Clinical Database and Biobank of Patients With Inflammatory Myopathies: the MASC Project (Myositis, DNA, Serum, Cells) (MASC)
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 1,273
- 试验地点
- 1
- 主要终点
- Characterisation of the different myositis subgroups based on clinical, radiological, electrophysiological and histo-biological evaluations
研究概览
简要总结
Myositis are rare diseases for which the development of a cohort associated with a bank of biological samples (biobank) will allow for the conduct of researches to better delineate the underlying pathophysiology and find cures. This prospective cohort of patients with myositis will allow for identification of factors favouring the occurrence of myositis, whether they are constitutional (genetic) or acquired (environmental or drug). Different subgroups of myositis used for prognostication will be identified based on clinico-demographical variables, the nature of the organs involved beyond peripheral muscles (cardiac, diaphragm) and biomarkers abnormalities.
详细描述
Myositis is a rare autoimmune disease in which the immune system mistakenly attacks the patient's own peripheral muscles. This aggression manifests by muscle inflammation and necrosis responsible for a motor deficit of varying severity.
The treatments available today are insufficient and are non-specific. Biological criteria, issued from simple blood or muscle tests are missing, and they will help to define the activity of the disease and the efficacy of treatments.
The MASC protocol will include patients with myositis, and investigators will collect clinical, radiological, electrophysiological, histological and biological data to be used for researches aiming at better understanding this entity. A biobank (muscle biopsy, DNA, serum, plasma, PBMCs) will be acquired on this prospective cohort.
The study itself will be composed of a baseline visit and monthly to yearly follow-up visits which will assess:
- Clinical examination with an evaluation of the muscle strength and function impairment/handicap, including but not limited to:
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •All patients who had a confirmed (muscular biopsy, electromyogram, magnetic resonance imaging) or suspected clinically myositis. Myositis criteria are as follow:
- •Dermatomyositis or polymyositis according to Bohan and Peter criteria (1975)
- •Body inclusion myositis according to Griggs et al. criteria (1995)
- •Necrotizing autoimmune myopathy according to Hoogendijk et al. criteria (2004)
- •Drug-induced myositis
- •Signature of the informed consent form for the study and for the biobank
- •Age over 18 years old
排除标准
- 未提供
结局指标
主要结局
Characterisation of the different myositis subgroups based on clinical, radiological, electrophysiological and histo-biological evaluations
时间窗: baseline: first 30 days after inclusion
Characterisation of the different myositis subgroups based on clinical, radiological, electrophysiological and histo-biological evaluations, including but not limited to: sexe, age, profession, a history of infection, cancer or other autoimmune and inflammatory diseases, diagnosis criteria, creatine phosphokinase, autoantibodies, immune systeme evaluation based on peripheral blood mononuclear cells, DNA sequencing muscular biopsies
次要结局
- Characterisation of an immune system signature, using peripheral blood mononuclear cells and muscular biopsies, DNA and RNA sequencing, and autoantibodies(baseline: first 30 days after inclusion)
- Characterisation of a global activity scale using biological data (CPK), muscle weakness (muscle testing) and other visceral involvements(up to twenty years after inclusion)
- Incidence of major cardio-vascular events(up to twenty years after inclusion)
- Change of the quality of life, using quality of life questionnaires, depending of patients and disease characteristics(up to twenty years after inclusion)
- Risk factors for All-cause mortality depending on patient's and disease characteristics(up to twenty years after inclusion)
- Characterisation of a quality-of-life scale using biological data (CPK), muscle weakness (muscle testing) and other visceral involvements(up to twenty years after inclusion)
- Consequences on outcomes of major cardio-vascular events(up to twenty years after inclusion)
- Correlation of myositis with the development of extra-muscular diseases including but not limited to dermatological, rheumatological, cardiological and pneumological associated diseases(up to twenty years after inclusion)
- Characterisation of diaphragmatic failure with pulmonary function test and thoracic tomodensitometry(up to twenty years after inclusion)
- Follow up of respiratory function with pulmonary function test and thoracic tomodensitometry(up to twenty years after inclusion)
- Characterisation of the natural history of myositis subgroups :responses to treatments, prognosis factors, evolution(up to twenty years after inclusion)
- Change of activity impairment using an evaluation of daily life activity by both patient and physician using a Visual Analogue Scale depending of patients and disease characteristics(up to twenty years after inclusion)
- Characterisation of respiratory function with pulmonary function test and thoracic tomodensitometry(up to twenty years after inclusion)
- Follow up of diaphragmatic failure with pulmonary function test and thoracic tomodensitometry(up to twenty years after inclusion)
研究者
Joe Elie Salem
Clinical Professor
Groupe Hospitalier Pitie-Salpetriere