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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.
Age
9 - No limit years
Sex
MALE
Healthy Volunteers
No
Children's National Hospital
Washington D.C., District of Columbia, United States
Start Date
August 26, 2022
Primary Completion Date
September 1, 2023
Completion Date
September 1, 2028
Last Updated
July 2, 2025
1
ACTUAL participants
Somatropin
DRUG
Lead Sponsor
Children's National Research Institute
NCT06662045
NCT02413138
Data Source & Attribution
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