E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Active enthesitis-related arthrtitis as a category of juvenile idiopathic arthritis (ERA-JIA) as determined by International League of Associations for Rheumatology (ILAR) criteria. |
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E.1.1.1 | Medical condition in easily understood language |
Active enthesitis-related arthrtitis is a category of juvenile idiopathic arthritis (ERA-JIA) |
Die Enthesitis-assoziierte Arthritis (ERA) ist ein Subtyp der juvenilen idiopathischen Arthritis. |
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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 | 100000004859 |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
This study is intended to generate first evidence that treatment with etanercept is safe and effective in patients diagnosed with ERA-JIA who are able to aquire stable remission (inactive disease). |
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E.2.2 | Secondary objectives of the trial |
To evaluate the stability of remission off medication in patients treated with etanercept. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Parents / legal guardian and patient are willing to participate in the study and signed voluntarily the Informed Consent form.
Parents / legal guardian are willing to actively supervise storage and administration of study drug and to ensure that the time of each dose is accurately recorded in the subject's diary.
Patient and parents / legal guardian agree to comply with study requirements and are able to be at the clinic for all required study visits.
Patient is at least 6 years old and has not reached his 18th birthday.
Patient is currently not treated with a disease-modifying antirheumatic drug (DMARD) or if the patient is treated with sulfasalazine and treatment is planned to be continued throughout the study period, stable dose of sulfasalazin has been given for at least 4 weeks. If patient has been treated with other DMARD (Methotrexate, Leflunomide, Azathiopine, Hydroxychloroquine, Chloroquine …) treatment has be discontinued at least 28 days before baseline. Stable dosage of NSAIDs or at least 4 weeks before baseline, stable dosage of corticosteroids (≤ 0.2 mg of prednisone per kilogram per day, with a maximum of 10 mg per day) for at least 4 weeks before baseline, or both are permitted.
IN FEMALE PATIENT IN WHOM MENARCHE HAS OCCURRED
• Negative serum pregnancy test prior to administration of study medication.
• Willingness to use an adequate method of contraception
Adequate contraception can include abstinence if the investigator deems appropriate.
Diagnosis of active ERA-JIA as determined by International League of Associations for Rheumatology (ILAR) criteria. The activity of the disease is judged with
(I) a minimum of 3 active joints with either swelling not due to deformity or if no
swelling is present with limiting of motion and pain or pain on movement,
(II) a least a score of 3 of 10 for global assessment of the severity of disease by the physician
(III) a least a score of 3 of 10 for global assessment of overall well-being by the
patient or parent
Patient have to meet all criteria for eligibility for treatment with etanercept according to SPC and local guidelines, with expection of the requirement of a minimum of five affected joints.
Either the subject or an available adult must be capable (according to the investigator´s judgment of reconstituting and administering injections of SC etanercept.
Patient must be evaluated for active or latent TB infection according to the instructions of the protocol. If applicable: Guidelines regarding the treatment of latent TB must be followed prior to the administration of study medication.
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E.4 | Principal exclusion criteria |
Chronic or active infection or any major episode of infection requiring hospitalization or treatment with intravenous antibiotics within 30 days prior to study screening
Any preceding diagnosis of malignancy.
Pregnant or breast feeding female.
Female not willing to use appropriate contraception or sexual abstinence.
Active gastrointestinal disease (e.g., inflammatory bowel disease)
Significant blood clotting defect
Preceding diagnosis of tuberculosis or any opportunistic infection including herpes zoster at any time.
Patient has a history of any chronic disease other than JIA, especially chronic renal disease, liver disease, hematological, gastrointestinal, pulmonary, cardiological or neurological disease, which in the opinion of the investigator may influence the efficacy or safety of the study medication or which in the opinion of the investigator leads to an unacceptable risk for the patient if he participates in the study.
Patient had a significant illness during a period of 4 weeks prior to the first administration of study medication other than JIA-related.
Patient is abusing alcohol or drugs.
AST (SGOT), ALT (SGPT), or BUN levels more than two times the upper level of normal, creatinine levels more than 1.5 mg/dl, or any other laboratory abnormality considered to be clinically significant within 28 days prior to baseline
Received any investigational medication within 30 days prior to the first dose of study medication or scheduled to receive an investigational drug (other than the study medications) during the course of the study
Patient plans to change dosing of oral corticosteroids within the study period.
Patient has received i.v., i.m., i.a. or soft tissue injections of corticosteroids within 4 weeks before first administration of study medication.
Patient has previously been admitted to this study.
Patient has been treated with any other investigational agent within 30 days or 5 half-lifes of the agent, whichever is longer, prior to the screening evaluation.
HIV infected
Known past or current hepatitis infection
Patient has a history of an expanding CNS neoplasm, active CNS infection, demyelinating disease, degenerative neurological disease or any progressive CNS disease.
Patient has a poorly controlled diabetes.
Received a live virus vaccine within 1 month prior to baseline
Any concurrent medical condition which would, in the investigator's opinion, compromise the patient's ability to tolerate the study drug or would make the patient unable to comply with the protocol.
Patient has a history of or current psychiatric illness that would interfere with ability to comply with protocol requirements or give informed consent.
Patient has a recent history of alcohol or drug abuse within the past 6 months that would interfere with ability to comply with protocol requirements.
Any other inability to comply with the study requirements.
Any contraindication listed in the German 'Fachinformation' of the drug Enbrel®.
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E.5 End points |
E.5.1 | Primary end point(s) |
inactive disease of ERA-JIA defined as
• No active synovitis
• No fever, rash, serositis, splenomegaly, or generalized
lymphadenopathy attributable to JIA
• No active uveitis
• Normal CRP
• Physician’s global assessment of disease activity indicates no
active disease
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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 |
withdrawal design; open label treatment phase,12 weeks before controlled double blind phase |
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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.8.2.4 | Number of treatment arms in the trial | 2 |
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 | 12 |
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
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last visit after 70 days after adminitration 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 | 3 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 0 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |