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

Primary Purpose

Growth Disorders

Status
Enrolling by invitation
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Somatropin
Sponsored by
Children's National Research Institute
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Growth Disorders

Eligibility Criteria

9 Years - undefined (Child, Adult, Older Adult)MaleDoes not accept healthy volunteers

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.

Sites / Locations

  • Children's National Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Growth hormone

Arm Description

The participant will receive escalating dose of growth hormone until an IGF-1 level is maintained between the mean and +2 standard deviations.

Outcomes

Primary Outcome Measures

Growth hormone dose
Dose of growth hormone required in mg/kg/day to achieve an IGF-1 level above the mean

Secondary Outcome Measures

Growth Velocity
Growth velocity during 1st year of growth hormone treatment
Height standard deviation
Change in height standard deviation over the course of the 1st year of treatment with growth hormone

Full Information

First Posted
May 16, 2022
Last Updated
August 29, 2022
Sponsor
Children's National Research Institute
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1. Study Identification

Unique Protocol Identification Number
NCT05382637
Brief Title
Growth Hormone in a Patient With a Dominant-Negative GHR Mutation
Official Title
Growth Hormone in a Patient With a Dominant-Negative GHR Mutation
Study Type
Interventional

2. Study Status

Record Verification Date
August 2022
Overall Recruitment Status
Enrolling by invitation
Study Start Date
August 26, 2022 (Actual)
Primary Completion Date
September 1, 2023 (Anticipated)
Study Completion Date
September 1, 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Children's National Research Institute

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

Brief 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Growth Disorders

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
1 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Growth hormone
Arm Type
Experimental
Arm Description
The participant will receive escalating dose of growth hormone until an IGF-1 level is maintained between the mean and +2 standard deviations.
Intervention Type
Drug
Intervention Name(s)
Somatropin
Intervention Description
Daily growth hormone at a starting dose of 50 mcg/kg/day and escalating
Primary Outcome Measure Information:
Title
Growth hormone dose
Description
Dose of growth hormone required in mg/kg/day to achieve an IGF-1 level above the mean
Time Frame
1 year
Secondary Outcome Measure Information:
Title
Growth Velocity
Description
Growth velocity during 1st year of growth hormone treatment
Time Frame
1 year
Title
Height standard deviation
Description
Change in height standard deviation over the course of the 1st year of treatment with growth hormone
Time Frame
1 year

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
9 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Andrew Dauber, MD
Organizational Affiliation
Children's National Research Institute
Official's Role
Principal Investigator
Facility Information:
Facility Name
Children's National Hospital
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20010
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
No

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

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