Safety and Efficacy of Pegvisomant in Pediatric Growth Hormone Excess

Abstract

Abstract Objective: Pediatric growth hormone excess (GHE) and gigantism are mainly managed by pituitary surgery. However, a large percentage of patients require further medical management. Data for the pediatric population are scares and decisions are usually based on adult published data. We have initiated a study to investigate the efficacy and safety of pegvisomant in children and adolescents with GHE. MethodsThe study is registered in ClinicalTrials.gov (Identifier: NCT03882034, An open-label phase 3 study of the safety and efficacy of pegvisomant in children with growth hormone excess). Eligible patients must be children or adolescents (age: 24 months -18 years) with GHE with persistent disease after surgical or radiation therapy. The patients will receive pegvisomant via subcutaneous daily injection for one year and outcomes will be measured at baseline and at the end of the study. The main measure of efficacy is the change of IGF-1 from baseline to the end of the study. The main measure of safety is the description of side effects over the duration of the study. ConclusionsWe will collect data on the safety and efficacy of medical treatment for pediatric patients with GHE.