UCAN CAN-DU: Canada-Netherlands Personalized Medicine Network in Childhood Arthritis and Rheumatic Disease
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 4,100
- 试验地点
- 19
- 主要终点
- Prospectively collect essential clinical data elements from children with new onset JIA
研究概览
简要总结
Childhood arthritis is a chronic disabling disease. New medications called biologic therapies are now available to treat arthritis that target key biologic molecules that cause inflammation. Biologic therapies, while very effective in treating arthritis in children, may have serious side effects including infections and potentially cancers, and are very expensive and doctors don't know, which one to choose for which child. The investigators will develop tests that enable them to learn about the biology of each child's arthritis and be able to predict when and which biologic therapy to start and when to stop.
详细描述
UCAN CAN-DU is a multicenter observational cohort study that will collect prospective data from children with arthritis. Biologic samples, clinical data and patient reported outcomes will be collected.
In addition, the study will also include a health economics component which will include a number of complementary approaches for quantifying and comparing benefits and risks that promote evidence-based, patient centered health care. This will address both the personal and societal economic burden of disease and include qualitative methods to inform the measurement of preferences, economic and simulation modelling to assess the value of biomarker testing. The socioeconomic impact of biomarker based treatment will be evaluated.
All clinical, biological and patient-derived data will be collected at an aggregation point housed and managed by High Performance Computing 4 Health (HPC4Health), a private hospital-only secure cloud-computing service within Compute Canada and physically located at SickKids/UHN. These databases and apps include biospecimen data and data collected through the eHealth platform. This will enable the study team to share and integrate data in near real-time into analytic models throughout the study course; hence providing a near real-time feedback from bench to bedside and vice versa.
The analysis of the cohorts will help define and confirm the biologic pathways predictive of disease course, treatment response and disease remission. This knowledge will then be used to develop a comprehensive clinical predictive tool to guide effective and safe treatment of childhood arthritis.
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
- — 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Cohort 1: - Biologic Basis of JIA
- •≤18 years*
- •Active objective arthritis suspected to be JIA or diagnosed with JIA within 6 months of enrolment
- •Treatment naïve except for NSAIDs, allowed to have received NSAIDS within 6 months of diagnosis
- •Cohort 2 - Start Biologics
- •JIA diagnosis as per ILAR criteria (all subtypes)
- •≤18 years*
- •Active arthritis
- •For sJIA, active disease not necessarily with arthritis.
- •Time of start, restart or switch biologic therapy: e.g. failure, insufficient/partial response or intolerance
- •Cohort 3 - Stop Biologics
- •JIA diagnosis as per ILAR criteria (all subtypes)
- •≤18 years*
- •Inactive disease
- •Discontinuing/tapering biologics for inactive disease
- •Cohort 4: Extreme Phenotypes
- •Unexplained systemic inflammation with arthritis/arthralgia as a part of manifestations
- •High suspicion of genetic contribution
- •Severely affected patients with difficult to control disease (ie failure of multiple biologics)
排除标准
- •Arthritis explained by another diagnosis
- •Joint injections as previous treatment less than 4 weeks prior to enrollment
- •Arthritis explained by any other cause
- •Start on biologics as an indication for uveitis only
- •Tapering scheme > 12 months to complete biologics stop
- •Arthritis explained by another diagnosis
结局指标
主要结局
Prospectively collect essential clinical data elements from children with new onset JIA
时间窗: Up to 24 months
Evaluate clinical outcomes associated with the use of therapeutic agents in children with JIA
时间窗: Up to 24 months
Evaluate biological outcomes associated with the use of therapeutic agents in children with JIA
时间窗: Up to 24 months
Evaluate clinical outcomes associated with the de-prescribing of therapeutic agents in children with JIA
时间窗: Up to 24 months
Prospectively collect essential biological data elements from children with new onset JIA
时间窗: Up to 24 months
Evaluate biological outcomes associated with the de-prescribing of therapeutic agents in children with JIA
时间窗: Up to 24 months
Prospectively collect essential biological data elements from children with extreme phenotypes of JIA
时间窗: Up to 12 months
Evaluate the socioeconomic impact associated with the use of therapeutic agents in children with JIA
时间窗: Up to 12 months
Prospectively collect essential socioeconomic data elements from children with extreme phenotypes of JIA
时间窗: Up to 12 months
Prospectively collect essential clinical data elements from children with extreme phenotypes of JIA.
时间窗: Up to 12 months
Prospectively collect essential socioeconomic data elements from children with new onset JIA
时间窗: Up to 12 months
Evaluate the socioeconomic impact associated with the de-prescribing of therapeutic agents in children with JIA
时间窗: Up to 24 months
次要结局
未报告次要终点
研究者
Rae Yeung
Professor, Staff Physician and Senior Scientist
The Hospital for Sick Children