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

Growth Disorders Clinical Trial

Official title:

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT05382637
• Other study ID #: STUDY00000211
• Status: Active, not recruiting
• Phase: Phase 2
• Start date: August 26, 2022
• Est. completion date: September 1, 2028

Study information

• Verified date: February 2024
• Source: Children's National Research Institute
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 1
• Est. completion date: September 1, 2028
• Est. primary completion date: September 1, 2023
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 9 Years and older

Eligibility

Inclusion Criteria:

1. Provision of signed and dated informed consent form

2. Stated willingness to comply with all study procedures and availability for the duration of the study

3. Be the specific subject with the a specific mutation in GHR leading to high GHBP.

Exclusion Criteria:

There are no exclusion criteria for this study.

Related Conditions & MeSH terms

• Growth Disorders

Intervention

Drug:
• Somatropin
Daily growth hormone at a starting dose of 50 mcg/kg/day and escalating

Locations

Country	Name	City	State
United States	Children's National Hospital	Washington	District of Columbia

Sponsors (1)

Lead Sponsor	Collaborator
Children's National Research Institute

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Growth hormone dose	Dose of growth hormone required in mg/kg/day to achieve an IGF-1 level above the mean	1 year
Secondary	Growth Velocity	Growth velocity during 1st year of growth hormone treatment	1 year
Secondary	Height standard deviation	Change in height standard deviation over the course of the 1st year of treatment with growth hormone	1 year