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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Growth hormone | Experimental | The participant will receive escalating dose of growth hormone until an IGF-1 level is maintained between the mean and +2 standard deviations. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Somatropin | Drug | Daily growth hormone at a starting dose of 50 mcg/kg/day and escalating |
|
| Measure | Description | Time Frame |
|---|---|---|
| Growth Hormone Dose | Dose of growth hormone required in mg/kg/day to achieve an IGF-1 level above the mean | 1 year |
| Measure | Description | Time Frame |
|---|---|---|
| Growth Velocity | Growth velocity during 1st year of growth hormone treatment | 1 year |
| Height Standard Deviation Score Using Center for Disease Control (CDC) Growth Chart | 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. |
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Inclusion Criteria:
Exclusion Criteria:
There are no exclusion criteria for this study.
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| Name | Affiliation | Role |
|---|---|---|
| Andrew Dauber, MD | Children's National Research Institute | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's National Hospital | Washington D.C. | District of Columbia | 20010 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 38597155 | Derived | Merchant 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. |
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| ID | Title | Description |
|---|---|---|
| FG000 | 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. Somatropin: Daily growth hormone at a starting dose of 50 mcg/kg/day and escalating |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
1
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| ID | Title | Description |
|---|---|---|
| BG000 | 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. Somatropin: Daily growth hormone at a starting dose of 50 mcg/kg/day and escalating |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Growth Hormone Dose | Dose of growth hormone required in mg/kg/day to achieve an IGF-1 level above the mean | Posted | Number | mg/kg/day | 1 year |
|
|
1 year of study
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | 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. Somatropin: Daily growth hormone at a starting dose of 50 mcg/kg/day and escalating |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Andrew Dauber, MD | Children's National Hospital | 202-476-2121 | adauber@childrensnational.org |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Apr 28, 2023 | Jan 2, 2025 | Prot_SAP_000.pdf |
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| ID | Term |
|---|---|
| D006130 | Growth Disorders |
| ID | Term |
|---|---|
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
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| ID | Term |
|---|---|
| D019382 | Human Growth Hormone |
| ID | Term |
|---|---|
| D013006 | Growth Hormone |
| D010908 | Pituitary Hormones, Anterior |
| D010907 | Pituitary Hormones |
| D036361 | Peptide Hormones |
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| Baseline to 1 Year |
| Participants |
|
| Age, Continuous | Mean | Full Range | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| Baseline Height standard deviation score | Baseline 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. | Number | Standard deviation score from the CDC |
|
|
| Secondary | Growth Velocity | Growth velocity during 1st year of growth hormone treatment | Posted | Number | cm/year | 1 year |
|
|
|
| Secondary | Height Standard Deviation Score Using Center for Disease Control (CDC) Growth Chart | 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 | Number | Standard deviations | Baseline to 1 Year |
|
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| 0 |
| 1 |
| 0 |
| 1 |
| 0 |
| 1 |
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| D006728 |
| Hormones |
| D006730 | Hormones, Hormone Substitutes, and Hormone Antagonists |
| D010455 | Peptides |
| D000602 | Amino Acids, Peptides, and Proteins |