Systemic Juvenile Idiopathic Arthritis (SJIA) MedDRA version: 8.1 Level: PT Classification code 10059177 Term: Juvenile arthritis
Conditions
Interventions
Product Name: ACZ885
Product Code: ACZ885
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: ACZ885 Drug Substance
Current Sponsor code: ACZ885
Other descriptive n
Sponsors
Novartis Pharma Services AG
Eligibility
Sex/Gender
All
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 and has active disease defined as active arthritis (using ACR definition of active joint) in at least one joint for the last 6 weeks (does not have to be the same joint) and within 2 weeks of study entry at least one of the following systemic features considered by the treating physician to be due to SJIA: spiking, intermittent fever (body temperature > 38.9°C only for several hours during the day), and CRP > 50 mg/L (normal range =65 years) no F.1.3.1 Number of subjects for this age 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 - 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 < 2.5 g/L, cytopenia, hypertriglyceridemia, decreased platelet count, increased aspartate transaminase, hyperferritinemia). - Familial and social conditions rendering regular medical assessment not possible.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: • To evaluate the safety, tolerability and immunogenicity of sc administration of ACZ885 in pediatric subjects with active Systemic Juvenile Idiopathic Arthritis (SJIA). • To assess the initial efficacy profile (% responders to treatment and time to relapse) of sc dosing of ACZ885 in pediatric subjects with active SJIA according to the modified American College of Rheumatology (ACR) pediatric 30 definitions and the ability of ACZ885 to control the systemic manifestations of SJIA such as fever and rash. • To assess the PK of ACZ885 administered sc in children. • To assess PK and PD relationships in order to derive a dose and dosing regimen for Phase III (dose and dosing frequency required for relief of signs and symptoms and response according to at least ACR 30 definition).;Secondary Objective: • To assess the proportion of patients with inactive disease at each dose level. • To investigate the possibility of corticosteroid tapering. • To establish a biomarker and pharmacogenomic characterization of these patients at baseline and to evaluate the treatment response to ACZ885.;Primary end point(s): See E.2.1 | — |
Countries
France, Italy, United Kingdom
Outcome results
None listed