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About the Study

Who runs this study?

  • This study is being done at the National Institute of Child Health and Human Development at the National Institutes of Health.
  • Principal Investigator: Dr. Constantine A. Stratakis

What is this study about?

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.

What kind of commitment is involved?

  • Enrolled patients will need to take the medication for 12 months, unless otherwise decided by the principal investigator.
  • Participation in the study involves 3 visit at the NIH over one year, and regular visits with the local physician.

How can I ask questions or ask for help?