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Trial · Interventional

Active, not recruitingPhase 2Results postedRegistry data

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

NCTNCT05382637 (opens ClinicalTrials.gov in a new tab)Other idSTUDY00000211
Open at ClinicalTrials.govAsk about this trial

Overview

Status
Active, not recruiting
Phase
Phase 2
Enrollment
1 participants (actual)
Sponsor
Children's National Research Institute · Other
Design
Allocation not applicable · Open label (no masking)
Countries
1 country

United States

Start
26 Aug 2022
Primary completion
1 Sept 2023
Completion
1 Sept 2028 (estimated)
First posted
19 May 2022
Last update posted
2 Jul 2025
Conditions
Growth Disorders

Timeline

1 trial · 1 ongoing · 1 with posted results

  • Completed
  • Ongoing
  • Not yet recruiting
  • Stopped
  • Estimated dates (est.)
  • Results posted
2022202420262028
  1. Ph 2

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

    NCT05382637

    26 Aug 2022 to est. 1 Sept 2028

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

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.

Interventions

  • SomatropinDrug

Arms1

  • 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.

Primary outcomes1

Outcomes the sponsor listed as primary.

  1. Growth Hormone Dose

    Time frame · 1 year

    Registry description

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

Secondary outcomes(2)

  1. Growth Velocity

    Time frame · 1 year

    Registry description

    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

    Registry description

    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.

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

Primary outcome results

Primary outcomeNumber· 1 year

Growth Hormone Dose

GroupValue (mg/kg/day)
Growth Hormone0.250
Secondary and other outcome results(2)
Secondary outcomeNumber· 1 year

Growth Velocity

GroupValue (cm/year)
Growth Hormone8.7
Secondary outcomeNumber· Baseline to 1 Year

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

GroupValue (Standard deviations)
Growth Hormone0.81

Adverse events, as posted

Adverse events as posted: participants affected out of participants at risk, by group
GroupDeathsSerious eventsOther events
Growth HormoneThe participant will receive escalating dose of growth hormone until an IGF-1 level is maintained between the mean and +2 standard deviations. Somatropin: Daily growth hormone at a starting dose of 50 mcg/kg/day and escalating0 / 10 / 10 / 1

Each cell reads participants affected / participants at risk, as the registry posted them.

Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies0

Studies that cite this trial.

No study cites this trial.

Other registry references

References listed at the registry that are not in PepBank. They link to PubMed.

  • DerivedMerchant N, Houchin L, Boucher K, Dauber A. A Clinical Trial of High-Dose Growth Hormone in a Patient With a Dominant-Negative Growth Hormone Receptor Mutation. J Clin Endocrinol Metab. 2024 Oct 15;109(11):2937-2942. doi: 10.1210/clinem/dgae244. PMID 38597155

Eligibility

Who could join, as posted.

Sex
Male
Age
from 9 Years
Healthy volunteers
Not accepted
Inclusion and exclusion criteria, as posted
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.

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