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

• Sponsor: Children's National Research Institute (Other)
• Overall Status: Not yet recruiting
• CT.gov ID: NCT05382637
• Collaborator: (none)
• Enrollment: 1
• Location: 1
• Arm: 1
• Anticipated Duration (Months): 14
• Patients Per Site Per Month: 0.1

Study Details

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.

Condition or Disease	Intervention/Treatment	Phase
Growth Disorders	Drug: Somatropin	Phase 2

Study Design

• Study Type: Interventional
• Anticipated Enrollment: 1 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Growth Hormone in a Patient With a Dominant-Negative GHR Mutation
• Anticipated Study Start Date: Jul 1, 2022
• Anticipated Primary Completion Date: Sep 1, 2023
• Anticipated Study Completion Date: Sep 1, 2023

Arms and Interventions

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

Outcome Measures

Primary Outcome Measures

1. Growth hormone dose [1 year]

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

Secondary Outcome Measures

1. Growth Velocity [1 year]

   Growth velocity during 1st year of growth hormone treatment

2. Height standard deviation score [1 year]

   Change in height standard deviation score over the course of the 1st year of treatment with growth hormone

Eligibility Criteria

Criteria

• Ages Eligible for Study: 9 Years and Older
• Sexes Eligible for Study: Male
• Accepts Healthy Volunteers: No

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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Children's National Hospital	Washington	District of Columbia	United States	20010

Sponsors and Collaborators

• Children's National Research Institute

Investigators

• Principal Investigator: Andrew Dauber, MD, Children's National Research Institute

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Children's National Research Institute
• ClinicalTrials.gov Identifier: NCT05382637
• Other Study ID Numbers: STUDY00000211
• First Posted: May 19, 2022
• Last Update Posted: May 19, 2022
• Last Verified: May 1, 2022

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Additional relevant MeSH terms: Growth Disorders

Study Results

No Results Posted as of May 19, 2022