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Introduction

Growth hormone excess is a rare condition associated with high levels of growth hormone (GH). When it occurs prior to the complete fusion of growth plates, it leads to pathological tall stature, and it is called gigantism. Gigantism may be associated with debilitating cardiovascular disease and/or diabetes. Children and adolescents with gigantism are currently treated with surgery, radiation therapy, and/or medications; however, these treatments may lack efficacy and have significant side effects. Additionally no medication has been tested in pediatric population specifically. Pegvisomant is a GH-receptor (GHR) antagonist that blocks the action of GH. In adults with growth hormone excess, pegvisomant has been shown to effectively reduce serum insulin-like-growth factor type- 1 (IGF-1) concentrations and lead to clinical improvement. However, experience in children and adolescents is limited to a small number of case series.

We are conducting a clinical trial at the NICHD, NIH, to assess the efficacy and safety of pegvisomant in children and adolescents with gigantism. The study duration is 60-weeks, including 8 weeks of screening period and 48 weeks (12 months) of treatment with pegvisomant.

To get started:

  • Navigate through the menu to learn more about the study.
  • Log in to the application and fill out questionnaires.

Please contact us if you have any questions or concerns.