Pegylated Somatropin dose-response trial in 96 SGA children establishes basis for Phase III short stature treatment.

Children born Small for Gestational Age (SGA) often experience persistent short stature, impacting their quality of life. Recombinant human growth hormone (rhGH) is a standard treatment, but its daily administration can lead to poor adherence and significant burden for patients and families. Developing a long-acting, weekly formulation like Pegylated Somatropin could significantly improve treatment convenience and compliance, addressing a critical gap in current therapeutic options for SGA short stature management. This study aims to optimize dosing for such an improved regimen.

This multicenter, randomized, open-label, dose-response trial in China included 96 GH-treatment-naïve, non-GH-deficient, prepubertal short children born SGA. Participants were randomized 1:1 to receive weekly subcutaneous administration of Jintrolong 0.1 mg/kg/week or 0.2 mg/kg/week for 52 weeks. Children completing the main phase proceeded to an extension phase, initially receiving Jintrolong 0.2 mg/kg/week. Dose adjustments, based on annualized height velocity (AHV) and insulin-like growth factor I (IGF-I) response, were made up to a maximum of 0.4 mg/kg/week until near-adult height (NAH) was achieved or voluntary discontinuation.

The study successfully completed its 52-week main phase, providing a scientific and reliable basis for dose selection in future Phase III clinical trials. The trial design involved the randomization of 96 children, with weekly subcutaneous administration of Jintrolong at initial doses of 0.1 mg/kg/week or 0.2 mg/kg/week. An ongoing safety extension phase continues until near-adult height (NAH), allowing for dose adjustments up to a maximum of 0.4 mg/kg/week based on annualized height velocity (AHV) and insulin-like growth factor I (IGF-I) response. No specific efficacy or safety results, such as height velocity improvements or adverse event rates, are detailed in this abstract. However, the study's primary objective was to evaluate optimal dosing and initial efficiency and safety. > The main phase provided a scientific, reliable basis for Phase III clinical trials for dose selection.