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Growth Hormone in a Patient With a Dominant-Negative GHR Mutation
Sponsor: Children's National Research Institute
Summary
This is a prospective interventional study designed for a single patient with a dominant-negative mutation in the growth hormone receptor gene (GHR) which results in increased levels of growth hormone binding protein (GHBP). The patient will receive escalating doses of growth hormone titrated to achieve an insulin like growth factor-1 level above the mean and then growth response to therapy will be monitored.
Key Details
Gender
MALE
Age Range
9 Years - Any
Study Type
INTERVENTIONAL
Enrollment
1
Start Date
2022-08-26
Completion Date
2028-09-01
Last Updated
2025-07-02
Healthy Volunteers
No
Conditions
Interventions
Somatropin
Daily growth hormone at a starting dose of 50 mcg/kg/day and escalating
Locations (1)
Children's National Hospital
Washington D.C., District of Columbia, United States