Schnitzler Syndrome
Conditions
Keywords
Schnitzler's syndrome, Canakinumab, ILARIS, Post-marketing surveillance, All-case surveillance
Brief summary
The aim of this study is to assess the effectiveness and safety of Ilaris (canakinumab) in clinical use in patients with Schnitzler's syndrome.
Interventions
None listed
Sponsors
Novartis Pharmaceuticals
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
0 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1\. Patients diagnosed with Schnitzler's syndrome using canakinumab for the first time for the indication of Schnitzler's syndrome as described in the package insert. Patients starting treatment with canakinumab before conclusion of the contract for this study will also be included in the study so that all patients diagnosed with Schnitzler's syndrome who received canakinumab will be registered in this study.
Exclusion criteria
1. Patients previously treated with canakinumab in Study IACT21071. 2. All patients treated with canakinumab for the following indications: Indications: Cryopyrin-associated periodic syndromes (familial cold autoinflammatory syndrome, Muckle-Wells syndrome, neonatal onset multisystem inflammatory disease), hyper immunoglobulin D (IgD) syndrome (mevalonate kinase deficiency), tumor necrosis factor (TNF) receptor-associated periodic syndrome, familial mediterranean fever, systemic juvenile idiopathic arthritis, adult-onset Still's disease. 3. Patients receiving canakinumab for off-label use under the Clinical Trials Act or GCP (e.g., patient-proposed healthcare services, investigator-initiated clinical trial).
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Proportion of Patients Achieving Complete Clinical Response in the Period From 7 Days to 8 Weeks After the First Dose | Day 8 to Week 8 | Complete clinical response is defined as a Physician's Global Assessment (PGA) score of ≤ 5, with none of 5 items (urticaria, fatigue, fever and chills, myalgia, and arthralgia and bone pain) rated as \> 1. The PGA is a tool designed to evaluate each of 5 items (urticaria, fatigue, fever/chills, myalgia, and arthralgia/bone pain), which are clinical symptoms observed in patients with Schnitzler's syndrome, on a 5-point scale of 0 (no), 1 (minimal), 2 (mild), 3 (moderate), and 4 (severe) based on the physician's clinical examination to calculate the total score. |
| Proportion of Patients Achieving Partial or Complete Clinical Response at all Time Points From 7 Days After the First Dose to 48 Weeks After the First Dose | Day 8, Weeks 8, 16, 24, 32, 40, and at treatment discontinuation, up to and including Week 48 | Complete clinical response is defined as a PGA score of ≤ 5, with none of 5 items (urticaria, fatigue, fever and chills, myalgia, and arthralgia and bone pain) rated as \> 1. Partial clinical response is defined as a PGA score of ≥ 6, with a decrease in PGA of ≥ 30% from baseline, or PGA score of ≤ 5, with any of 5 items (urticaria, fatigue, fever and chills, myalgia, and arthralgia and bone pain) rated as \> 1. The PGA is a tool designed to evaluate each of 5 items (urticaria, fatigue, fever/chills, myalgia, and arthralgia/bone pain), which are clinical symptoms observed in patients with Schnitzler's syndrome, on a 5-point scale of 0 (no), 1 (minimal), 2 (mild), 3 (moderate), and 4 (severe) based on the physician's clinical examination to calculate the total score. |
