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| Name | Class |
|---|---|
| Pfizer | INDUSTRY |
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The purpose of this research study is to learn about brain development in very young girls with Turner syndrome (TS) and the effect that growth hormone (GH) therapy has on early brain development.
Studies in older children and adults have found that IQ (intelligence quotient) in individuals with Turner syndrome (TS) and the general population are similar. However, many individuals with TS have a nonverbal learning disability which may cause problems with imagining and working with objects in space (for example: building blocks, working puzzles, copying designs, driving a car) as well as problems in planning, paying attention, and getting along with people their own age. Studies of brain structure have found changes in the size and function of specific areas of the brain that are involved in these nonverbal processes. However, there are no data published on brain structure or function in girls with Turner Syndrome in the first few years of life when brains are learning and growing most rapidly.
In a recent study, very young, short girls with Turner Syndrome (TS)(averaging 2 years in age) were treated with Growth Hormone (GH) and almost reached an average height after 2 years. The risks of GH for the young girls in that study appeared to be the same as those for older girls. Therefore, it is now recommended that GH therapy be considered as soon as a child with TS has growth failure. Growth failure often occurs during infancy; therefore, more children with TS are now receiving GH therapy as young as 12 months of age. In this study, the investigators wish to start understanding what effect GH has on learning and brain growth when given between the ages of 12 and 24 months. In this study, the girls with TS will have developmental studies, a physical examination, magnetic resonance imaging (MRI), and blood drawn at one and two years of age. Some of the girls in this study will receive GH from 12 until 24 months of life, while others will not. Brain growth and development will be compared between those who have been treated with GH and those who have not. This is a pilot study in which the investigators will gather data to design a larger study that can answer these questions.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Growth Hormone | Experimental | Subjects in the somatotropin (growth hormone, GH) arm will receive GH injections from 12-24 months of life. |
|
| Control | No Intervention | Subjects will receive no GH or placebo. |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| somatotropin | Drug | Subjects will receive 5 mg somatotropin (growth hormone) pens with cartridges. Subcutaneous injections are to be given every evening around bedtime. Dosing regimen: 50 mcg/kg/day to be adjusted at 4 month intervals to the closest 0.1 mg. Subjects will be given 12 months of treatment (from 12 to 24 months of life). Subjects will visit their pediatrician or pediatric endocrinologist at 4 and 8 months of life. |
| Measure | Description | Time Frame |
|---|---|---|
| Total Brain Volume | Percent change in total brain volume as determined by magnetic resonance imaging (MRI) | Change in volume from 12 months of age scan in 24 months of age scan |
| Measure | Description | Time Frame |
|---|---|---|
| Volume of Brain Lobes (Occipital) | Percent change in volumes of occipital lobes as determined by MRI. | Change in volume from 12 months of age scan in 24 months of age scan |
| White Matter Tracts (SLF) |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Rebecca Knickmeyer | University of North Carolina, Chapel Hill | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of North Carolina at Chapel Hill | Chapel Hill | North Carolina | 27599 | United States |
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Subjects were recruited locally in the UNC_Chapel Hill pediatric endocrinology clinic and nationally through the Turner Syndrome Society, Turner Syndrome family internet groups, and advertisements on our UNC research website.
