Clinical Trial: Individualized, Target-Driven Treatment Of Children With Idiopathic Short Stature
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: A Four Year Open Label Multi Center Randomized Two Arm Study Of Genotropin In Idiopathic Short Stature Patients: Comparing An Individualized, Target Driven Treatment Regim
Brief Summary: 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
Detailed Summary:
Sponsor: Pfizer
Current Primary Outcome: Absolute On-target Difference (AOTD) at 24 Months [ Time Frame: 2 years ]
Original Primary Outcome: 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
Current Secondary Outcome:
- Variability of Height SDS at 24 Months [ Time Frame: 2 years ]The continuous endpoint of variability of height SDS at 24 months was defined as the SD of the 24 month height SDS.
- Time Cost (Months Until >= -2 SDS) [ Time Frame: 2 years ]Time cost was defined as the number of months needed until height SDS was within the normal limit (ie, >= -2SDS).
- Computed Cost of Height Gain at 48 Months [ Time Frame: 4 years ]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.
- Estimated Cost of Height Gain Estimated Until Full Adult Height (FAH) at 48 Months [ Time Frame: 4 years ]The estimated cost of long-term height gain until FAH was calculated.
- Change From Baseline in Height SDS at 48 Months. [ Time Frame: 4 years ]Change in height SDS was measured at 48 months.
Original Secondary Outcome:
- More uniformly achieve the desired height gain at 24 months with the goal of minimizing the variability between subjects.
- Demonstrate safety of Genotropin treatment with this treatment paradigm
- Identify pharmacogenomic variants related to growth/stature
- Explore treatment efficiency over four years of two formula-based dose regimens (sub-arms) compared to standard treatment
- Explore maintained treatment efficacy at four years of two formula-based dose regimens (sub-arms) compared to standard treatment
Information By: Pfizer
Dates:
Date Received: November 2, 2006
Date Started: December 2006
Date Completion:
Last Updated: March 9, 2016
Last Verified: March 2016