| Rate of Each Clinical Response | Baseline, Day 8, Weeks 8, 16, 24, 32, 40, and at treatment discontinuation, up to and including Week 48 | The proportion of patients who achieve a clinical response: * Complete clinical response: PGA score of ≤ 5, with none of 5 items (urticaria, fatigue, fever and chills, myalgia, and arthralgia and bone pain) rated as \> 1 * Partial clinical response: PGA score of ≥ 6, with a decrease in PGA of ≥ 30% from baseline, or PGA score of ≤ 5, with any of 5 items (urticaria, fatigue, fever and chills, myalgia, and arthralgia and bone pain) rated as \> 1 * No clinical response: PGA score that increases, is stable, or decreases by \< 30% as compared to baseline * Relapse or worsening of clinical symptoms: Increase in PGA score of ≥ 50% from baseline The PGA is a tool designed to evaluate each of 5 items, which are clinical symptoms observed in patients with Schnitzler's syndrome, on a 5-point scale of 0 (no), 1 (minimal), 2 (mild), 3 (moderate), and 4 (severe) based on the physician's clinical examination to calculate the total score. |
| Mean PGA Score | Baseline, Day 8, Weeks 8, 16, 24, 32, 40, and at treatment discontinuation, up to and including Week 48 | The PGA is a tool designed to evaluate each of 5 items (urticaria, fatigue, fever/chills, myalgia, and arthralgia/bone pain), which are clinical symptoms observed in patients with Schnitzler's syndrome, on a 5-point scale of 0 (no), 1 (minimal), 2 (mild), 3 (moderate), and 4 (severe) based on the physician's clinical examination to calculate the total score. |
| Change From Baseline in PGA Score | Baseline to Day 8, Weeks 8, 16, 24, 32, 40, and at treatment discontinuation, up to and including Week 48 | The PGA is a tool designed to evaluate each of 5 items (urticaria, fatigue, fever/chills, myalgia, and arthralgia/bone pain), which are clinical symptoms observed in patients with Schnitzler's syndrome, on a 5-point scale of 0 (no), 1 (minimal), 2 (mild), 3 (moderate), and 4 (severe) based on the physician's clinical examination to calculate the total score. |
| Mean White Blood Cell Count | Baseline, Day 8, Weeks 8, 16, 24, 32, 40, and at treatment discontinuation, up to and including Week 48 | — |
| Change From Baseline in White Blood Cell Count | Baseline to Day 8, Weeks 8, 16, 24, 32, 40, and at treatment discontinuation, up to and including Week 48 | — |
| Mean Neutrophil Count | Baseline, Day 8, Weeks 8, 16, 24, 32, 40, and at treatment discontinuation, up to and including Week 48 | — |
| Change From Baseline in Neutrophil Count | Baseline to Day 8, Weeks 8, 16, 24, 32, 40, and at treatment discontinuation, up to and including Week 48 | — |
| Mean C-reactive Protein (CRP) Level | Baseline, Day 8, Weeks 8, 16, 24, 32, 40, and at treatment discontinuation, up to and including Week 48 | — |
| Change From Baseline in CRP Level | Baseline to Day 8, Weeks 8, 16, 24, 32, 40, and at treatment discontinuation, up to and including Week 48 | — |
| Mean Alkaline phosphatase (ALP) Level | Baseline, Day 8, Weeks 8, 16, 24, 32, 40, and at treatment discontinuation, up to and including Week 48 | — |
| Change From Baseline in ALP Level | Baseline to Day 8, Weeks 8, 16, 24, 32, 40, and at treatment discontinuation, up to and including Week 48 | — |
| Mean Serum Immunoglobulin (Ig) G and IgM Levels | Baseline, Day 8, Weeks 8, 16, 24, 32, 40, and at treatment discontinuation, up to and including Week 48 | — |
| Change From Baseline in Serum IgG and IgM Levels | Baseline to Day 8, Weeks 8, 16, 24, 32, 40, and at treatment discontinuation, up to and including Week 48 | — |
| Rate of Physicians Global Assessment of Impression of Improvement | Day 8, Weeks 8, 16, 24, 32, 40, and at treatment discontinuation, up to and including Week 48 | The proportion of patients summarized by improvement status as assessed by the physician. Improvement assessment categories: markedly responded, responded, not responded, worsen, or indeterminate. |
Secondary
| Measure | Time frame |
|---|---|
| Number and Percentage of Patients With Adverse Reactions | From Baseline to Week 48 |
| Number and Percentage of Patients With Serious Adverse Events | From Baseline to Week 48 |
Countries
Japan
Contacts
CONTACTNovartis Pharmaceuticals
STUDY_DIRECTORNovartis Pharmaceuticals
Novartis Pharmaceuticals
Outcome results
None listed