MYOPROSP: A Prospective Cohort Study to Identify a Stratified Approach in the Diagnosis, Treatment and Delivery of Care in Adult Idiopathic Inflammatory Myopathy
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 300
- 试验地点
- 1
- 主要终点
- Number of participants with a significant change levels of diagnostic biomarkers.
研究概览
简要总结
Adult patients with suspected or confirmed idiopathic inflammatory myopathy (IIM) will be recruited. Patients will be approached, consented, have baseline demographics, diagnostics and disease activity measures recorded, and blood taken. The collection of data and biological material will mirror usual clinical practice as far as possible. Subjects will ideally attend further visits at 3, 6 and 12 months to have bloods taken, outcome measures recorded and questionnaires completed.
In addition, blood, muscle biopsies and imaging undertaken as part of usual care will also be collected for research purposes to measure a number of biomarkers for the assessment of diagnostic accuracy and clinical utility evaluation. As per usual practice, a muscle biopsy will be performed at baseline, and a further biopsy offered at 6 months to assess treatment response. A magnetic resonance (MR) muscle protocol will also be performed as per usual clinical practice, and a gadolinium-enhanced MR heart scan offered. Both these scans will be repeated at 6 months. An existing electronic database entry system will be used for data entry and capture on an anonymised basis.
The study will thus be based around diagnostic evaluations and outcome measures to improve quality of care in IIM.
详细描述
The bioresource from this protocol, ethics application and future funding will comprise the following:
i) UKMYONET cross-sectional study ii) MYOPROSP inception cohort study iii) MYOACT novel therapies registry iv) UK neuromuscular biobank
Primary endpoint The primary study endpoint will be sensitivity to change of the biomarkers of interest.
Secondary endpoints
This will include 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
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Polymyositis
- •Dermatomyositis (including amyopathic)
- •Inclusion body myositis
- •Anti-synthetase syndrome
- •Myositis overlapping with another connective tissue disorder
- •Cancer-associated myositis
- •Immune-mediated necrotising myopathy including statin-induced myositis
- •Juvenile myositis persisting into adulthood
- •Fasciitis including eosinophilic myofasciitis
- •Vasculitis affecting muscle
- •Granulomatous myositis
- •Focal myositis
- •Orbital myositis
- •Suspected myositis under investigation
- •CTD features in association with a myositis specific / associated antibody
排除标准
- •Patients with disease duration >2 years
- •Patients < 18 years
- •Confirmed non-inflammatory myopathies
- •Myositis secondary to alcohol or drug abuse
- •Patients unwilling or unable to give consent
- •Patients with poor or no venous access
- •Patients where MR imaging is contraindicated (for MR substudy)
- •Study population description Patients referred to tertiary level UK myositis clinics
- •Sampling methods N/A, this is a prospective study of consecutive eligible patients
结局指标
主要结局
Number of participants with a significant change levels of diagnostic biomarkers.
时间窗: 5 years
This will depend on the specific biomarker/cytokine being measured
次要结局
- Number of participants with a 20% improvement in myositis specific disease activity measures from baseline(5 years)
- Differences in frequency of genetic variants associated with IIM and subtypes compared to population matched controls(5 years)
研究者
Hector Chinoy
Professor
University of Manchester