Joint inflammation with children Systemic Juvenile Idiopathic Arthritis (SJIA)
Conditions
Interventions
Run-in-period: maximum of 72 hours, for patients who will stop treatment with
anakinra
Treatment period: Anti-IL-1&beta
Monoclonal Antibody (ACZ885) given subcutaneously
Stage I
Patient treatment on mg/kg basis. Repeated single dose escalation phase in 3
cohorts.
Cohort I (n=6) : starting dose 0.5 mg/kg ACZ885 s.c.,
Sponsors
Novartis
Eligibility
Age
2 Years to 99 Years
Inclusion criteria
Inclusion criteria: - Male and female subjects aged 4 to 20 years at the time of the screening visit, having passed screening examinations. Parents* or legal guardian*s written informed consent (patient*s informed consent for >= 18 years of age) and child*s assent, if appropriate, are required prior to study participation. - Female subjects of child-bearing potential may participate if they have a negative serum pregnancy test at screening and prior to dosing, and are willing to practice double-barrier contraception during the study (from the date of screening) and for at least 3 months following the last dose. - Patient meets the diagnostic criteria for SJIA, has at least 6 months disease duration and has active disease defined at the time of enrollment defined as follows: At least 2 joints with active arthritis (using ACR definition of active joint) / spiking, intermittent fever (body temperature > 38.9°C only for several hours during the day) / CRP > 50 mg/L (normal range
Exclusion criteria
Exclusion criteria: - Etanercept in the four weeks prior to the Baseline visit. - Adalimumab in the eight weeks prior to the Baseline visit. - Infliximab in the eight weeks prior to the Baseline visit. - Any other investigational biologics in the eight weeks prior to the Baseline visit. - Leflunomide in the four weeks prior to the Baseline visit. Documentation of a completion of a full cholestyramine elimination procedure after most recent leflunomide use will be required. - Thalidomide, Growth hormone , Cyclosporine, in the four weeks prior to the Baseline visit. - Sulfasalazine or hydroxychloroquine in the eight weeks prior to the Baseline visit. - i.v. immunoglobulin (i.v. Ig) in the eight weeks prior to the Baseline visit. - 6-Merceptopurine, azathioprine, cyclophosphamide, or chlorambucil, in the 24 weeks prior to the Baseline visit. - History of recurrent bacterial, fungal or viral infection. - Evidence of currently active bacterial, fungal or viral infection. - Administration of live attenuated vaccine. - Uncontrolled severe systemic symptoms and/or biologic features of Macrophage Activation Syndrome (hemorrhages, central nervous system dysfunction, hepatomegaly, serum fibrinogen level
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Efficacy: response assessment pre-dose on day 1, at each further clinical visit until a clear relapse and at study completion. Response variables: visual analogue scale (VAS), functional ability: Childhood Health Assessment Questionnaire (CHAQ), number of joints with active arthritis (ACR), number of joints with limitation of motion, CRP, fever, parent*s assessment of patient*s pain scale, patient*s daily diary. Safety and tolerability: All patients who received at least one treatment will be evaluated by treatment group (dose level) on vital signs, ECG, hematology, blood chemistry, urine, (serious) adverse events, tolerability (pain, redness, swelling, induration, hemorrhage and itching), infection, concomitant medication / significant non drug therapies. PK and PD data-analysis of all completed patients. Optional: Pharmacogenomic data-analysis: mRNA analysis (performed only on patients who gave their specific consent). | — |
Secondary
| Measure | Time frame |
|---|---|
| N.a.p. | — |
Countries
Netherlands
Outcome results
None listed