Recombinant Human Growth Hormone (rhGH) and Recombinant Human Insulin-like Growth Factor-1 rhIGF-1) Combination Therapy in Children With Short Stature Associated With IGF-1 Deficiency: A Six-year, Randomized, Multi-center, Open-label, Parallel-group, Active Treatment Controlled, Dose Selection Trial

MS316

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.

2007-12-01

2010-04-01

2012-03-01

Terminated

Early phase I

106

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

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2023-03-30