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| ID | Title | Description |
|---|---|---|
| FG000 | Growth Hormone | Subjects in the growth hormone (GH) arm will receive GH injections from 12-24 months of life. somatotropin: Subjects will receive 5 mg somatotropin (growth hormone) pens with cartridges. Subcutaneous injections are to be given every evening around bedtime. Dosing regimen: 50 mcg/kg/day to be adjusted at 4 month intervals to the closest 0.1 mg. Subjects will be given 12 months of treatment (from 12 to 24 months of life). Subjects will visit their pediatrician or pediatric endocrinologist at 4 and 8 months of life. |
| FG001 | Control | Subjects will receive no GH or placebo. |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Growth Hormone | Subjects in the growth hormone (GH) arm will receive GH injections from 12-24 months of life. somatotropin: Subjects will receive 5 mg somatotropin (growth hormone) pens with cartridges. Subcutaneous injections are to be given every evening around bedtime. Dosing regimen: 50 mcg/kg/day to be adjusted at 4 month intervals to the closest 0.1 mg. Subjects will be given 12 months of treatment (from 12 to 24 months of life). Subjects will visit their pediatrician or pediatric endocrinologist at 4 and 8 months of life. |
| 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 | Total Brain Volume | Percent change in total brain volume as determined by magnetic resonance imaging (MRI) | Note that only subjects with usable MRI data are included in this analysis (this is a subset of all children completing the protocol). | Posted | Mean | Standard Deviation | percentage change | Change in volume from 12 months of age scan in 24 months of age scan |
|
Baseline and 1 year
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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 | Subjects in the somatotropin (growth hormone, GH) arm will receive GH injections from 12-24 months of life. somatotropin: Subjects will receive 5 mg somatotropin (growth hormone) pens with cartridges. Subcutaneous injections are to be given every evening around bedtime. Dosing regimen: 50 mcg/kg/day to be adjusted at 4 month intervals to the closest 0.1 mg. Subjects will be given 12 months of treatment (from 12 to 24 months of life). Subjects will visit their pediatrician or pediatric endocrinologist at 4 and 8 months of life. |
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Low scan success rate and subject drop out resulted in small sample size (5 GH treated, 1 control).
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Rebecca Knickmeyer Santelli | UNC Chapel Hill | 9199662216 | rebecca_knickmeyer@med.unc.edu |
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| ID | Term |
|---|---|
| D014424 | Turner Syndrome |
| ID | Term |
|---|---|
| D006059 | Gonadal Dysgenesis |
| D012734 | Disorders of Sex Development |
| D014564 | Urogenital Abnormalities |
| D052776 | Female Urogenital Diseases |
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| ID | Term |
|---|---|
| D013006 | Growth Hormone |
| D019382 | Human Growth Hormone |
| ID | Term |
|---|---|
| D010908 | Pituitary Hormones, Anterior |
| D010907 | Pituitary Hormones |
| D036361 | Peptide Hormones |
| D006728 | Hormones |
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|
|
Change in the fractional anisotropy (FA) of white matter tracts using Diffusion Tensor Imaging (DTI); superior longitudinal fasciculus
| Change in FA from 12 months of age scan in 24 months of age scan |
| Volume of Brain Lobes (Central) | Percent change in volumes of central brain region (precentral gyrus, postcentral gyrus, rolandic operculum) as determined by MRI. | Change in volume from 12 months of age scan in 24 months of age scan |
| Volume of Brain Lobes (Frontal) | Percent change in volumes of frontal lobes as determined by MRI. | Change in volume from 12 months of age scan in 24 months of age scan |
| Volume of Brain Lobes (Temporal) | Percent change in volumes of temporal lobes as determined by MRI. | Change in volume from 12 months of age scan in 24 months of age scan |
| Volume of Brain Lobes (Parietal) | Percent change in volumes of parietal lobes as determined by MRI. | Change in volume from 12 months of age scan in 24 months of age scan |
| Volume of Brain Lobes (Limbic) | Percent change in volumes of parietal lobes as determined by MRI. | Change in volume from 12 months of age scan in 24 months of age scan |
