To demonstrate that an individualized, formula-based Genotropin regimen for children with Idiopathic Short Stature will lead to a targeted height gain (to reach the target of 10th percentile (%), or -1.3 SDS) during 24 months of treatment. The endpoint at 4 years is to explore treatment efficiency over four years of two formula-based dose regimens (sub-arms) compared to standard treatment
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
316
Compare daily injections of formula-based HGH treatment to daily injections of standard HGH treatment in subjects with Idiopathic Short Stature over 24 months period followed by an exploratory 24 months period.
Compare daily injections of formula-based HGH treatment to daily injections of standard HGH treatment in subjects with Idiopathic Short Stature over 24 months period followed by an exploratory 24 months period.
Absolute On-target Difference (AOTD) at 24 Months
This was defined as an absolute difference between the 24-month height standard deviation score (SDS) and targeted 24-month height SDS (10th percentile (%), or -1.3 SDS). SDS indicates how similar the participant was to the reference population. These were calculated using 2000 Center for the Disease Control (CDC) growth reference tables (by age and gender).
Time frame: 2 years
Variability of Height SDS at 24 Months
The continuous endpoint of variability of height SDS at 24 months was defined as the SD of the 24 month height SDS.
Time frame: 2 years
Time Cost (Months Until >= -2 SDS)
Time cost was defined as the number of months needed until height SDS was within the normal limit (ie, \>= -2SDS).
Time frame: 2 years
Computed Cost of Height Gain at 48 Months
The computed cost of height gain was defined as the amount of drug used relative to the observed height-gain, in terms of mg/cm, this was calculated at Month 48.
Time frame: 4 years
Estimated Cost of Height Gain Estimated Until Full Adult Height (FAH) at 48 Months
The estimated cost of long-term height gain until FAH was calculated.
Time frame: 4 years
Change From Baseline in Height SDS at 48 Months.
Change in height SDS was measured at 48 months.
Time frame: 4 years
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