Real Life Non-interventional Study on Safety and Effectiveness of Ilaris® (Canakinumab) 150 mg for Subcutaneous Injection in Hereditary Periodic Fever Syndrome (CAPS, crFMF, TRAPS and HIDS/MKD) Patients and sJIA Patients (REASSURE)
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 25
- 试验地点
- 2
- 主要终点
- Incidence of adverse events and serious adverse events
研究概览
简要总结
This is a study to evaluate safety and effectiveness of Ilaris in adult and pediatric patients receiving the drug in a clinical setting for any of the following indications, Hereditary Periodic Fever Syndromes, Cryopyrin-associated periodic syndromes (CAPS), colchicine resistance familial Mediterranean fever (crFMF), TNF receptor associated periodic syndrome (TRAPS), Hyper-IgD syndrome / Mevalonate kinase deficiency (HIDS/MKD) or Systemic juvenile idiopathic arthritis (sJIA).
详细描述
This is a prospective observational, multicenter, uncontrolled, open-label non-interventional study in ≥2 year and <19 year-old pediatric and ≥19 year-old adult hereditary periodic fever syndrome patients and ≥2 year and <19 year-old sJIA patients receiving Ilaris for the treatment of CAPS, crFMF, TRAPS, HIDS/MKD and sJIA, respectively, partially using retrospective observation to collect and evaluate data on the safety and effectiveness of Ilaris in patients receiving this drug in a clinical setting for any of these indications. The whole study period is up to 4 years, consisting of a 2-year enrollment period and 2-year observation period.
As all pediatric and adult hereditary periodic fever syndrome patients and all sJIA patients receiving Ilaris for approved indications will be enrolled, this study has no fixed sample size.
For subjects who started Ilaris before enrolling in this study, the safety and effectiveness baseline and early period data will be retrospectively collected.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 2 Years 至 100 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Written informed consent/assent of the patient or their legal representative/parent (≥2 year and <19 year-old pediatric patient) for voluntarily participating in this study
- •Age: ≥2 year and <19 year-old pediatric and ≥19 year-old adult hereditary periodic fever syndrome (CAPS, crFMF, TRAPS and HIDS/MKD) patients and ≥2 year and <19 year-old sJIA patients
- •Patient who have an agreement to be treated or who have already started treatment with Ilaris in accordance with the approved label information
排除标准
- •Patients receiving Ilaris treatment for autoimmune disease other than CAPS, crFMF, TRAPS, HIDS/MKD or sJIA
- •Patients participating in an interventional clinical trial which would have an impact on routine clinical treatment
研究组 & 干预措施
Ilaris
Patients treated with Ilaris in a clinical setting
干预措施: Ilaris (Biological)
结局指标
主要结局
Incidence of adverse events and serious adverse events
时间窗: Up to 104 weeks from Ilaris treatment
Adverse events and serious adverse events in hereditary periodic fever syndrome (CAPS, crFMF, TRAPS and HIDS/MKD) and sJIA patients treated with Ilaris.
次要结局
- Percent change from baseline in Health related quality of life (HRQOL) measured by Child Health Questionnaire-Parent Form 50 (CHQ-PF50)(Up to 104 weeks from Ilaris treatment)
- Proportion of complete responders(Up to 16 weeks from Liars treatment)
- Proportion of patients with C- reactive protein (CRP) serological response(Up to 104 weeks from Ilaris treatment)
- Percent change from baseline in Health related quality of life (HRQOL) measured by 36-item Short Form Health Survey (SF-36)(Up to 104 weeks from Ilaris treatment)
- Proportion of participants with Physician Global Assessment of Disease Activity (PGA) score <2(Up to 104 weeks from Ilaris treatment)
- Proportions of patients with serum amyloid A (SAA) normalization(Up to 104 weeks from Ilaris treatment)
- Proportion of patients classified in each severity level in the physician's severity assessment of key disease -specific signs and symptoms(Up to 104 weeks from Ilaris treatment)
- Proportions of patients classified in each severity level in symptoms likely to significantly affect affect physical functioning and vital prognosis(Up to 104 weeks from Ilaris treatment)
- Percent change from baseline in Health related quality of life (HRQOL) measured by Work Productivity and Activity Impairment Specific Health Problem v2.0 (WPAI-SHP)(Up to 104 weeks from Ilaris treatment)