Clinical Trial

Growth Hormone in a Patient With a Dominant-Negative GHR Mutation

Active, Not Recruiting Phase 2

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.

Protocol Amendment History 6 amendments

This ClinicalTrials.gov record has been amended 6 times since 2022-05-16; most recent amendment 2025-06-30.

Status change: Enrolling by Invitation → Active, Not Recruiting 2024-02-12

Status change: Not Yet Recruiting → Enrolling by Invitation 2022-08-29

Trial Details

NCT Number	NCT05382637
Lead Sponsor	Children's National Research Institute
Conditions	Growth Disorders
Enrollment	1 participants
Start Date	2022-08-26
Primary Completion	2023-09-01 (estimated)
Study Completion	2028-09-01 (estimated)
Updated on ClinicalTrials.gov	2025-07-02