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Active, Not Recruiting

NCT Number: NCT05382637

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

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.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Key information

Conditions

Growth Disorders Pathologic Processes Pathological Conditions, Signs and Symptoms

Age range

9 year and older

Sex eligibility

Male

Study type

Interventional

Phase

Phase 2

Primary location

Children's National Hospital

Washington D.C., District of Columbia, 20010, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Somatropin

Drug

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

Primary outcomes

  1. 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

Secondary outcomes

  1. Growth Velocity

    Time frame: 1 year

    Growth velocity during 1st year of growth hormone treatment

  2. Height Standard Deviation Score Using Center for Disease Control (CDC) Growth Chart

    Time frame: Baseline to 1 Year

    Change in age and sex adjusted height standard deviation score based on the US CDC growth charts over the course of the 1st year of treatment with growth hormone. Height standard deviation score based on the US CDC growth chart data. The CDC provides tables of both height percentiles and standard deviations of the distribution of heights by age and sex. The CDC has a SAS program that allows for calculation of an individual's height standard deviation score based on the population data. This is the standard way that height is recorded in many growth studies.

Sponsors and collaborators

Lead sponsor

Children's National Research Institute

Other

Registry information

Important dates

Study start
2022
Primary completion
2023
Study completion
2028
First posted
May 19, 2022
Registry last updated
Jul 2, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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