 [95% CI (-0.4868, 0.2332)], confirming that weekly Pegpesen® is non-inferior to daily rhGH. The LS Means of ΔHt SDS CA, ΔHt SDS BA, were similar across both groups (all P > 0.05). Safety profiles and adherence were comparable.

Conclusions: Pegpesen® was non-inferior to daily rhGH, with similar safety, lower dosage requirements, thus presenting a new therapeutic option for children with GHD.