A MULTICENTER STUDY FOR THE VALIDATION OF ALS BIOMARKERS

验证 ALS 生物标志物的多中心研究

• 批准号: 8166934
• 负责人: TAHSEEN MOZAFFAR
• 金额: $ 0.03万
• 依托单位: UNIVERSITY OF CALIFORNIA, SAN DIEGO
• 依托单位国家: 美国
• 财政年份: 2009
• 资助国家: 美国
• 起止时间: 2009-12-01 至 2010-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8166934
• 关键词: Age; Amyotrophic Lateral Sclerosis; Biological Markers; Blood; Blood specimen; Clinical Trials; Computer Retrieval of Information on Scientific Projects Database; Detection; Development; Diagnostic; Disease; Disease Progression; Early treatment; Funding; Gender; Grant; Individual; Institution; Intervention; Laboratories; Lead; Monitor; Multicenter Studies; Myopathy; Neurologic; Neurologist; Pathogenesis; Patients; Primary Care Physician; Radiculopathy; Research; Research Personnel; Resources; Sampling; Site; Source; Specialist; Specificity; Symptoms; Testing; Treatment Efficacy; United States; United States National Institutes of Health; Validation; medical specialties; therapy development; volunteer

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. The primary objective is to identify factors that contribute to the pathogenesis of amyotrophic lateral sclerosis (ALS). Development of disease biomarkers and diagnostic laboratory tests would facilitate earlier treatment intervention, help monitor treatment efficacy; and, ultimately, lead to the identification of targets that could be used in therapy development. Specific aim 1 is to develop a laboratory test that can be used by primary care physicians, neurologists and other clinicians to provide guidance with regard to something to worry about (possible ALS). It would provide benefit in generating appropriate referrals to neurological specialists early in disease progression. False negatives could be tolerated but false positives cannot (i.e. high specificity required with acceptable sensitivity). The test will identify people with ALS and distinguish ALS from disorders that present with similar symptoms (e.g. carpal tunnel, radiculopathies, myopathies). A test with high sensitivity for ALS can be very useful for specialty neurologist as well for early and accurate detection for inclusion in clinical trials. Blood and CSF samples will be collected at multiple sites representing the four regions of the United States from age and gender matched healthy individuals, disease mimics, and ALS patients. Specific aim 2 is to assess how biomarkers discovered in aim 1 change with disease course. Blood samples will be collected longitudinally from ALS and suspected ALS volunteers.

这个子项目是许多研究子项目中的一个 由NIH/NCRR资助的中心赠款提供的资源。子项目和 研究者（PI）可能从另一个NIH来源获得了主要资金， 因此可以在其他CRISP条目中表示。所列机构为 研究中心，而研究中心不一定是研究者所在的机构。 主要目的是确定导致肌萎缩侧索硬化症（ALS）发病的因素。 疾病生物标志物和诊断实验室检测的发展将促进早期治疗干预，帮助监测治疗效果;并最终导致可用于治疗开发的目标的识别。 具体目标1是开发一种实验室测试，可供初级保健医生，神经学家和其他临床医生使用，以提供有关担心（可能的ALS）的指导。它将有助于在疾病进展早期向神经科专家提供适当的转诊。 可以容忍假阴性，但不能容忍假阳性（即需要高特异性和可接受的灵敏度）。 该测试将识别ALS患者，并将ALS与具有类似症状的疾病（例如腕管，神经根病，肌病）区分开来。 ALS的高灵敏度测试对于专业神经科医生以及临床试验中早期和准确的检测非常有用。将在代表美国四个地区的多个研究中心从年龄和性别匹配的健康个体、疾病模拟者和ALS患者中采集血液和CSF样本。 具体目标2是评估目标1中发现的生物标志物如何随病程变化。将从ALS和疑似ALS志愿者中纵向采集血样。