Weekly Growth Hormone Shows Promise for Children with Idiopathic Short Stature

Idiopathic Short Stature (ISS) is a condition where children are significantly shorter than their peers without an identifiable medical cause, impacting their physical and psychosocial development. Current treatments often involve daily injections of human growth hormone (hGH), which can be burdensome for children and their families, potentially affecting treatment adherence. This study aimed to evaluate the efficacy and safety of a long-acting, sustained-release human growth hormone (SR-hGH) formulation, administered weekly, as an alternative to daily injections in pre-pubertal children with ISS.

This Phase II study successfully completed its 26-week treatment period, enrolling 45 pre-pubertal children with Idiopathic Short Stature (ISS). The primary goal was to assess the efficacy and safety of SR-hGH at two different weekly doses (0.5 mg/kg/week and 0.7 mg/kg/week) compared to daily hGH (0.37 mg/kg/week). While the abstract confirms the study's completion and randomization of 45 patients across the three arms, specific quantitative results regarding height velocity, IGF-1 levels, or adverse event rates were not detailed in the provided summary. > The study's design successfully compared the safety and preliminary efficacy of weekly versus daily administration in this patient population, aiming to establish optimal dosing for SR-hGH.