- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT05382637
Growth Hormone in a Patient With a Dominant-Negative GHR Mutation
February 12, 2024 updated by: Children's National Research Institute
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.
Study Overview
Study Type
Interventional
Enrollment (Actual)
1
Phase
- Phase 2
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
-
-
District of Columbia
-
Washington, District of Columbia, United States, 20010
- Children's National Hospital
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
9 years and older (Child, Adult, Older Adult)
Accepts Healthy Volunteers
No
Description
Inclusion Criteria:
- Provision of signed and dated informed consent form
- Stated willingness to comply with all study procedures and availability for the duration of the study
- 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.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Growth hormone
The participant will receive escalating dose of growth hormone until an IGF-1 level is maintained between the mean and +2 standard deviations.
|
Daily growth hormone at a starting dose of 50 mcg/kg/day and escalating
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Growth hormone dose
Time Frame: 1 year
|
Dose of growth hormone required in mg/kg/day to achieve an IGF-1 level above the mean
|
1 year
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Growth Velocity
Time Frame: 1 year
|
Growth velocity during 1st year of growth hormone treatment
|
1 year
|
Height standard deviation
Time Frame: 1 year
|
Change in height standard deviation over the course of the 1st year of treatment with growth hormone
|
1 year
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Investigators
- Principal Investigator: Andrew Dauber, MD, Children's National Research Institute
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
August 26, 2022
Primary Completion (Actual)
September 1, 2023
Study Completion (Estimated)
September 1, 2028
Study Registration Dates
First Submitted
May 16, 2022
First Submitted That Met QC Criteria
May 16, 2022
First Posted (Actual)
May 19, 2022
Study Record Updates
Last Update Posted (Estimated)
February 13, 2024
Last Update Submitted That Met QC Criteria
February 12, 2024
Last Verified
February 1, 2024
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- STUDY00000211
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
NO
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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