E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Juvenile idiopathic arthritis (JIA) |
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E.1.1.1 | Medical condition in easily understood language |
Joint inflammation in children |
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E.1.1.2 | Therapeutic area | Diseases [C] - Musculoskeletal Diseases [C05] |
MedDRA Classification |
E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To evaluate the effect of increasing the Human growth hormone (hGH) dose (0.6 versus 0.46 milligram per kilogram per week [mg/kg/week ] or 1.8 versus 1.4 international unit per kilogram per week [IU/kg/week]) on the statural response in subjects initially in the treated group of Study CTN 97-8129-016, by comparing the statural response observed with that obtained during the first therapeutic phase at a dose of 0.46 mg/kg/week, or 1.4 IU/kg/week
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E.2.2 | Secondary objectives of the trial |
To assess the value of early treatment during the course of arthritic disease by comparing
changes to the body composition and bone mineralization in the 2 groups of children.
To assess safety by monitoring of arthritic disease (clinical) and glucose tolerance
(biological).
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. All subjects had to be previously included in Study CTN 97-8129-016 to continue somatropin treatment until FH was reached.
2. All subjects should have received corticosteroid therapy, whatever the dose or the method of administration.
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E.4 | Principal exclusion criteria |
1. Discontinuation of corticosteroid therapy for more than a year during Study CTN 97-8129-016.
2. Concomitant severe, chronic disease: renal failure (glomerular filtration less than or equal to [<=] 50 mililiter per min per meter [ml/mn/1m] ^2 73), nephrotic syndrome, hepatic failure, diabetes.
3. Endocrine disorder except for adequately compensated hypothyroidism.
4. Subjects withdrawing from the study prematurely.
5. Non-compliant subjects.
6. Inclusion in another protocol.
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E.5 End points |
E.5.1 | Primary end point(s) |
1) Change From Baseline in Annual Rate of Growth Standard Deviation Score (SDS) at Year 3
2) Change From Baseline in Annual Rate of Growth Standard Deviation Score (SDS) at Final Height
3) Change From Baseline in Height Standard Deviation Score (SDS) at Year 3
4) Change From Baseline in Height Standard Deviation Score (SDS) at Final Height
5) Change From Baseline in Predicted Height Standard Deviation Score (SDS) at Year 3
6) Change From Baseline in Predicted Height Standard Deviation Score (SDS) at Final Height
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
1) Baseline, Year 3
2) Baseline, final height (assessed up to Year 9.5)
3) Baseline, Year 3
4) Baseline, final height (assessed up to Year 9.5)
5) Baseline, Year 3
6) Baseline, final height (assessed up to Year 9.5)
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E.5.2 | Secondary end point(s) |
1) 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
2) Change From Baseline in Lean Mass and Fat Mass at Year 1, 2, 3, 4, 5, 6, 7, 8 and 9
3) Change From Baseline in Bone Mineralization at Year 1, 2, 3, 4, 5, 6, 7, 8 and 9
4) 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
5) 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
6) 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
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
1) 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
2) Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9
3) Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9
4) 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
5) 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
6)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
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 2 |
E.8.3 |
Will this trial be conducted at a single site globally?
| Yes |
E.8.4 | Will this trial be conducted at multiple sites globally? | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | Specify the countries outside of the EEA in which trial sites are planned |
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E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.2 | In all countries concerned by the trial years | 9 |
E.8.9.2 | In all countries concerned by the trial months | 7 |
E.8.9.2 | In all countries concerned by the trial days | 3 |