rhGH and rhIGF-1 Combination Therapy in Children With Short Stature Associated With IGF-1 Deficiency
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About
IGF-1 (insulin-like growth factor-1) is a hormone that is normally produced in the body in response to another hormone called growth hormone. Growth Hormone is produced by a small gland at the base of the brain (the pituitary). Together IGF-1 and GH are large contributors to growth during infancy, childhood, and adolescence.
Children with IGF Deficiency are short and have an imbalance in the levels of growth hormone and IGF-1 that their body produces. Their growth hormone levels are normal or even high, but IGF-1 levels do not increase normally in response to growth hormone. As a result, they have a type of growth hormone insensitivity and an inability to grow normally.
This study is a test to see whether daily dosing with a combination of rhIGF-1 and rhGH will help children with IGFD grow taller more quickly than children treated with rhGH alone. The study medications, rhIGF-1 and rhGH, are approved by the US Food and Drug Administration (FDA) for use in some growth disorders in children, but the combination of rhIGF-1 and rhGH in children with IGF-1 deficiency (IGFD) is investigational.
Enrollment
Sex
Ages
Volunteers
Inclusion criteria
- Parents or legally authorized representatives must give signed informed consent before any trial-related activities
- IGF-1 SDS of ≤ -1 for age and gender
- Short stature, as defined by a height SDS of ≤ -2 for age and gender
- Chronological age ≥ 5 years
- Bone age ≤ 11 years in boys and ≤ 9 years in girls
- GH sufficiency, defined as a maximal stimulated GH response of greater than or equal to 10 ng/mL at Visit 2 (note: upon approval of the Medical Monitor, the result of a prior GH stimulation test may satisfy this requirement).
- Prepubertal status
- Adequate nutrition as evidenced by a body mass index (BMI) greater than or equal to the 5th percentile for age and gender
Exclusion criteria
- Severe Primary IGFD (defined as height and IGF-1 SDS ≤ 3, and stimulated GH response greater than or equal to 10 ng/mL)
- Prior or current use of medications with the potential to alter growth patterns including GH, IGF-1, IGFBP-3, gonadotrophin agonists (e.g., Lupron), aromatase inhibitors, androgens and estrogens
- Known or suspected allergy to rhGH, rhIGF-1 or a constituent of their formulations
- Current use of medications for attention deficit disorder
- A chronic health condition that requires anti-inflammatory steroids or daily medication unless approved by the Medical Monitor
Trial design
Primary purpose
Allocation
Interventional model
Masking
106 participants in 4 patient groups
Trial contacts and locations
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Data sourced from clinicaltrials.gov
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