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Systemic Juvenile Idiopathic ArthritisPhase 3NCT02396212

Study of Efficacy and Safety of Canakinumab in Japanese Patients With SJIA

A Phase 3 study testing Canakinumab in people with Systemic Juvenile Idiopathic Arthritis, run by Novartis Pharmaceuticals at 7 sites in Japan.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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). Read more about this study

Who is it for?
Ages 2 to 19 years
Conditions the study is for
Systemic Juvenile Idiopathic Arthritis
What is being tested?
Canakinumab
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
19 participants
Where
7 sitesJapan
Study dates
7 May 2015to 1 Aug 2018
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 2 to 19 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • 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)

Who cannot join

  • 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.

Trial locations and contacts

7 sites in 1 country.

7 sites in 1 country

Japan7 sites
  • Novartis Investigative Site

    Chiba, Chiba, 266-0007

  • Novartis Investigative Site

    Kagoshima, Kagoshima-ken, 890 8520

  • Novartis Investigative Site

    Kanazawa, Ishikawa-ken, 920-8641

  • Novartis Investigative Site

    Ōbu, Aichi-ken, 474 8710

  • Novartis Investigative Site

    Sendai, Miyagi, 989-3126

  • Novartis Investigative Site

    Yokohama, Kanagawa, 232-8555

  • Novartis Investigative Site

    Yokohama, Kanagawa, 236-0004

Other trials in Japan

About this study

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)

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.

Being tested
Canakinumab
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
Medical subject headings
Arthritis, Juvenile, Bronchiolitis Obliterans Syndrome, Arthralgia, Rheumatoid Arthritis, Systemic Juvenile

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • CanakinumabBiological

    Also called ACZ885

    canakinumab was provided as a 150 mg/1 mL solution for subcutaneous injection and administered at 4mg/kg every 4 weeks.

Study groups in NCT02396212
Study groupRoleWhat they receive
CanakinumabExperimentalAll patients received canakinumab (ACZ885) as open-label study medication. Patients were administered canakinumab 4 mg/kg every 4 weeks. The maximal total single dose of canakinumab allowed was 300 mg.
Receives: Biological: Canakinumab

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Percentage of Participants Who Achieved a Minimum Adapted American College of Rheumatology (ACR) Pediatric 30 Criteria

    Minimum Adapted ACR Pediatric 30 criteria is defined as improvement from baseline at least 30% in at least 3 of response variables 1 to 6 in Adapted ACR Pediatric response variables and no intermittent fever (i.e. axillary, oral, or rectal body temperature ≤ 38°C) in the preceding week (variable 7), with no more than one variable 1-6 worsening by more than 30%. Adapted ACR Pediatric response variables consists of following 7 variables: 1. Physician's Global Assessment of disease activity on a 0-100 mm VAS; 2. Parent's or Patient's (if appropriate in age) Global Assessment of Patient's overall wellbeing based upon the 0-100 mm VAS in the Child Health Assessment Questionnaire (CHAQ); 3. Functional ability: CHAQ; 4. Number of joints with active arthritis; 5. Number of joints with limitation of motion; 6. Laboratory measure of inflammation: CRP (mg/L); 7. Absence of intermittent fever due to SJIA during the preceding week.

    Measured: Week 8

  2. Percentage of Participants With Canakinumab Treatment Who Were Able to Taper Corticosteroids Successfully

    To evaluate the percentage of participants with canakinumab treatment who were able to taper corticosteroids successfully at Week 28

    Measured: Week 28

Secondary outcomes (14)
  1. Percentage of Participants Who Met the Adapted ACR Pediatric 30/50/70/90/100 Criteria of Canakinumab Over Time

    Adapted ACR Pediatric 30/50/70/90/100 criteria was assessed based on the following 7 variables: 1. Physician's Global Assessment of disease activity on a 0-100 mm VAS; 2. Parent's or Patient's (if appropriate in age) Global Assessment of Patient's overall wellbeing based upon the 0-100 mm VAS in the CHAQ; 3. Functional ability: CHAQ; 4. Number of joints with active arthritis; 5. Number of joints with limitation of motion; 6. Laboratory measure of inflammation: CRP (mg/L); 7. Absence of intermittent fever due to SJIA during the preceding week. Response was defined as more than or equal to (≥) 30%/50%/70%/90% or 100% improvement in at least 3 of 6 response variables and no intermittent fever in the preceding week (variable 7) with no more than one variable 1-6 worsening by more than 30%.

