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

NCT05382637 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 1

Last updated 2025-07-02

Study results available
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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.

Conditions

  • Growth Disorders

Interventions

DRUG

Somatropin

Daily growth hormone at a starting dose of 50 mcg/kg/day and escalating

Sponsors & Collaborators

  • Children's National Research Institute

    lead OTHER

Principal Investigators

  • Andrew Dauber, MD · Children's National Research Institute

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
9 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-08-26
Primary Completion
2023-09-01
Completion
2028-09-01
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05382637 on ClinicalTrials.gov