| Volume of Brain Lobes (Insular Cortex) | Percent change in volumes of parietal lobes as determined by MRI. | Change in volume from 12 months of age scan in 24 months of age scan |
| BG001 | Control | Subjects will receive no GH or placebo. |
| BG002 | Total | Total of all reporting groups |
| Participants |
|
| Age, Continuous | Mean | Standard Deviation | months |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| Units | Counts |
|---|---|
| Participants |
|
|
| Secondary | Volume of Brain Lobes (Occipital) | Percent change in volumes of occipital lobes as determined by MRI. | Note that only subjects with usable MRI data are included in this analysis (this is a subset of all children completing the protocol). | Posted | Mean | Standard Deviation | percentage change | Change in volume from 12 months of age scan in 24 months of age scan |
|
|
|
| Secondary | White Matter Tracts (SLF) | Change in the fractional anisotropy (FA) of white matter tracts using Diffusion Tensor Imaging (DTI); superior longitudinal fasciculus | Note that only subjects with usable MRI data are included in this analysis (this is a subset of all children completing the protocol). | Posted | Mean | Standard Deviation | percentage change | Change in FA from 12 months of age scan in 24 months of age scan |
|
|
|
| Secondary | Volume of Brain Lobes (Central) | Percent change in volumes of central brain region (precentral gyrus, postcentral gyrus, rolandic operculum) as determined by MRI. | Note that only subjects with usable MRI data are included in this analysis (this is a subset of all children completing the protocol). | Posted | Mean | Standard Deviation | percentage change | Change in volume from 12 months of age scan in 24 months of age scan |
|
|
|
| Secondary | Volume of Brain Lobes (Frontal) | Percent change in volumes of frontal lobes as determined by MRI. | Note that only subjects with usable MRI data are included in this analysis (this is a subset of all children completing the protocol). | Posted | Mean | Standard Deviation | percentage change | Change in volume from 12 months of age scan in 24 months of age scan |
|
|
|
| Secondary | Volume of Brain Lobes (Temporal) | Percent change in volumes of temporal lobes as determined by MRI. | Note that only subjects with usable MRI data are included in this analysis (this is a subset of all children completing the protocol). | Posted | Mean | Standard Deviation | percentage change | Change in volume from 12 months of age scan in 24 months of age scan |
|
|
|
| Secondary | Volume of Brain Lobes (Parietal) | Percent change in volumes of parietal lobes as determined by MRI. | Note that only subjects with usable MRI data are included in this analysis (this is a subset of all children completing the protocol). | Posted | Mean | Standard Deviation | percentage change | Change in volume from 12 months of age scan in 24 months of age scan |
|
|
|
| Secondary | Volume of Brain Lobes (Limbic) | Percent change in volumes of parietal lobes as determined by MRI. | Note that only subjects with usable MRI data are included in this analysis (this is a subset of all children completing the protocol). | Posted | Mean | Standard Deviation | percentage change | Change in volume from 12 months of age scan in 24 months of age scan |
|
|
|
| Secondary | Volume of Brain Lobes (Insular Cortex) | Percent change in volumes of parietal lobes as determined by MRI. | Note that only subjects with usable MRI data are included in this analysis (this is a subset of all children completing the protocol). | Posted | Mean | Standard Deviation | percentage change | Change in volume from 12 months of age scan in 24 months of age scan |
|
|
|
| 0 |
| 11 |
| 0 |
| 11 |
| EG001 | Control | Subjects will receive no GH or placebo. | 0 | 6 | 0 | 6 |
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| D005261 | Female Urogenital Diseases and Pregnancy Complications |
| D000091642 | Urogenital Diseases |
| D058533 | Sex Chromosome Disorders of Sex Development |
| D052801 | Male Urogenital Diseases |
| D006330 | Heart Defects, Congenital |
| D018376 | Cardiovascular Abnormalities |
| D002318 | Cardiovascular Diseases |
| D006331 | Heart Diseases |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D025064 | Sex Chromosome Disorders |
| D025063 | Chromosome Disorders |
| D030342 | Genetic Diseases, Inborn |
| D006058 | Gonadal Disorders |
| D004700 | Endocrine System Diseases |
| D006730 |
| Hormones, Hormone Substitutes, and Hormone Antagonists |
| D010455 | Peptides |
| D000602 | Amino Acids, Peptides, and Proteins |