    Measured: Weeks 4, 8, 28, 48, 96, 144, end of study (EOS) (up to Week 164)

  2. Absolute Change From Baseline in the Adapted ACR Pediatric Criteria of Canakinumab Over Time: ACR Component: Physician's Global Assessment of Disease Activity

    ACR component, Physician's Global Assessment of disease activity on a 0 - 100 mm VAS by visit is the first response ACR variable in the ACR pediatric criteria. The VAS scale ranges from no disease activity (0 mm) to very severe disease activity (100 mm). Lower scale indicates decreased disease activity. Change from baseline was calculated by subtracting baseline value from post baseline value.

    Measured: Baseline, Weeks 4, 8, 28, 48, 96, 144, EOS (up to Week 164)

  3. 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

    ACR component, Parent's or Patient's (if appropriate in age)Global Assessment of patient's overall well-being as part of CHAQ on a 0 - 100 mm VAS by visit is the second response variable in the ACR pediatric criteria. The VAS scale ranges from 0-100 mm, from very well (0 mm) to very poor (100 mm). Lower scale indicates improvement of patient's overall well-being. Absolute change is calculated by subtracting baseline value from post baseline value.

    Measured: Baseline, Weeks 4, 8, 28, 48, 96, 144, EOS up to Week 164

  4. Absolute Change From Baseline in the Adapted ACR Pediatric Criteria of Canakinumab Over Time: ACR Component: CHAQ: Functional Ability Score

    Disability Score as part of CHAQ per functional ability score (range from 0 to 3) is one of the variable in the ACR ped criteria. The CHAQ was used to assess physical ability \& functional status of patients as well as quality of life. The disability dimension consists of 20 multiple choice items concerning difficulty in performing 8 common activity categories of daily living: dressing \& grooming, arising, eating, walking, reaching, personal hygiene, gripping \& other "activities". Subjects choose from 4 responses, ranging from 0 (without any difficulty), 1 (with some difficulty), 2 (with much difficulty) \& 3 (unable to do). Standard Disability Index (SDI) was computed by summing up the computed scores for each activity category and dividing by the number of categories answered. The lower the response the more positive the results \& the higher the response, the less positive the results. Change from baseline was calculated by subtracting baseline value from post baseline value.

    Measured: Baseline, Weeks 4, 8, 28, 48, 96, 144, EOS (up to Week 164)

  5. Absolute Change From Baseline in the Adapted ACR Pediatric Criteria of Canakinumab Over Time: ACR Component: Number of Joints With Active Arthritis

    ACR component, Number of joints with active arthritis was assessed as the forth response variables of ACR Pediatric Criteria.

    Measured: Baseline, Weeks 4, 8, 28, 48, 96, 144, EOS (up to Week 164)

  6. Absolute Change From Baseline in the Adapted ACR Pediatric Criteria of Canakinumab Over Time: ACR Component: Number of Joints With Limitation of Motion

    ACR component, Number of joints with limitation of motion is the fifth response variable in the ACR ped criteria.

    Measured: Baseline, Weeks 4, 8, 28, 48, 96, 144, EOS (up to Week 164)

  7. Number of Participants Having Fever in the Adapted ACR Pediatric Criteria of Canakinumab Over Time

    ACR component, Number of participants having fever is the seventh response variable in the ACR ped criteria.

