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

NCT04717635 | Completed Phase 3 Results

• 2 other identifiers: CACZ885G13022021-003706-50
• Study Type: interventional
• Target: 14
• Locations: 1 country
• Sites: 9

Brief 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). 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.

Conditions

Adult Onset Still's Disease

Keywords

Canakinumab; AOSD; IL-1β; Japanese participants

Outcome Measures

Primary Outcomes (1)

• 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%: 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.

  Baseline, Week 8

Secondary Outcomes (19)

• Number of Participants Who Were Able to Taper Corticosteroids Based on Success Criteria at Week 28.

  Baseline, Week 28

• Number of Participants Who Achieved Adapted ACR 30 Response Criteria

  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

  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

  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

  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)

Study Arms (1)

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.

Biological: Canakinumab

Interventions

Canakinumab BIOLOGICAL

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

Also known as: ACZ885

Canakinumab

Eligibility Criteria

• Age: 16 Years - 100 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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
• 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.

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (9)

Novartis Investigative Site

Chiba, Chiba, 260 8677, Japan

Location

Novartis Investigative Site

Sapporo, Hokkaido, 0608648, Japan

Location

Novartis Investigative Site

Yokohama, Kanagawa-ku, 236-0004, Japan

Location

Novartis Investigative Site

Iruma-gun, Saitama, 3500495, Japan

Location

Novartis Investigative Site

Bunkyo Ku, Tokyo, 1138655, Japan

Location

Novartis Investigative Site

Chuo Ku, Tokyo, 1048560, Japan

Location

Novartis Investigative Site

Mitaka, Tokyo, 181-8611, Japan

Location

Novartis Investigative Site

Shinjuku-ku, Tokyo, 1608582, Japan

Location

Novartis Investigative Site

Ishikawa, 9208641, Japan

Location

Related Links

• A Plain Language Trial Summary is available on www.novctrd.com

MeSH Terms

Conditions

Still's Disease, Adult-Onset

Interventions

canakinumab

Condition Hierarchy (Ancestors)

Arthritis, Rheumatoid; Arthritis; Joint Diseases; Musculoskeletal Diseases; Rheumatic Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases

Results Point of Contact

• Title: Study Director
• Organization: Novartis Pharmaceuticals

novartis.email@Novartis.com

+1 862 778 8300

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 18, 2020
• First Posted: January 22, 2021
• Study Start: March 30, 2021
• Primary Completion: April 16, 2025
• Study Completion: April 16, 2025
• Last Updated: March 31, 2026
• Results First Posted: March 23, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will share

Locations

JP (9)