Pilot Demonstration

试点示范

• 批准号: 8929489
• 负责人: Michael Benatar
• 金额: $ 6.31万
• 依托单位: UNIVERSITY OF MIAMI SCHOOL OF MEDICINE
• 依托单位国家: 美国
• 财政年份: 2014
• 资助国家: 美国
• 起止时间: 2014-09-30 至
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/8929489
• 关键词: Advanced Development; Alzheimer' s Disease; Amyotrophic Lateral Sclerosis; Biological; Biological Assay; Biological Markers; Biology; Blood; Blood specimen; Cerebrospinal Fluid; Cerebrospinal Fluid Proteins; Clinical Research; Collection; Communities; Cross-Sectional Studies; Data; Development; Diagnosis; Disease; Disease Progression; Electromyography; Evaluation; Extramural Activities; Functional RNA; Functional disorder; Funding; Future; Genetic Risk; Goals; Grant; Laboratories; Magnetic Resonance Imaging; Monitor; Motor; Motor Neurons; Myography; NGFR Protein; Neurologic Examination; Patients; Pilot Projects; Process; Publishing; RNA; Request for Applications; Research; Research Design; Research Infrastructure; Research Personnel; Research Project Grants; Resources; Saliva; Sample Size; Sampling; Science; Scientist; Staging; Suggestion; Testing; Therapeutic Agents; Urine; Validation; Work; base; cohort; deep sequencing; diagnostic accuracy; disorder risk; electric impedance; extracellular; follow-up; improved; member; neuroimaging; novel; outcome forecast; programs; repository; response; success; therapeutic development; urinary

PILOT/DEMONSTRATION PROJECTS The primary purpose of the CREATE RDCRC pilot/demonstration research program is to promote biomarker discovery and validation for ALS and related disorders through a small research grant program that funds studies of potential biomarkers during their early stages of development. Priority is given to projects with the most promising clinical research potential. The activities of the CREATE Consortium - longitudinal evaluation of patients with familial and sporadic disease, collection of deep phenotypic data, and the establishment of a repository of biological samples (blood, urine, cerebrospinal fluid) that has been annotated clinically in a detailed manner - are all expected to enhance and facilitate biomarker discovery through the pilot/ demonstration research program. The first pilot/demonstration project will focus on early development of a cerebrospinal fluid biomarker for ALS. Potential projects in subsequent years include development of a urinary biomarker of disease progression and a neuroimaging marker of the imbalance between cortical inhibition and excitability. The CREATE RDCRC will utilize a "request for applications" on an annual basis, to solicit applications and to select the most promising project for funding.

试点/示范项目 CREATE RDCRC试点/示范研究计划的主要目的是通过一个小型研究资助计划促进ALS和相关疾病的生物标志物发现和验证，该计划资助潜在生物标志物在其早期发展阶段的研究。优先考虑最有临床研究潜力的项目。CREATE联盟的活动-家族性和散发性疾病患者的纵向评估，深度表型数据的收集，以及已在临床上详细注释的生物样本（血液，尿液，脑脊液）的储存库的建立-都有望通过试点/示范研究计划增强和促进生物标志物的发现。第一个试点/示范项目将侧重于ALS脑脊液生物标志物的早期开发。未来几年的潜在项目包括开发疾病进展的尿生物标志物和皮质抑制和兴奋性之间不平衡的神经影像学标志物。CREATERDCRC将每年利用“申请请求”，征求申请并选择最有前途的项目供资。