Developing a Comprehensive Biomarker Panel for Monitoring Progression and Early Detection in ALS Patients
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 入组人数
- 200
- 试验地点
- 2
- 主要终点
- Development of a biomarker panel for diagnosis, prognosis, and patient stratification in ALS
研究概览
简要总结
Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease for which reliable biomarkers for early diagnosis, prognosis, and patient stratification remain limited. Previous genetic, proteomic, imaging, and electrophysiological studies have identified potential biomarkers and phenotype modifiers, improving the understanding of motor neuron degeneration mechanisms. However, these findings have not yet been translated into a clinically useful biomarker algorithm. This observational study aims to develop a biomarker panel to support the diagnosis, prognosis, and stratification of patients with ALS. Clinical and molecular biomarkers previously associated with ALS phenotypes will be analyzed simultaneously and integrated into a multivariable predictive model. Clinical data and biological samples will be collected and analyzed to identify combinations of biomarkers associated with ALS phenotypes.
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
- •Age greater than 18 years.
- •Male and female patients with ALS diagnosed according to the El Escorial diagnostic criteria.
- •Sporadic or familial ALS cases.
- •Spinal-onset or bulbar-onset ALS cases.
排除标准
- •Refusal to participate.
- •Individuals deprived of liberty (Article L1121-6), including those subject to judicial or administrative decisions or involuntary hospitalization.
- •Adults under legal protection (guardianship, curatorship, or judicial protection measures) (Article L1121-8).
- •Individuals not affiliated with, or not beneficiaries of, a French social security scheme (Article L1121-8-1).
- •Individuals participating in another research study with an ongoing exclusion period (Article L1121-12).
结局指标
主要结局
Development of a biomarker panel for diagnosis, prognosis, and patient stratification in ALS
时间窗: From baseline to 12 months
A multivariable biomarker panel integrating clinical variables, genetic variants associated with ALS survival, and serum protein and immunological biomarkers will be evaluated. Biomarkers include cytokines, neurofilament light chain (NF-L), GFAP, phosphorylated TDP-43, TDP-43, total Tau, phosphorylated Tau, and UCHL1, together with genotyping of ALS-associated survival variants. The panel will be assessed for its ability to support prognosis and patient stratification in amyotrophic lateral sclerosis (ALS).
次要结局
未报告次要终点