Recombinant Human Growth Hormone Trial Aims to Improve Motor Development in Prader-Willi Syndrome

Prader-Willi Syndrome (PWS) is a complex genetic disorder characterized by hypotonia, short stature, cognitive impairment, and hyperphagia leading to obesity. Growth hormone deficiency is common in PWS, contributing to poor growth, reduced muscle mass, and impaired motor development. Current management often involves multidisciplinary care, but specific treatments to address motor and developmental delays are limited. Recombinant human growth hormone (rhGH) has shown promise in improving growth and body composition in PWS, but its impact on motor development warrants further investigation.

This single-arm, multicenter Phase III clinical trial is designed to evaluate the efficacy and safety of Recombinant Human Growth Hormone (rhGH) Injection (Jintropin AQ) in 30 patients with Prader-Willi Syndrome. Participants receive rhGH via subcutaneous injection once daily for a total of 52 weeks. The dosing regimen begins at 0.5 mg/m2/d for the initial 4 weeks, then escalates to 1.0 mg/m2/d for the subsequent 48 weeks. The primary endpoint is the change in total motor quotient, assessed using the Peabody Developmental Motor Scale at baseline, 26 weeks, and 52 weeks.

This Phase III clinical trial is currently evaluating the efficacy and safety of recombinant human growth hormone in patients with Prader-Willi Syndrome. As a trial registration, specific results are not yet available. However, the study aims to quantify improvements across several key developmental and anthropometric measures. The primary outcome focuses on motor development: The change in total motor quotient, as calculated by the Peabody Developmental Motor Scale, will be assessed at 26 weeks and 52 weeks post-treatment initiation. Secondary outcomes include changes in gross motor quotient and fine motor quotient, also measured by Peabody Developmental Motor Scale. Anthropometric changes are also key, with researchers tracking the change in height standard deviation score (SDS), body weight, and BMI standard deviation score at multiple time points (4 weeks, 13 weeks, 26 weeks, 39 weeks, and 52 weeks). Other developmental quotients, such as global, locomotor, personal-social, and language development quotients, are also being monitored.