Individualized, Target-Driven Treatment Of Children With Idiopathic Short Stature

Study Overview
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Brief Summary
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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

Official Title
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A Four Year Open Label Multi Center Randomized Two Arm Study Of Genotropin In Idiopathic Short Stature Patients: Comparing An Individualized, Target Driven Treatment Regimen To Standard Dosing Of Genotropin

Conditions
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Idiopathic Short Stature

Intervention / Treatment
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* Drug: Genotropin
* Drug: Genotropin


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Prepubertal children with bone ages between 3 and 10 years of age for males and 3 and 9 years of age for females Naive to Growth Hormone treatment Exclusion Criteria: Abnormal karyotype. Small Gestational Age and Skeletal dysplasia.

Ages Eligible for Study
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Minimum Age: 3 Years
Maximum Age: 10 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: Standard<br>Standard daily HGH treatment | Drug: Genotropin<br>* 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.<br>|
| Active Comparator: Formula-based<br>Formula-based dose regimen | Drug: Genotropin<br>* 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.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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). | 2 years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. | 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). | 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. | 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. | 4 years | 
| Change From Baseline in Height SDS at 48 Months. | Change in height SDS was measured at 48 months. | 4 years | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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ISS, human growth hormone