systemic juvenile idiopathic arthritis MedDRA version: 20.0 Level: LLT Classification code 10079454 Term: Systemic juvenile idiopathic arthritis System Organ Class: 100000004859
Conditions
Interventions
Sponsors
Asklepios Klink Sankt Augustin
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Parents / legal guardian and patient are willing to participate in the study and signed voluntarily the Informed Consent form. ? I 2 Patient and parents / legal guardian agree to comply with study requirements and are able to be at the clinic for all required study visits. ? I 3 Patient is at least 2 years old and has not reached his 18th birthday. ? I 4 Patient is currently not treated with a disease-modifying antirheumatic drug (DMARD). Patient has not been treated with synthetic DMARDs (such as Methotrexate, Leflunomide, Azathioprine, Hydroxychloroquine, Chloroquine …) or biologic DMARDs (such as Anakinra, Canakinumab, Tocilizumab, Etanercept, Adalimumab, Golimumab …) before baseline. ? I 5 Stable dosage of NSAIDs ? I 6 Patient is currently not treated with corticosteroids. Patient has previously not been treated with corticosteroids for more than 7 days after diagnosis. ? I 7 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. ? I 8 Diagnosis of active systemic onset JIA as determined by International League of Associations for Rheumatology (ILAR) criteria (Petty RE, Southwood TR, Manners P, Baum J, Glass DN, Goldenberg J, et al. J Rheumatol. 2004;31:390–392) or the Yamaguchi’s criteria for Still’disease ( Yamaguchi M. et al., J Rheumatol. 19:424-30, 1992) with confirmed diagnosis of SJIA as per ILAR definition: Arthritis in one or more joints with or preceded by fever of at least 2 weeks duration that is documented to be daily/ quotidian for at least 3 days and accompanied by one or more of the following: · Evanescent nonfixed erythematous rash, · Generalized lymph node enlargement, · Hepatomegaly and/ or splenomegaly, · Serositis or confirmed diagnosis of Still’s disease as per Yamaguchi’s criteria: Five or more criteria, of whom two or more must be major Major criteria ? Fever >39 °C, lasting 1 week or longer ? Arthralgia or arthritis, lasting 2 weeks or longer ? Typical rash ? Leukocytosis >10,000/mm3 with >80% polymorphonuclear cells Minor criteria ? Sore throat ? Recent development of significant lymphadenopathy ? Hepatomegaly or splenomegaly ? Abnormal liver function tests ? Negative tests for antinuclear antibody (IF) and rheumatoid factor (IgM) Exclusion criteria ? Infections ? Malignancies (mainly malignant lymphoma) ? Other rheumatic disease (mainly systemic vasculitides) The activity of the disease is judged with (I) active arthritis 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 (IV) active systemic signs of the disease for at least 3 days before baseline with either fever of more than 38.5°C, and/or pericarditis and/or rash. ? I 9 Patient have to meet all criteria for eligibility for treatment with Canakinumab according to SPC and local guidelines, with exception of the requirement of a minimum of five affected joints. ? I 10 Either the subject or an available adult must be capable (according to the investigator´s judgment of reconstitu
Exclusion criteria
Exclusion criteria: E 1 Chronic or active infection or any major episode of infection requiring hospitalization or treatment with intravenous antibiotics within 30 days prior to study screening E 2 Any preceding diagnosis of malignancy. E 3 Pregnant or breast feeding female. E 4 Female not willing to use appropriate contraception or sexual abstinence. E 5 Active gastrointestinal disease (e.g., inflammatory bowel disease) E 6 Significant blood clotting defect E 7 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. E 8 Patient had a significant illness during a period of 4 weeks prior to the first administration of study medication other than JIA-related. E 9 Previous use of systemic corticosteroids for more than 7 days (>0.2 mg/kg bw) E10 Previous use of immunosuppressants (such as methotrexate, cyclosporine) E11 Previous use of biologic agents (such as Anakinra, Canakinumab, Tocilizumab) E12 Active macrophage activation syndrome with biologic features of MAS [such as hemorrhages, central nervous system dysfunction, plasma fibrinogen level < 2.5 g/L, cytopenia, hypertriglyceridemia, decreased platelet count, hyperferritinemia at screening or a history of recurrent pericarditis, myocarditis, serositis. E 13 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 E 14 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 E 15 Patient has previously been admitted to this study. E 16 Patients is known to be HIV-infected E 17 Known past or current hepatitis infection E 18 Patient has a history of an expanding CNS neoplasm, active CNS infection, demyelinating disease, degenerative neurological disease or any progressive CNS disease. E 19 Patient has a poorly controlled diabetes. E 20 Received a live virus vaccine within 1 month prior to baseline E 21 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. E 22 Patient has a history of or current psychiatric illness that would interfere with ability to comply with protocol requirements or give informed consent. E 23 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. E 24 Any contraindication listed in the German 'Fachinformation' of the drug Ilaris ®
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To demonstrate high rate of response to monotherapy with Canakinumab in newly diagnosed sJIA/Still’s disease patients not treated with corticosteroids. ;Secondary Objective: Steroid free remission at month 6 defined as no fever>38.0°C, no arthritis defined as joint swelling or pain on motion and limited range of motion, normal CRP, no organ involvement attributed to sJIA/Still’s disease. To demonstrate improvement by validated outcome criteria (JADAS, ACR-Response, ACR-Remission-criteria. To demonstrate normal growth. To demonstrate safety of Canakinumab injection in sJIA / juvenile Still’s disease patients not treated with corticosteroids. To evaluate number of patients who have reached a steroid dose of 0 at month 6. To explore the time to inactive disease. To validate biomarkers for early diagnosis of Still’s disease To establish new panel diagnostics To monitor immune activation during follow-up. ;Primary end point(s): High Response rates with a high percentage of children reaching steroid free inactive disease at month 3 and medication free Remission at month 12;Timepoint(s) of evaluation of this end point: Month3, Month 12 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): High percentage of patients with steroid free remission at month 6 defined as no fever>38.0°C, no arthritis defined as joint swelling or pain on motion and limited range of motion, normal CRP, no organ involvement attributed to sJIA / juvenile Still’s disease. High percentage of improvement by validated outcome criteria (JADAS, ACR-Response, ACR-Remission-criteria throughout the study). Normal growth rates (relation of length SDS baseline to length SDS at month 6 and month 12) acceptable safety of canakinumab injection in sJIA/still’s disease patients not treated with corticosteroids. High number of patients who have reached a steroid dose of 0 at month 6 and month 12. • To validate biomarkers for early diagnosis of sJIA / juvenile Still’s disease. • To establish new panel diagnostics to monitor immune activation during follow-up ;Timepoint(s) of evaluation of this end point: Months 3,6,12 | — |
Countries
Germany
Contacts
Public ContactAriane Klein
Asklepios Klinik Sankt Augustin
Outcome results
None listed