E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Juvenile ankylosing spondylitis |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 10 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10002556 |
E.1.2 | Term | <Manually entered code. Term in E.1.1> |
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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 |
The study is designed to evaluate the safety and efficacy of treatment of juvenile ankylosing spondylitis (JAS) with adalimumab. The primary objectives are • to demonstrate the superiority of adalimumab with respect to the ASAS Working Group response criterion ASAS40 as compared to placebo • to contrast the safety profile of adalimumab with placebo in subjects with JAS.
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E.2.2 | Secondary objectives of the trial |
Secondary objectives of the trial are • to show improvement of each of the 4 categories contributing to the ASAS40 criterion 1. Spinal inflammation 2. Back pain (total and nocturnal) 3. Patient's global assessment 4. Physical function • to show superiority of adalimumab with respect to the ACR Ped 30 response criterion as compared to placebo • to show improvement of each of the 6 categories contributing to the ACR Ped Score 1. Physician's global assessment of subject's disease activity 2. Parent's global assessment of subject's overall well-being 3. Number of active joints 4. Number of joints with limitation of motion 5. Measure of physical function (in this study the Childhood Health Assessment Questionnaire CHAQ will be used) 6. Laboratory measure of inflammation (in this study the CRP will be used). |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
IN1: Parents / legal guardian and patient are willing to participate in the study and signed voluntarily the Informed Consent form.
IN4: Patient is at least 12 years old and has not reached his 18th birthday.
IN5: The weight of the patient is > 30 kg.
IN7: Patient has a diagnosis of juvenile ankylosing spondylitis, i.e. A bilateral active sacroiliitis confirmed by magnetic resonance imaging is present OR B unilateral active sacroiliitis confirmed by magnetic resonance imaging is present, and patient has active peripheral joint disease restricted to the lower extremities AND pain or history of pain at the dorsolumbar junction of the lumbar spine.
IN8: Patient has active disease, i.e. 1. spinal inflammation score of at least 3 AND at least 2 of the following domains: 2. back pain score of at least 3 3. patient global assessment of disease activity of at least 3 4. physical function score of at least 3.
IN9:The disease has been refractory to at least two different non-steroidal antiphlogistic drugs given at appropriate dosage and for appropriate period of time. |
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E.4 | Principal exclusion criteria |
EX1: Patient has been diagnosed to have systemic onset of JRA / JIA or has active systemic features including fever or rash.
EX2: Patient has active uveitis within a period of 4 weeks prior to the first administration of study medication.
EX7: Preceding diagnosis of tuberculosis or any opportunistic infection including herpes zoster at any time.
EX10: Patient had a significant illness during a period of 4 weeks prior to the first administration of study medication other than JAS-related.
EX27: Any contraindication listed in the German 'Fachinformation' of the drug Humira®. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Response rates according to the ASAS40 criteria at Week 12. A responder is defined as a patient who achieved improvement in at least 3 of 4 domains contributing to the ASAS, with no worsening in the remaining domain: 1. Spinal inflammation 2. Back pain 3. Patient's global assessment 4. Physical function. |
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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 | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | Yes |
E.8.1.7.1 | Other trial design description |
Open-label follow-up treatment phase of further 12 weeks |
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E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
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 | 9 |
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 | Information not present in EudraCT |
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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Last visit plus 70 days after administration for adverse event collection. |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 1 |
E.8.9.1 | In the Member State concerned months | 6 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 6 |
E.8.9.2 | In all countries concerned by the trial days | 0 |