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)

Plain English Summary

Study of Efficacy and Safety of Canakinumab in Japanese Patients With AOSD is a Phase 3 clinical trial sponsored by Novartis Pharmaceuticals studying Adult Onset Still's Disease. Tests the effectiveness and safety of Canakinumab in treating Adult-Onset Still's Disease (AOSD) for at least 48 weeks. For Japanese patients aged 16 and older with confirmed AOSD. Participation involves receiving subcutaneous injections of Canakinumab every 4 weeks at designated study sites. Alternative treatments include traditional medications and other biologics. The trial aims to enroll 14 participants.

Official 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.

Who Can Participate

Here is what you need to know about eligibility for this trial. Eligible: Japanese patients aged 16 and older with confirmed AOSD. Not eligible: Pregnant or nursing females, those with significant hypersensitivity to the study drug, or those with active infections. Age: 16 years and older. Health: Confirmed AOSD diagnosis, active disease at baseline. This trial is studying Adult Onset Still's Disease, so participants generally need a confirmed diagnosis.

What They're Measuring

The primary outcome measures the percentage of participants achieving an ACR 30 response at Week 8, indicating improvement in symptoms and disease activity. The specific primary outcome measures are: Percentage of Participants Who Achieved Adapted American College of Rheumatology (ACR) 30 Response at Week 8 (Baseline, Week 8). These endpoints are how researchers determine whether the treatment is effective and will form the basis of any future regulatory submissions.

About This Phase

This trial is in Phase 3, the final and most rigorous stage before seeking FDA approval. Phase 3 trials involve 300-3,000+ patients across multiple sites and compare the new treatment directly against the current standard of care. These pivotal trials generate the evidence needed for regulatory review. About 58% of Phase 3 drugs receive FDA approval. Successful Phase 3 results typically lead to a New Drug Application submission.

Why This Trial Matters

This trial addresses the treatment gap for AOSD by evaluating Canakinumab's efficacy and safety in a specific patient population. As a Phase 3 trial, positive results could directly lead to FDA approval, making this treatment available to the broader patient population. This research targets Adult Onset Still's Disease, where improved treatment options are needed.

Investor Insight

Market size is significant, with AOSD affecting thousands of patients globally. The competitive landscape is promising, with Canakinumab showing potential for approval. Phase 3 trials have approximately a 50-60% chance of gaining FDA approval if they reach this stage.

Is This Trial Right for Me?

Ask your doctor if you meet the eligibility criteria and if Canakinumab is right for you. Participation involves regular visits to the study site for injections and assessments. The trial is being conducted at 9 sites. Always discuss clinical trial participation with your healthcare provider before making any decisions. This information is for educational purposes only and is not medical advice.

AI-generated analysis for educational purposes only. This is not medical advice. Discuss clinical trial participation with your doctor. Data sourced from ClinicalTrials.gov.

Study Design

• Study Type: INTERVENTIONAL
• Allocation: NA
• Model: SINGLE_GROUP
• Masking: NONE
• Enrollment: 14 participants

Interventions

• BIOLOGICAL: Canakinumab — 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 k

Primary Outcomes

• Percentage of Participants Who Achieved Adapted American College of Rheumatology (ACR) 30 Response at Week 8 (Baseline, Week 8)

Secondary Outcomes

• 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))

Full Eligibility Criteria

Inclusion Criteria:

* 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

Exclusion Criteria:

* 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

• Novartis Investigative Site, Chiba, Chiba, Japan
• Novartis Investigative Site, Sapporo, Hokkaido, Japan
• Novartis Investigative Site, Yokohama, Kanagawa-ku, Japan
• Novartis Investigative Site, Iruma-gun, Saitama, Japan
• Novartis Investigative Site, Bunkyo Ku, Tokyo, Japan
• Novartis Investigative Site, Chuo Ku, Tokyo, Japan
• Novartis Investigative Site, Mitaka, Tokyo, Japan
• Novartis Investigative Site, Shinjuku-ku, Tokyo, Japan
• Novartis Investigative Site, Ishikawa, Japan

Frequently Asked Questions

Related Conditions

• Related conditions: Juvenile Idiopathic Arthritis, Systemic Lupus Erythematosus, Rheumatoid Arthritis
• Therapeutic areas: Autoimmune Diseases, Rheumatology, Immunology

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