Prevention of Growth Retardation by Early Treatment With Growth Hormone (GH) in Children With CJA Treated by Corticosteroid Therapy

Study Overview
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Brief Summary
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To evaluate the effect of increasing the growth hormone dose on the statural response To assess the value of early treatment during the course of arthritic disease by comparing the height acquired in the medium term by children in the two groups: treated from the start, or 1 year to 15 months after the diagnosis of CJA, or treated for 4 years after the diagnosis

Detailed Description
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This trial terminated on 10-Jun-2011 due to prolonged issues with drug accountability and data collection discrepancies. The decision to terminate was not based on any safety concerns.

Official Title
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Prevention of Retarded Growth by Early Treatment With Recombinant Human Growth Factor Genotonorm (Registered) in Children With Systemic Forms of Chronic Juvenile Arthritis Receiving Long-term Corticosteroid Therapy. Extension of the Study Beyond Three Years

Conditions
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Endocrine System Diseases

Intervention / Treatment
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* Drug: Somatropin


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: This study will be proposed to all patients previously included in study CTN 97-8129-016 Exclusion Criteria: Discontinuation of corticosteroid therapy for more than a year during study CTN 97-8129-016 Patients withdrawing from the study prematurely

Ages Eligible for Study
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Minimum Age: 4 Years
Maximum Age: 14 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: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Somatropin<br> | Drug: Somatropin<br>* Liquid, daily to final height Maximum Dosage: 50 µg/kg/day<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change From Baseline in Annual Rate of Growth Standard Deviation Score (SDS) at Year 3 | Annual rate of growth SDS was obtained by measuring the annual growth rate, subtracting chronological age- and gender-appropriate mean annual growth rate and dividing the result by standard deviation of that mean (as obtained from chronological age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) participant's value was relative to the mean of the reference population. | Baseline, Year 3 | 
| Change From Baseline in Annual Rate of Growth Standard Deviation Score (SDS) at Final Height | Annual rate of growth SDS was obtained by measuring the annual growth rate, subtracting chronological age- and gender-appropriate mean annual growth rate and dividing the result by standard deviation of that mean (as obtained from chronological age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) participant's value was relative to the mean of the reference population. | Baseline, final height (assessed up to Year 9.5) | 
| Change From Baseline in Height Standard Deviation Score (SDS) at Year 3 | Height was measured using a wall mounted device (example, Harpenden stadiometer). Height SDS was obtained by measuring the height, subtracting chronological age- and gender-appropriate mean height and dividing the result by standard deviation of that mean (as obtained from chronological age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) participant's value was relative to the mean of the reference population. | Baseline, Year 3 | 
| Change From Baseline in Height Standard Deviation Score (SDS) at Final Height | Height was measured using a wall mounted device (example, Harpenden stadiometer). Height SDS was obtained by measuring the height, subtracting chronological age- and gender-appropriate mean height and dividing the result by standard deviation of that mean (as obtained from chronological age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) participant's value was relative to the mean of the reference population. | Baseline, final height (assessed up to Year 9.5) | 
| Change From Baseline in Predicted Height Standard Deviation Score (SDS) at Year 3 | Predicted height was calculated according to Greulich and Pyle using Bayley Pinneau method. Predicted height SDS was obtained by calculating the predicted height, subtracting chronological age- and gender-appropriate mean predicted height and dividing the result by standard deviation of that mean (as obtained from chronological age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) participant's value was relative to the mean of the reference population. | Baseline, Year 3 | 
| Change From Baseline in Predicted Height Standard Deviation Score (SDS) at Final Height | Predicted height was calculated according to Greulich and Pyle using Bayley Pinneau method. Predicted height SDS was obtained by calculating the predicted height, subtracting chronological age- and gender-appropriate mean predicted height and dividing the result by standard deviation of that mean (as obtained from chronological age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) participant's value was relative to the mean of the reference population. | Baseline, final height (assessed up to Year 9.5) | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change From Baseline in Insulin-like Growth Factor-1 (IGF-1) Concentration at Year 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 6.5, 7, 7.5, 8, 8.5 and 9 |  | Baseline, Year 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 6.5, 7, 7.5, 8, 8.5, 9 | 
| Change From Baseline in Insulin-like Growth Factor Binding Protein 3 (IGFBP3) Concentration at Year 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 6.5, 7, 7.5, 8, 8.5 and 9 |  | Baseline, Year 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 6.5, 7, 7.5, 8, 8.5, 9 | 
| Change From Baseline in Lean Mass and Fat Mass at Year 1, 2, 3, 4, 5, 6, 7, 8 and 9 | Lean mass and fat mass: measurements of body composition assessed using Dual Energy X-ray Absorptiometry (DEXA) scan. | Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9 | 
| Change From Baseline in Bone Mineralization at Year 1, 2, 3, 4, 5, 6, 7, 8 and 9 | Bone mineralization, an estimate of the amount of mineral (such as calcium) in the bone, was assessed using DEXA scan. | Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9 | 
| Change From Baseline in Weight Standard Deviation Score (SDS) at Year 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 6.5, 7, 7.5, 8, 8.5 and 9 | Body weight was measured using a balance scale. Weight SDS was obtained by measuring the weight, subtracting age- and gender- appropriate mean weight and dividing the result by standard deviation of that mean (as obtained from age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) participant's value was relative to the mean of the reference population. | Baseline, Year 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 6.5, 7, 7.5, 8, 8.5, 9 | 
| Change From Baseline in Corticosteroid Dose at 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 6.5, 7, 7.5, 8, 8.5 and 9 |  | Baseline, Year 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 6.5, 7, 7.5, 8, 8.5, 9 |