A Real-world Study of the Efficacy and Safety of PEGylated Recombinant Human Growth Hormone Injection in the Treatment of Short Stature in Chinese Children From the Global Registry for Novel Therapies For Rare Bone or Endocrine Conditions

What this Trial Is About

Researchers are examining the long-term safety and effectiveness of polyethylene glycol-recombinant human growth hormone (PEG-rhGH) treatment for children with growth hormone deficiency, idiopathic short stature, small for gestational age (SGA), Turner syndrome, and other conditions causing short stature. This study collects and analyzes real-world data to explore factors affecting treatment success and to develop a height prediction model after therapy. The study contributes data to a global registry focused on rare endocrine and bone disorders. The study observes children treated with PEG-rhGH injections at doses ranging from 0.1 to 0.3 mg/kg weekly. It includes patients with different growth-related conditions such as GHD, ISS, SGA, and Turner syndrome, among others. Data are collected retrospectively and prospectively to monitor ongoing treatment outcomes and safety. All collected information is shared with the Global Registry for New Therapies for Rare Endocrine Diseases and Bone Disorders (GloBE-Reg). Participants undergo height measurements to assess changes in height standard deviation scores between ages 3 and 10 years. The study tracks safety and treatment responses over time through clinical evaluations and data analysis. The trial spans from August 2023 until August 2029, allowing long-term follow-up and monitoring of the real-world use of PEG-rhGH in children up to 18 years old.

The Efficacy and Safety of PEGylated GH for the Treatment of Short Stature in Chinese Children-GLOBE Reg