This study is intended to determine whether twice daily weight based dosing with recombinant
human insulin-like growth factor (rhIGF-1) will safely and effectively increase the growth
of prepubertal children with short stature associated with low IGF-1 levels but who produce
sufficient growth hormone (GH). Subjects will be randomized to either an observation arm or
to active treatment.
Prepubertal growth failure associated with primary IGF-1 deficiency (IGFD). Primary IGFD is
a term that has been used to describe patients with intrinsic cellular defects in GH action.
In this protocol, primary IGFD is defined as short stature (height standard deviation
score[SDS]<-2 below the mean for age and gender), low serum IGF-1 (IGF-1 SDS <-2 below the
mean for age and gender), and levels of growth hormone (GH) that are normal (≥7ng/mL) after
a GH stimulation test. Primary IGFD is believed to result from a lower than normal
ability to produce IGF-1 when exposed to normal levels of GH, i.e., a type of GH
insensitivity or GH resistance.
This trial is one year, randomized, open label, observation-controlled, parallel-dose
comparison efficacy and safety study conducted in approximately 40 centers across the United
- Chronological age ≥ 3 and chronological or bone age less than or equal to 11 years
inclusive in girls;
- Chronological age ≥ 3 and chronological or bone age less than or equal to 12 years
inclusive for boys
- Height SD score of < -2
- IGF-1 SD score of < -2
- Prior treatment with rhGH, rhIGF-1, or other growth-influencing medications
- Growth failure associated with other identifiable causes (e.g., syndromes,
- Chronic illness such as diabetes, cystic fibrosis, etc.