Performance of Serum Neurofilament Light Chain in a Wide Spectrum of Clinical Courses of Amyotrophic Lateral Sclerosis - a Cross-sectional and Longitudinal Multicenter Study
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 3,000
- 试验地点
- 19
- 主要终点
- correlation of serum neurofilament light chain with ALS phenotypes
研究概览
简要总结
This study assesses the performance of serum neurofilament light chain (sNfL) in amyotrophic lateral sclerosis (ALS) in a wide range of disease courses, in terms of ALS progression, disease duration, and tracheostomy invasive ventilation (TIV). The aim of the research project is to investigate the correlation between NfL serum concentration and the natural course of the disease, the ALS progression rate, and specific phenotypes of ALS. Furthermore, the performance of NfL as a therapeutic biomarker will be studied. A systematic analysis of the NfL serum concentration in a cohort of 3,000 ALS patients using the Single Molecule Analysis method (SIMOA) will be performed. This analysis is carried out as a multi-center study.
详细描述
The aim of this study is to investigate the correlation between the NfL serum concentration and the natural course of the disease, the ALS progression rate as measured by the ALS functional rating scale (ALSFRS-R), and specific phenotypes of ALS. The results of the study will contribute to the assessment of disease progression and the prognosis making of ALS. Furthermore, the performance of NfL as a therapeutic marker of ALS medicines and non-pharmacologic treatment options will be investigated. A systematic analysis of the NfL serum concentration in an extended cohort of ALS patients using the Single Molecule Analysis method (SIMOA) will be performed.
Research objectives comprise:
- Correlation of NfL with disease progression, including duration of ALS disease
- Correlation of NfL with the course of ALS (classic ALS or variants in the motor neuron involvement or the regional propagation patterns)
- Correlation of NfL with the progression rate of ALS
Cohorts on phenotypic variants:
The clinical phenotype of ALS will be differentiated according to the motor neuron involvement or regional propagation patterns of disease onset and clinical course.
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
- •Diagnosis of amyotrophic lateral sclerosis including specific forms
- •Patient's informed consent to participate in this study
- •Minimum age of 18 years
- •Willingness for blood collection
排除标准
- •Unwillingness to store and share pseudonymized medical data collected in the study
- •Evaluation by the investigator, which excludes participation
结局指标
主要结局
correlation of serum neurofilament light chain with ALS phenotypes
时间窗: 2020-2024
correlation of serum neurofilament light chain with ALS phenotypes in terms of type of onset and clinical variants including progressive muscle atrophy, primary lateral sclerosis, flail-arm syndrome, flail-leg syndrome and other phenotypes
correlation of serum neurofilament light chain with ALS treatment options
时间窗: 2020-2024
correlation of serum neurofilament light chain to ALS interventions such as treatment with tofersen and other medicines
correlation of serum neurofilament light chain to ALS progression
时间窗: 2020-2024
correlation of serum neurofilament light chain (NfL) with the ALS progression rate as measured by the revised form of the ALS function rating scale (ALSFRS-R)
次要结局
- correlation of serum neurofilament light chain with non-pharmacologic ALS interventions(2022-2024)
研究者
Thomas Meyer, MD
Prof. Dr. Thomas Meyer
Charite University, Berlin, Germany