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 ]

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).


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