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| ID | Type | Description | Link |
|---|---|---|---|
| U111112218502 | Other Grant/Funding Number | Novo Nordisk |
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| Name | Class |
|---|---|
| Novo Nordisk A/S | INDUSTRY |
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The purpose of the study is to measure the functional effects of recombinant GH in skeletal muscle, in addition to growth promotion, in short prepubertal boys with either growth hormone deficiency or idiopathic short stature. Patients will be similarly short. The investigators will also compare these values in the short stature cohort to those obtained in testing performed in normally growing age-matched healthy control boys not on GH. The group on GH will be studied before and after 6 and 12 months of GH treatment.
Prepubertal boys with significant short stature (height SDS ≤-2.0) diagnosed with either GH deficiency or idiopathic short stature (ISS) who are identified as candidates for GH treatment will recruited. Subjects will have a battery of studies to assess skeletal muscle strength, agility, power and endurance, as well as assessment of body composition and energy expenditure before and after GH administration. GH (Somatropin) treatment at standard doses will be given as daily injections at bedtime. Subjects will be followed at three-month intervals per the clinical routine, when anthropometric measurements will be obtained. Baseline studies will be repeated at 6 and 12 months from initiation of treatment, each patient will serve as his own control pre and post GH.
A group of normally statured healthy boys will have the same testing as the study patients but without GH treatment to assess the impact of natural growth on the muscle measures above.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Short Stature Boys | Prepubertal boys with short stature defined as a height ≤-2 SDS with either GH deficiency (defined as peak GH responses to pharmacologic stimuli <10ng/ml) or idiopathic short stature (i.e., no identifiable pathology) will be studied pre and post 12 months of GH therapy. |
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| Normally Statured Boys | A group of 15 healthy, normally statured (between 10th- 90th %), age-matched boys not on Growth Hormone replacement, preferably siblings (although not exclusively), will be recruited to serve as healthy controls. |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Somatropin injection | Drug | Boys with short stature will be studied for measures of:
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| Measure | Description | Time Frame |
|---|---|---|
| Skeletal muscle strength (Newtons) | Upper and lower body muscle strength will be assessed using a handheld dynamometer for manual muscle testing of peak force | 12 months |
| Measure | Description | Time Frame |
|---|---|---|
| Skeletal muscle power (Watts) | Lower extremity power will be assessed by vertical jump performed using a Power time mat | 12 months |
| Muscle agility (seconds) | Muscle agility will be assessed by a timed shuttle run |
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Inclusion Criteria:
Short Stature Group
Normal Stature Group
Exclusion Criteria:
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Prepubertal boys with short stature defined as a height ≤-2 SDS with either GH deficiency (i.e., peak GH responses to pharmacologic stimuli <10ng/ml) or idiopathic short stature (i.e., normal GH, no identifiable pathology) will be studied.
Normally Statured Boys: A group of 15 healthy, normally statured (between 10th- 90th %), age-matched boys not on somatropin, preferably siblings (although not exclusively) will be studied similarly.
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| Name | Affiliation | Role |
|---|---|---|
| Nelly Mauras, MD | Nemours Children's Clinic | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Nemours Children's Clinic | Jacksonville | Florida | 32207 | United States |
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| ID | Term |
|---|---|
| D004393 | Dwarfism, Pituitary |
| D004392 | Dwarfism |
| ID | Term |
|---|---|
| D001848 | Bone Diseases, Developmental |
| D001847 | Bone Diseases |
| D009140 | Musculoskeletal Diseases |
| D001849 | Bone Diseases, Endocrine |
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| ID | Term |
|---|---|
| D019382 | Human Growth Hormone |
| D013006 | Growth Hormone |
| ID | Term |
|---|---|
| D010908 | Pituitary Hormones, Anterior |
| D010907 | Pituitary Hormones |
| D036361 | Peptide Hormones |
| D006728 | Hormones |
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Serum/plasma will be obtained.
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| 12 months |
| Muscle endurance | Upper body muscular endurance will be assessed using a modified push up test for repetitions | 12 months |
| Lean body mass accrual | Assessed by dual energy x-ray absorptiometry (DEXA) scan of the whole body | 12 months |
| Bone mineral density | Assessed by DEXA scan of the lumbar spine and whole body | 12 months |
| Resting energy expenditure | Indirect calorimetry will be performed after overnight fast | 12 months |
| D007018 | Hypopituitarism |
| D010900 | Pituitary Diseases |
| D007027 | Hypothalamic Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D004700 | Endocrine System Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D006730 |
| Hormones, Hormone Substitutes, and Hormone Antagonists |
| D010455 | Peptides |
| D000602 | Amino Acids, Peptides, and Proteins |