E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Polyarticular-course juvenile idiopathic arthritis |
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E.1.1.1 | Medical condition in easily understood language |
Childhood arthritis in several joints |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Musculoskeletal Diseases [C05] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10059176 |
E.1.2 | Term | Juvenile idiopathic arthritis |
E.1.2 | System Organ Class | 10028395 - Musculoskeletal and connective tissue disorders |
|
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 long term safety of tocilizumab treatment in patients with pcJIA from Germany who entered this extension. |
|
E.2.2 | Secondary objectives of the trial |
To investigate the long term efficacy during treatment with tocilizumab in patients with pcJIA from Germany who entered this extension study. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Patients– age 9 to 18- who completed visit 33 (week 104) of WA19977 study with at least JIA ACR30 clinical response to tocilizumab relative to baseline in WA19977 study with no AEs, SAEs or conditions that lead to unacceptable risk of continued treatment.
2. Patients should be scheduled to receive the first tocilizumab infusion in this study between 4 and 6 weeks after the last IV infusion in the core study (WA19977).
3. Written informed consent for study participation obtained from:
- Patient if the patient is 18 years or older
- Parents or legal guardian, with assent as appropriate by the patient, depending on the level of the patients understanding for patients that are less than 18 years old
In addition, written consent for data protection (legal requirement in Germany: datenschutz-rechtliche Einwilligung) must be provided.
4. Patient and parents or legal guardian able and willing to comply with the requirements of the extension study protocol.
5. If female and of child-bearing potential, the patient must have a negative urine pregnancy test at day 0 before infusion takes place. |
|
E.4 | Principal exclusion criteria |
1. According to investigator judgment patient did not satisfactory benefit from tocilizumab therapy within WA19977.
2. Treatment with any investigational agent since the last administration of study drug in the core study WA19977 or patients who participate currently in another clinical trial except WA19977.
3. Patient developed any other auto-immune, rheumatic disease or overlap syndrome other than the permitted polyarticular-course JIA subsets: Rheumatoid Factor Positive polyarticular JIA, Rheumatoid Factor Negative polyarticular JIA, and extended oligoarticular JIA. The excluded illnesses include but are not limited to systemic juvenile idiopathic arthritis, Lyme disease, enthesitis-related arthritis, psoriatic arthritis, Reiter´s syndrome, systemic lupus erythematosus, infectious or reactive arthritis or parvovirus infections.
4. Lack of peripheral venous access or unwilling to undergo multiple venopunctures.
5. Lack of patient´s or parents cooperation.
6. Any other reasons which in investigator´s opinion limit the possibility of patient´s participation in the study. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Safety
Number and percentage of AEs, SAEs, AE of Special Interest, Study Drug Related AEs |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
Efficacy
Proportion of patients with JIA ACR30/50/70/90
Proportion of patients with inactive disease at visits |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Screen/BL; every 12 weeks |
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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 | No |
E.6.5 | Efficacy | No |
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 | No |
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.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 3 |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
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
| |
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |