Adult Onset Still's DiseasePhase 3NCT04717635
Study of Efficacy and Safety of Canakinumab in Japanese Patients With AOSD
A Phase 3 study testing Canakinumab in people with Adult Onset Still's Disease, run by Novartis Pharmaceuticals at 9 sites in Japan.
Trial summary
This study was designed to evaluate the efficacy and safety of canakinumab administered subcutaneously every 4 weeks for at least 48 weeks in Japanese patients with Adult-Onset Still's Disease (AOSD). Read more about this study
- Who is it for?
- Ages 16 to 100 years
- Conditions the study is for
- Adult Onset Still's Disease
- 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
- 14 participants
- Where
- 9 sitesJapan
- Study dates
- 30 Mar 2021to 16 Apr 2025
- 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 16 to 100 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Signed informed consent had to be obtained prior to participation in the study. Parent's or legal guardian's written informed consent and child's assent, if appropriate, were required before any assessment was performed for participants < 20 years of age.
- Japanese male and female participants aged ≥ 16 years were eligible.
- Diagnosis of AOSD had to be confirmed as per Yamaguchi criteria, with disease onset at ≥ 16 years of age. The Yamaguchi criteria required at least five criteria, including two major criteria and no exclusion criteria.
- Participants had to have active disease at baseline, defined as follows:
- Fever (body temperature > 38°C) due to AOSD for at least 1 day within 1 week before baseline
- At least 2 active joints (tender or swollen)
- CRP ≥ 10 mg/L
Who cannot join
- Pregnant or nursing (lactating) female participants were excluded. Pregnancy was defined as the state of a female after conception and until the termination of gestation, confirmed by a positive hCG laboratory test (> 5 mIU/mL) at the screening visit.
- Participants with a history of significant hypersensitivity to the study drug or to biologics were excluded.
- Participants with a history or evidence of active macrophage activation syndrome or disseminated intravascular coagulation within 6 months prior to enrollment were excluded.
- Participants with underlying metabolic, renal, hepatic, infectious, or gastrointestinal conditions that, in the opinion of the investigator, compromised the participant and/or placed the participant at unacceptable risk for participation in immunomodulatory therapy were excluded.
- Participants with active or recurrent bacterial, fungal, or viral infections at the time of enrollment, including evidence of HIV infection, Hepatitis B, or Hepatitis C, were excluded.
- Participants with an absolute neutrophil count < 1500/mm³ at screening were excluded.
Trial locations and contacts
9 sites in 1 country.
9 sites in 1 country
Japan9 sites
Novartis Investigative Site
Bunkyo Ku, Tokyo, 1138655
Novartis Investigative Site
Chiba, Chiba, 260 8677
Novartis Investigative Site
Chuo Ku, Tokyo, 1048560
Novartis Investigative Site
Iruma-gun, Saitama, 3500495
Novartis Investigative Site
Ishikawa, 9208641
Novartis Investigative Site
Mitaka, Tokyo, 181-8611
Novartis Investigative Site
Sapporo, Hokkaido, 0608648
Novartis Investigative Site
Shinjuku-ku, Tokyo, 1608582
Novartis Investigative Site
Yokohama, Kanagawa-ku, 236-0004
About this study
Official title: An Open-label, Single-arm, Active-treatment Study to Evaluate Efficacy and Safety of Canakinumab (ACZ885) Administered for at Least 48 Weeks in Japanese Patients With Adult Onset Still's Disease (AOSD)
This study was designed to evaluate the efficacy and safety of canakinumab administered subcutaneously every 4 weeks for at least 48 weeks in Japanese patients with Adult-Onset Still's Disease (AOSD).
Interim analysis data collected at Weeks 28 and 48 supported the registration submission of canakinumab for the indication of Adult Still's Disease (ASD) in Japan.
Read the detailed description
This was a Phase III, open-label, single-arm active treatment study designed to evaluate the efficacy, safety, tolerability, and pharmacokinetics/pharmacodynamics (PK/PD) of canakinumab at a dose of 4 mg/kg (up to a maximum of 300 mg) administered subcutaneously every 4 weeks for at least 48 weeks in Japanese participants with ASD.
The study consisted of two epochs:
- Screening epoch: Day -28 to Day -1
- Open-label treatment epoch: Day 1 until either regulatory approval and commercial availability of canakinumab for adult Still's disease (ASD) in Japan, or study termination (whichever occurred first).
- Conditions
- Adult Onset Still's Disease
- Being tested
- Canakinumab
- Keywords
- Canakinumab, AOSD, IL-1β, Japanese participants
- Medical subject headings
- Still's Disease, Adult-Onset
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, administered at a dose of 4 mg/kg every four weeks. The medication was supplied by Novartis in individual 2 mL glass vials, each containing 150 mg of liquid canakinumab. Throughout the study, participants received subcutaneous injections of canakinumab 4 mg/kg (up to a maximum of 300 mg) at the study site every four weeks. Any participant who required a dose greater than a single dose of 150 mg (participants > 37.5 kg) received two s.c. injections per administration
| Study group | Role | What they receive |
|---|---|---|
| Canakinumab | Experimental | All participants received canakinumab as open-label study medication. Canakinumab was administered subcutaneously at a dose of 4 mg/kg every four weeks, with a maximum allowable single dose of 300 mg. Receives: Biological: Canakinumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Participants Who Achieved Adapted American College of Rheumatology (ACR) 30 Response at Week 8
Adapted ACR30 response was defined as a ≥30% improvement from baseline in at least 3 of the following 5 core response variables, with no more than one of these variables worsening by >30%:
- Physicians global assessment of disease activity (PhGA) on a 0-100 mm visual analogue scale (VAS) (0=very good and 100=very poor).
- Participant's assessment of disease activity (PtGA) on a 0-100 mm VAS (0=very good and 100=very poor).
- Health Assessment Questionnaire- disability index (HAQ-DI): 20 questions across 8 domains assessing the functional abilities. The total score was calculated as the average of the scores for each domain, ranging from 0 (no disability) to 3 (very severe disability).
- Number of active joints (68 joints evaluated for pain/tenderness and 66 for swelling)
- Index of inflammation: C-reactive Protein (CRP) levels Additionally, participants were required to have no intermittent fever during the preceding week.
Measured: Baseline, Week 8
Secondary outcomes (19)
Number of Participants Who Were Able to Taper Corticosteroids Based on Success Criteria at Week 28.
Number of participants achieving successful corticosteroid tapering at Week 28 was assessed. Successful tapering was defined as meeting any one of the following criteria and maintaining a minimum Adapted ACR30 response:
- Participants with a baseline prednisone equivalent dose > 0.8 mg/kg/day must reduce to ≤ 0.5 mg/kg/day
- Participants with a baseline dose between ≥ 0.5 and ≤ 0.8 mg/kg/day must reduce by ≥ 0.3 mg/kg/day
- Participants with any baseline dose must reduce to ≤ 0.2 mg/kg/day
- Participants with a baseline dose ≤ 0.2 mg/kg/day must achieve any reduction
The Adapted ACR30 response requires ≥30% improvement in at least 3 of the following 5 variables, with no more than one worsening by >30%, and no intermittent fever in the preceding week:
- PhGA [0-100 VAS]
- PtGA [0-100 VAS]
- HAQ-DI [0-3 scale]
- Number of active joints (pain/tenderness: 0-68; swelling: 0-66)
- CRP levels
Measured: Baseline, Week 28
Number of Participants Who Achieved Adapted ACR 30 Response Criteria
Adapted ACR30 response was defined as a ≥30% improvement from baseline in at least 3 of the following 5 core response variables, with no more than one of these variables worsening by >30%:
- Physicians global assessment of disease activity (PhGA) on a 0-100 mm VAS (0=very good and 100=very poor).
- Participant's assessment of disease activity (PtGA) on a 0-100 mm VAS (0=very good and 100=very poor).
- Health Assessment Questionnaire- disability index (HAQ-DI): 20 questions across 8 domains assessing the functional abilities. The total score was calculated as the average of the scores for each domain, ranging from 0 (no disability) to 3 (very severe disability).
- Number of active joints (68 joints evaluated for pain/tenderness and 66 for swelling)
- Index of inflammation: C-reactive Protein (CRP) levels Additionally, participants were required to have no intermittent fever during the preceding week.
Measured: Baseline, Day 15, Weeks 4, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, 180, 192, and End of Study visit (assesed up to approx. 208 weeks)
Number of Participants Who Achieved Adapted ACR 50 Response Criteria
Adapted ACR50 response was defined as 50% reduction between baseline in at least 3 of the 5 response variables, with no more than one of these variables worsening by more than 30%:
- Physicians global assessment of disease activity on a 0-100 mm VAS (0=very good and 100=very poor).
- Participant's assessment of disease activity (PtGA) on a 0-100 mm VAS (0=very good and 100=very poor).
- Health Assessment Questionnaire- disability index (HAQ-DI): 20 questions across 8 domains assessing the functional abilities. The total score was calculated as the average of the scores for each domain, ranging from 0 (no disability) to 3 (very severe disability).
- Number of active joints (68 joints evaluated for pain/tenderness and 66 for swelling)
- Index of inflammation: C-reactive Protein (CRP) levels Additionally, participants were required to have no intermittent fever during the preceding week.
Measured: Baseline, Day 15, Weeks 4, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, 180, 192, and End of Study visit (assesed up to approx. 208 weeks)
Number of Participants Who Achieved Adapted ACR 70 Response Criteria
Adapted ACR70 response was defined as 70% reduction between baseline in at least 3 of the 5 response variables, with no more than one of these variables worsening by more than 30%:
- Physicians global assessment of disease activity (PhGA) on a 0-100 mm VAS (0=very good and 100=very poor).
- Participant's assessment of disease activity (PtGA) on a 0-100 mm VAS (0=very good and 100=very poor).
- Health Assessment Questionnaire- disability index (HAQ-DI): 20 questions across 8 domains assessing the functional abilities. The total score was calculated as the average of the scores for each domain, ranging from 0 (no disability) to 3 (very severe disability).
- Number of active joints (68 joints evaluated for pain/tenderness and 66 for swelling)
- Index of inflammation: C-reactive Protein (CRP) levels Additionally, participants were required to have no intermittent fever during the preceding week.
Measured: Baseline, Day 15, Weeks 4, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, 180, 192, and End of Study visit (assesed up to approx. 208 weeks)
Number of Participants Who Achieved Adapted ACR 90 Response Criteria
Adapted ACR90 response was defined as 90% reduction between baseline in at least 3 of the 5 response variables, with no more than one of these variables worsening by more than 30%:
- Physicians global assessment of disease activity (PhGA) on a 0-100 mm VAS (0=very good and 100=very poor).
- Participant's assessment of disease activity (PtGA) on a 0-100 mm VAS (0=very good and 100=very poor).
- Health Assessment Questionnaire- disability index (HAQ-DI): 20 questions across 8 domains assessing the functional abilities. The total score was calculated as the average of the scores for each domain, ranging from 0 (no disability) to 3 (very severe disability).
- Number of active joints (68 joints evaluated for pain/tenderness and 66 for swelling)
- Index of inflammation: C-reactive Protein (CRP) levels Additionally, participants were required to have no intermittent fever during the preceding week.
Measured: Baseline, Day 15, Weeks 4, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, 180, 192, and End of Study visit (assesed up to approx. 208 weeks)
Number of Participants Who Achieved Adapted ACR 100 Response Criteria
Adapted ACR100 response was defined as 100% reduction between baseline in at least 3 of the 5 response variables, with no more than one of these variables worsening by more than 30%:
- Physicians global assessment of disease activity (PhGA) on a 0-100 mm VAS (0=very good and 100=very poor).
- Participant's assessment of disease activity (PtGA) on a 0-100 mm VAS (0=very good and 100=very poor).
- Health Assessment Questionnaire- disability index (HAQ-DI): 20 questions across 8 domains assessing the functional abilities. The total score was calculated as the average of the scores for each domain, ranging from 0 (no disability) to 3 (very severe disability).
- Number of active joints (68 joints evaluated for pain/tenderness and 66 for swelling)
- Index of inflammation: C-reactive Protein (CRP) levels Additionally, participants were required to have no intermittent fever during the preceding week.
Measured: Baseline, Day 15, Weeks 4, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, 180, 192, and End of Study visit (assesed up to approx. 208 weeks)
Change From Baseline in Systemic Feature Score (SFS)
The SFS evaluated 10 features, each scored as 1 (present) or 0 (absent). The total score ranged from 0 (none present) to 10 (all present). A negative change from baseline indicated clinical improvement.
Clinical features:
- Fever (>37.5°C for ≥5 consecutive days)
- Rash (salmon-pink during febrile episodes)
- Serositis
- Lymphadenopathy (>1.5 cm)
- Hepatomegaly/splenomegaly (confirmed by ultrasound or palpation)
Laboratory features:
At baseline, considered present if:
- ESR ≥20 mm/hour
- CRP ≥10 mg/L
- WBC count ≥12×10⁹/L
- Hemoglobin ≤11 g/dL
- Platelets ≥400×10⁹/L
During treatment and follow-up:
- ESR: 0 if <20 mm/hour or decreased ≥30%; 1 if increased or decreased <30%
- CRP: 0 if <10 mg/L or decreased ≥30%; 1 if increased or decreased <30%
- WBC count: 0 if <12×10⁹/L or decreased ≥20%; 1 if increased or decreased <20%
- Hemoglobin: 0 if >11 g/dL or increased ≥20%; 1 if decreased or increased <20%
- Platelets: 0 if <400×10⁹/L or decreased ≥20%; 1 if increased or decreased <20%
Measured: Baseline, Day 15, Weeks 4, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, 180, 192, and End of Study visit (assesed up to approx. 208 weeks)
Change From Baseline in the Component of Adapted ACR: Physician's Global Assessment of Disease Activity
The physician rated the participant's disease activity on a 0-100 mm VAS, ranging from no disease activity (0 mm) to very severe disease activity (100 mm). A negative change from baseline indicated improvement
Measured: aseline, Day 15, Weeks 4, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, 180, 192, and End of Study visit (assesed up to approx. 208 weeks)
Change From Baseline in the Component of Adapted ACR: Patient's Global Assessment of Disease Activity
The participant's assessment of disease activity was assessed on the VAS. The VAS scale ranges from 0-100 mm, from no pain/very well (0 mm) to very severe pain/very poor (100 mm). A negative change from baseline indicated improvement
Measured: Baseline, Day 15, Weeks 4, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, 180, 192, and End of Study visit (assesed up to approx. 208 weeks)
Change From Baseline in the Component of Adapted ACR: Health Assessment Questionnaire- Disability Index (HAQ-DI)
The HAQ was used to assess physical ability and functional status of participants as well as quality of life. The disability dimension consisted of 20 multiple choice items concerning difficulty in performing eight common activities of daily living; dressing, arising, eating, walking, hygiene, reach, grip and usual activities. Each item is scored from 0 (no difficulty) to 3 (unable to do). The score for each domain is the highest item score, and the total score is the mean of the domain scores., ranging from 0 (no disability) to 3 (severe disability). A negative change from baseline indicated improvement.
Measured: Baseline, Day 15, Weeks 4, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, 180, 192, and End of Study visit (assesed up to approx. 208 weeks)
Change From Baseline in the Component of Adapted ACR: Number of Active Joints
Active joints were defined as joints with swelling or pain/tenderness. 68 joints were assessed for pain/tenderness and 66 joints for swelling.
The number of active joints ranged from 0 (no active joints) to 68 (all joints tender and /or swollen). A negative change from baseline indicated improvement.
Measured: Baseline, Day 15, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, 180, 192, End of Study (up to approx. 208 weeks)
Change From Baseline in the Component of Adapted ACR: C-Reactive Protein (CRP) Levels
CRP levels in the blood were determined A negative change from baseline indicated improvement in systemic inflammation
Measured: Baseline, Day 15, Weeks 4, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, 180, 192, and End of Study visit (assesed up to approx. 208 weeks)
Number of Participants With Absence of Intermittent Fever in the Preceding Week (Component of the Adapted ACR)
The number of participants without intermittent fever associated to AOSD (defined as oral, rectal, or axillary body temperature > 38°C only for several hours during the day) was assessed.
Measured: Baseline, Day 15, Weeks 4, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, 180, 192, and End of Study visit (assesed up to approx. 208 weeks)
Change From Baseline in Oral Corticosteroid Dose
The oral corticosteroid dose was derived from the prednisone equivalent dose per day. The change from baseline was assessed. A negative change from baseline indicated reduced corticosteroid use.
Measured: Weeks 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, 180, 192, End of Study visit (assessed up to approx. 208 weeks)
Number of Participants With or Without Rash (Typical vs. Atypical)
The absence or presence of skin rash was assessed based on physical exam findings including whether it is typical or/and atypical.
Participants with a rash showing both typical and atypical features were counted in both sub-categories.
In the summary table, for each visit participants are classified as: rash present (typical and/or atypical), rash present (typical), rash present (atypical), or rash absent.
For every visit, the denominator used to calculate percentages corresponds to the number of participants with available data at that visit.
Measured: Baseline, Day 15, Weeks 4, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, 180, 192, and End of Study visit (assesed up to approx. 208 weeks)
Change From Baseline in Disease Activity Score (DAS)28 - CRP
DAS28-CRP was a composite index validated for patients with rheumatoid arthritis (RA). It took into account the following components: tender joint count (0-28), swollen joint count (0-28), C-reactive protein (CRP, mg/L), and the Patient's Global Assessment of Disease Activity (Global Health), rated from 0 (best) to 100 (worst). These results were combined to produce the DAS28-CRP score, which ranged from 1.0 (disease remission) to 9.4 (high disease activity). A negative change from baseline in DAS28-CRP indicated an improvement
Measured: Baseline, Day 15, Weeks 4, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, 180, 192, and End of Study visit (assesed up to approx. 208 weeks)
Canakinumab Concentrations Over Time
Serum canakinumab concentrations by visit
Measured: Baseline (pre-dose), Day 3, Day 15, and pre-dose at Weeks 4, 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, 180, 192.
Total IL-1β Levels
Total IL-1β (sum of IL-1β free and bound to canakinumab) in serum was assessed
Measured: Baseline (pre-dose), Day 3, Day 15, and pre-dose at Weeks 4, 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, 180, 192.
Number of Participants With Canakinumab Anti-drug Antibodies (ADA)
The ADAs against canakinumab were assessed in serum using a validated immunoassay assay. The number of participants who had a canakinumab ADA positive result was assessed
Measured: Pre-dose at baseline, Weeks 24, 48, 72, 96, 120, 144
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jan 2021
- Study start30 Mar 2021
- Primary completion16 Apr 2025
- Study completion16 Apr 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Adult Onset Still's DiseaseStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- 14 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04717635
- Sponsor's protocol ID
- CACZ885G1302
- Other IDs
- 2021-003706-50
- Status last verified
- Mar 2026
- First submitted
- 18 Dec 2020
- Last updated
- 31 Mar 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Novartis is committed to sharing access to patient-level data and supporting clinical documents from eligible studies with qualified external researchers. Requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to protect 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
Study at a glance
From the registry record, last updated by the sponsor on 31 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label, Single-arm, Active-treatment Study to Evaluate Efficacy and Safety of Canakinumab (ACZ885) Administered for at Least 48 Weeks in Japanese Patients With Adult Onset Still's Disease (AOSD)
- ClinicalTrials.gov ID
- NCT04717635
- Acronym / sponsor ID
- CACZ885G1302
- Conditions
- Adult Onset Still's Disease
- Keywords
- Canakinumab, AOSD, IL-1β, Japanese participants
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 14 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 30 Mar 2021 (actual)
- Primary completion
- 16 Apr 2025 (actual)
- Study completion
- 16 Apr 2025 (actual)
- First posted
- 22 Jan 2021 (actual)
- Results
- Posted 23 Mar 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study was designed to evaluate the efficacy and safety of canakinumab administered subcutaneously every 4 weeks for at least 48 weeks in Japanese patients with Adult-Onset Still's Disease (AOSD).
Interim analysis data collected at Weeks 28 and 48 supported the registration submission of canakinumab for the indication of Adult Still's Disease (ASD) in Japan.
Detailed description
This was a Phase III, open-label, single-arm active treatment study designed to evaluate the efficacy, safety, tolerability, and pharmacokinetics/pharmacodynamics (PK/PD) of canakinumab at a dose of 4 mg/kg (up to a maximum of 300 mg) administered subcutaneously every 4 weeks for at least 48 weeks in Japanese participants with ASD.
The study consisted of two epochs:
- Screening epoch: Day -28 to Day -1
- Open-label treatment epoch: Day 1 until either regulatory approval and commercial availability of canakinumab for adult Still's disease (ASD) in Japan, or study termination (whichever occurred first).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Canakinumab | Experimental | Biological: CanakinumabAll participants received canakinumab as open-label study medication. Canakinumab was administered subcutaneously at a dose of 4 mg/kg every four weeks, with a maximum allowable single dose of 300 mg. |
Who may qualify
- Ages
- 16 to 100 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- Signed informed consent had to be obtained prior to participation in the study. Parent's or legal guardian's written informed consent and child's assent, if appropriate, were required before any assessment was performed for participants < 20 years of age.
- Japanese male and female participants aged ≥ 16 years were eligible.
- Diagnosis of AOSD had to be confirmed as per Yamaguchi criteria, with disease onset at ≥ 16 years of age. The Yamaguchi criteria required at least five criteria, including two major criteria and no exclusion criteria.
- Participants had to have active disease at baseline, defined as follows:
- Fever (body temperature > 38°C) due to AOSD for at least 1 day within 1 week before baseline
Key exclusion criteria (6 listed)
- Pregnant or nursing (lactating) female participants were excluded. Pregnancy was defined as the state of a female after conception and until the termination of gestation, confirmed by a positive hCG laboratory test (> 5 mIU/mL) at the screening visit.
- Participants with a history of significant hypersensitivity to the study drug or to biologics were excluded.
- Participants with a history or evidence of active macrophage activation syndrome or disseminated intravascular coagulation within 6 months prior to enrollment were excluded.
- Participants with underlying metabolic, renal, hepatic, infectious, or gastrointestinal conditions that, in the opinion of the investigator, compromised the participant and/or placed the participant at unacceptable risk for participation in immunomodulatory therapy were excluded.
- Participants with active or recurrent bacterial, fungal, or viral infections at the time of enrollment, including evidence of HIV infection, Hepatitis B, or Hepatitis C, were excluded.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CanakinumabAlso: ACZ885Canakinumab was provided as a 150 mg/1 mL solution for subcutaneous injection, administered at a dose of 4 mg/kg every four weeks. The medication was supplied by Novartis in individual 2 mL glass vials, each containing 150 mg of liquid canakinumab. Throughout the study, participants received subcutaneous injections of canakinumab 4 mg/kg (up to a maximum of 300 mg) at the study site every four weeks. Any participant who required a dose greater than a single dose of 150 mg (participants > 37.5 kg) received two s.c. injections per administration | Biological | Canakinumab |
Primary outcome
Percentage of Participants Who Achieved Adapted American College of Rheumatology (ACR) 30 Response at Week 8
Time frame: Baseline, Week 8
Adapted ACR30 response was defined as a ≥30% improvement from baseline in at least 3 of the following 5 core response variables, with no more than one of these variables worsening by >30%: 1. Physicians global assessment of disease activity (PhGA) on a 0-100 mm visual analogue scale (VAS) (0=very good and 100=very poor). 2. Participant's assessment of disease activity (PtGA) on a 0-100 mm VAS (0=very good and 100=very poor). 3. Health Assessment Questionnaire- disability index (HAQ-DI): 20 questions across 8 domains assessing the functional abilities. The total score was calculated as the average of the scores for each domain, ranging from 0 (no disability) to 3 (very severe disability). 4. Number of active joints (68 joints evaluated for pain/tenderness and 66 for swelling) 5. Index of inflammation: C-reactive Protein (CRP) levels Additionally, participants were required to have no intermittent fever during the preceding week.
19 secondary outcomes
Number of Participants Who Were Able to Taper Corticosteroids Based on Success Criteria at Week 28.
Time frame: Baseline, Week 28
Number of Participants Who Achieved Adapted ACR 30 Response Criteria
Time frame: Baseline, Day 15, Weeks 4, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, 180, 192, and End of Study visit (assesed up to approx. 208 weeks)
Number of Participants Who Achieved Adapted ACR 50 Response Criteria
Time frame: Baseline, Day 15, Weeks 4, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, 180, 192, and End of Study visit (assesed up to approx. 208 weeks)
Number of Participants Who Achieved Adapted ACR 70 Response Criteria
Time frame: Baseline, Day 15, Weeks 4, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, 180, 192, and End of Study visit (assesed up to approx. 208 weeks)
Number of Participants Who Achieved Adapted ACR 90 Response Criteria
Time frame: Baseline, Day 15, Weeks 4, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, 180, 192, and End of Study visit (assesed up to approx. 208 weeks)
Number of Participants Who Achieved Adapted ACR 100 Response Criteria
Time frame: Baseline, Day 15, Weeks 4, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, 180, 192, and End of Study visit (assesed up to approx. 208 weeks)
Change From Baseline in Systemic Feature Score (SFS)
Time frame: Baseline, Day 15, Weeks 4, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, 180, 192, and End of Study visit (assesed up to approx. 208 weeks)
Change From Baseline in the Component of Adapted ACR: Physician's Global Assessment of Disease Activity
Time frame: aseline, Day 15, Weeks 4, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, 180, 192, and End of Study visit (assesed up to approx. 208 weeks)
Change From Baseline in the Component of Adapted ACR: Patient's Global Assessment of Disease Activity
Time frame: Baseline, Day 15, Weeks 4, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, 180, 192, and End of Study visit (assesed up to approx. 208 weeks)
Change From Baseline in the Component of Adapted ACR: Health Assessment Questionnaire- Disability Index (HAQ-DI)
Time frame: Baseline, Day 15, Weeks 4, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, 180, 192, and End of Study visit (assesed up to approx. 208 weeks)
Change From Baseline in the Component of Adapted ACR: Number of Active Joints
Time frame: Baseline, Day 15, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, 180, 192, End of Study (up to approx. 208 weeks)
Change From Baseline in the Component of Adapted ACR: C-Reactive Protein (CRP) Levels
Time frame: Baseline, Day 15, Weeks 4, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, 180, 192, and End of Study visit (assesed up to approx. 208 weeks)
Number of Participants With Absence of Intermittent Fever in the Preceding Week (Component of the Adapted ACR)
Time frame: Baseline, Day 15, Weeks 4, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, 180, 192, and End of Study visit (assesed up to approx. 208 weeks)
Change From Baseline in Oral Corticosteroid Dose
Time frame: Weeks 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, 180, 192, End of Study visit (assessed up to approx. 208 weeks)
Number of Participants With or Without Rash (Typical vs. Atypical)
Time frame: Baseline, Day 15, Weeks 4, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, 180, 192, and End of Study visit (assesed up to approx. 208 weeks)
Change From Baseline in Disease Activity Score (DAS)28 - CRP
Time frame: Baseline, Day 15, Weeks 4, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, 180, 192, and End of Study visit (assesed up to approx. 208 weeks)
Canakinumab Concentrations Over Time
Time frame: Baseline (pre-dose), Day 3, Day 15, and pre-dose at Weeks 4, 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, 180, 192.
Total IL-1β Levels
Time frame: Baseline (pre-dose), Day 3, Day 15, and pre-dose at Weeks 4, 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, 180, 192.
Number of Participants With Canakinumab Anti-drug Antibodies (ADA)
Time frame: Pre-dose at baseline, Weeks 24, 48, 72, 96, 120, 144
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Mar 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- A Plain Language Trial Summary is available on www.novctrd.comLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Novartis is committed to sharing access to patient-level data and supporting clinical documents from eligible studies with qualified external researchers. Requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to protect 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
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
9 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Canakinumab in people with Adult Onset Still's Disease, run by Novartis Pharmaceuticals at 9 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 7 inclusion and 6 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 31 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 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 23 Mar 2026). 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 Adapted American College of Rheumatology (ACR) 30 Response at Week 8 (time frame: Baseline, Week 8). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 31 Mar 2026.