Biomarkers for Fatigue in Patients with Myasthenia Gravis
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 115
- 试验地点
- 1
- 主要终点
- blood biomarker
研究概览
简要总结
Rationale: Myasthenia Gravis (MG) is a chronic autoimmune disease affecting the neuromuscular junction. Although a hallmark of MG is muscle fatigability due to dysfunction of the neuromuscular junction (peripheral fatigue), a large number of MG patients also report symptoms of central or cognitive fatigue. Central fatigue is defined as an experienced lack of energy, physically and/or mentally. In October 2019 we performed a cross-sectional survey study (P15.287) among 420 Dutch MG patients showing a clinically relevant central fatigue rate of 62% on the Checklist Individual Strength-Fatigue subscale (CIS-f). In this prior study, we identified a number of factors associated with fatigue, but these factors cannot fully explain the observed high prevalence of fatigue in MG and very little is known on its biological substrates and pathophysiology.
Objectives: The main objective is to investigate if there are biomarkers for fatigue in MG. The secondary objective is to investigate the muscle origin of these biomarkers.
Study design: This study will be an exploratory study, subdivided in part I and part II (optional).
Study population: Patients with Myasthenia Gravis recruited after previous participation in the cross-sectional fatigue survey study (P15.287).
Intervention:
Phase I:
Visit 1, time: 0
- blood withdrawal
- muscle biopsy (not mandatory for participation)
- QMG
- questionnaires on medication, disease severity, fatigue, depressive symptoms and physical condition
Phase II (optional):
Visit 2, time: 6-12 months after visit 1, after analysis of phase I results.
- blood withdrawal (similar analysis as phase I)
- QMG
- questionnaires on medication, disease severity, fatigue, depressive symptoms and physical condition
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
- •Male of female patients aged ≥ 18 years
- •Subjects must understand the requirements of the study and provide written informed consent.
- •Diagnosis of MG:
- •Clinical signs or symptoms characteristic for MG and
- •A positive serologic test for AChR antibodies
排除标准
- •A medical history of other active auto-immune disorders for which the patient currently receives a medical treatment, such as thyroid disease or rheumatoid arthritis.
- •A medical history of neoplasms within the last year.
- •Substance abuse.
- •Exclusion criteria for muscle biopsy
- •The use of anticoagulants therapy.
- •Allergy for lidocaine
结局指标
主要结局
blood biomarker
时间窗: 2022
To investigate whether there is a potential biomarker, which corresponds with fatigue in MG, among the biomarkers that were previously identified in other disorders. For this objective we aim to determine and compare the diagnostic yield of several serum biomarkers which were (possibly) associated with central fatigue in other disorders in previous studies.
次要结局
- Brain muscle axis(2022-2023)
研究者
Martijn R. Tannemaat, MD PhD
MD PhD
Leiden University Medical Center