    Measured: Baseline, Day 3, Weeks 2, 8, 28, 48, 56, 96, 124, 144, EOS (up to Week 164)

  8. Percentage Change From Baseline in the Adapted ACR Pediatric Criteria of Canakinumab Over Time: ACR Component: Standardized C-Reactive Protein (CRP)

    ACR component, Standardized CRP is the sixth response variable in the ACR ped criteria. CRP values were standardized to a normal range of 0 to 10 mg/L.

    Measured: Baseline, Weeks 4, 8, 28, 48, 96, 144, EOS (up to Week 164)

  9. Percentage of Participants Who Had Flares With Canakinumab Treatment Over Time

    Flare was defined by at least 1 of the following: Reappearance of SJIA-related (e.g., not due to infection) fever (> 38°C) lasting for at least 2 consecutive days \&/OR Flare according to the JIA pediatric criteria for flare (all criteria must be met): ≥ 30% worsening in at least 3 of the 6 response variables and ≥ 30% improvement in at not more than 1 of the 6 response variables if the physician's or parent's global assessment is 1of 3 response variables used to define flare, worsening of ≥ 20 mm must be present, if the number of active joints or joints with limitation of motion is one of 3 response variables used to define flare, worsening in ≥ 2 joints must be present if CRP is used to define flare, CRP must be > 30 mg/L

    Measured: > Day3, to <= Week 124

  10. Percentage of Participants Who Achieved Inactive Disease (With and Without Duration of Morning Stiffness) With Canakinumab Treatment Over Time

    Inactive disease was defined as meeting all of the following: No joints with active arthritis; No fever (body temperature ≤ 38°C); No rheumatoid rash, serositis, splenomegaly, hepatomegaly or generalized lymphadenopathy attributable to JIA; Normal CRP; Physician's global assessment of disease activity score ≤ 10 mm

    Measured: Weeks 4, 8, 28, 48, 96, 144, EOS (up to Week 164)

  11. Percentage of Participants With Canakinumab Treatment Who Were Able to Taper Corticosteroids Successfully Over Time

    To evaluate the percentage of participants with canakinumab treatment who were able to taper corticosteroids successfully over time

    Measured: Weeks 28, 48, 96, 144, EOS (up to Week 164)

  12. Absolute Change From Baseline of Corticosteroids Dose Reduction With Canakinumab Treatment Over Time

    To evaluate the change from baseline of corticosteroids dose reduction with canakinumab treatment over time

    Measured: Baseline, Weeks 28, 48, 96, 144, EOS (up to Week 164)

  13. Serum Concentration of Canakinumab

    To evaluate serum concentration (mean, standard deviation) of canakinumab.

    Measured: Baseline, Weeks 4, 24, 48, 72, 96, EOS (up to Week 164)

  14. Pharmacodynamics (PD) Assessment: Total IL-1 Beta

    To evaluate serum total IL-1 Beta concentration by visit.

    Measured: Baseline, Weeks 4, 24, 48, 72, 96, EOS (up to Week 164)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted24 Mar 2015 (estimated)
  2. Study start7 May 2015
  3. Primary completion7 Mar 2017
  4. Study completion1 Aug 2018

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
19 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02396212
Sponsor's protocol ID
CACZ885G1301
Other IDs
2018-002355-15, 2018
Status last verified
Aug 2019
First submitted
25 Feb 2015
Last updated
13 Sep 2019
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
Yes: Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

Publications

  • 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.

    PubMed 32552266

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Canakinumab in people with Systemic Juvenile Idiopathic Arthritis, run by Novartis Pharmaceuticals at 7 sites in Japan.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 6 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 13 Sep 2019. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 7 sites in Japan. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 13 Sep 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Percentage of Participants Who Achieved a Minimum Adapted American College of Rheumatology (ACR) Pediatric 30 Criteria (time frame: Week 8). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02396212, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Sep 2019.

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