Clinical trial NCT02396212: Study of Efficacy and Safety of Canakinumab in Japanese Patients With SJIA
Official title: An Open-label, Single-arm, Active-treatment, Efficacy and Safety Study of Canakinumab (ACZ885) Administered for at Least 48 Weeks in Japanese Patients With Systemic Juvenile Idiopathic Arthritis (SJIA)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Systemic Juvenile Idiopathic Arthritis
Keywords: ACZ885
canakinumab
Juvenile Rheumatoid arthritis (JRA) chronic
systemic inflammatory disorder
painful joints
inflammation of the synovial membrane
auto-immune rheumatoid disease
reactive rheumatoid arthritis
Systemic Juvenile Rheumatoid arthritis (SJRA)
Japanese patients
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2015-03-24
Enrollment: 19
Sex: ALL
Minimum age (years): 2
Maximum age (years): 19
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This was a phase III study designed to provide efficacy and safety data for canakinumab administered for at least 48 weeks as subcutaneous (s.c.) injection every 4 weeks (q4wk) in Japanese patients with Systemic Juvenile Idiopathic Arthritis (SJIA). Interim analysis (IA) data at Week 28 and 48 from this study supported a registration submission of canakinumab in the indication of SJIA in Japan.


Eligibility criteria: Inclusion Criteria

- Confirmed diagnosis of SJIA as per International League Against Rheumatism (ILAR) definition (Petty, et al. 2004) that must have occurred at least 3 months prior to enrollment with an onset of disease \< 16 years of age: 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: Rash due to SJIA, lymphadenopathy, Hepatomegaly/Splenomegaly, Serositis

* Active disease at the time of baseline defined as follows:
* At least 2 joints with active arthritis
* Documented spiking, intermittent fever (body temperature \> 38°C) for at least 1 day during the screening epoch and within 1 week before first canakinumab dose
* C-Reactive Protein (CRP) \> 30 mg/L(3 mg/dL) (normal range \< 10 mg/L(1 mg/dL))
* Negative TB screen (Chest X-ray and T-SPOT test)

Exclusion Criteria:

* With active or recurrent bacterial, fungal or viral infection at the time of enrollment, including patients with evidence of Human Immunodeficiency Virus (HIV) infection, Hepatitis B and Hepatitis C infection. Patients with resolved/previous hepatitis B infection (a negative HBs antigen, but a positive anti-HBs antibody and/or anti-HBc antibody).
* With underlying metabolic, renal, hepatic, infectious or gastrointestinal conditions which in the opinion of the investigator immunocompromises the patient and /or places the patient at unacceptable risk for participation.
* With neutropenia (absolute neutrophil count \< 1500/mm3) at screening.

Primary outcomes:
measure: Percentage of Participants Who Achieved a Minimum Adapted American College of Rheumatology (ACR) Pediatric 30 Criteria
timeFrame: Week 8
measure: Percentage of Participants With Canakinumab Treatment Who Were Able to Taper Corticosteroids Successfully
timeFrame: Week 28
Secondary outcomes:
measure: Percentage of Participants Who Met the Adapted ACR Pediatric 30/50/70/90/100 Criteria of Canakinumab Over Time
timeFrame: Weeks 4, 8, 28, 48, 96, 144, end of study (EOS) (up to Week 164)
measure: Absolute Change From Baseline in the Adapted ACR Pediatric Criteria of Canakinumab Over Time: ACR Component: Physician's Global Assessment of Disease Activity
timeFrame: Baseline, Weeks 4, 8, 28, 48, 96, 144, EOS (up to Week 164)
measure: Absolute Change From Baseline in the Adapted ACR Pediatric Criteria of Canakinumab Over Time: ACR Component: CHAQ: Parent's or Patient's Global Assessment of Patient's Overall Well-being as Part of CHAQ
timeFrame: Baseline, Weeks 4, 8, 28, 48, 96, 144, EOS up to Week 164
measure: Absolute Change From Baseline in the Adapted ACR Pediatric Criteria of Canakinumab Over Time: ACR Component: CHAQ: Functional Ability Score
timeFrame: Baseline, Weeks 4, 8, 28, 48, 96, 144, EOS (up to Week 164)
measure: Absolute Change From Baseline in the Adapted ACR Pediatric Criteria of Canakinumab Over Time: ACR Component: Number of Joints With Active Arthritis
timeFrame: Baseline, Weeks 4, 8, 28, 48, 96, 144, EOS (up to Week 164)
measure: Absolute Change From Baseline in the Adapted ACR Pediatric Criteria of Canakinumab Over Time: ACR Component: Number of Joints With Limitation of Motion
timeFrame: Baseline, Weeks 4, 8, 28, 48, 96, 144, EOS (up to Week 164)
measure: Number of Participants Having Fever in the Adapted ACR Pediatric Criteria of Canakinumab Over Time
timeFrame: Baseline, Day 3, Weeks 2, 8, 28, 48, 56, 96, 124, 144, EOS (up to Week 164)
measure: Percentage Change From Baseline in the Adapted ACR Pediatric Criteria of Canakinumab Over Time: ACR Component: Standardized C-Reactive Protein (CRP)
timeFrame: Baseline, Weeks 4, 8, 28, 48, 96, 144, EOS (up to Week 164)
measure: Percentage of Participants Who Had Flares With Canakinumab Treatment Over Time
timeFrame: > Day3, to <= Week 124
measure: Percentage of Participants Who Achieved Inactive Disease (With and Without Duration of Morning Stiffness) With Canakinumab Treatment Over Time
timeFrame: Weeks 4, 8, 28, 48, 96, 144, EOS (up to Week 164)
measure: Percentage of Participants With Canakinumab Treatment Who Were Able to Taper Corticosteroids Successfully Over Time
timeFrame: Weeks 28, 48, 96, 144, EOS (up to Week 164)
measure: Absolute Change From Baseline of Corticosteroids Dose Reduction With Canakinumab Treatment Over Time
timeFrame: Baseline, Weeks 28, 48, 96, 144, EOS (up to Week 164)
measure: Serum Concentration of Canakinumab
timeFrame: Baseline, Weeks 4, 24, 48, 72, 96, EOS (up to Week 164)
measure: Pharmacodynamics (PD) Assessment: Total IL-1 Beta
timeFrame: Baseline, Weeks 4, 24, 48, 72, 96, EOS (up to Week 164)
Other outcomes:
Locations:
city: Obu
country: Japan
facility: Novartis Investigative Site
state: Aichi
city: Chiba-city
country: Japan
facility: Novartis Investigative Site
state: Chiba
city: Kanazawa-city
country: Japan
facility: Novartis Investigative Site
state: Ishikawa
city: Kagoshima city
country: Japan
facility: Novartis Investigative Site
state: Kagoshima
city: Yokohama-city
country: Japan
facility: Novartis Investigative Site
state: Kanagawa
city: Yokohama-city
country: Japan
facility: Novartis Investigative Site
state: Kanagawa
city: Sendai-city
country: Japan
facility: Novartis Investigative Site
state: Miyagi
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References:
citation: Nishimura K, Hara R, Umebayashi H, Takei S, Iwata N, Imagawa T, Shimizu M, Tomiita M, Seko N, Kitawaki T, Yokota S. Efficacy and safety of canakinumab in systemic juvenile idiopathic arthritis: 48-week results from an open-label phase III study in Japanese patients. Mod Rheumatol. 2021 Jan;31(1):226-234. doi: 10.1080/14397595.2020.1783163. Epub 2020 Jul 29.