Clinical Core

临床核心

• 批准号: 9802927
• 负责人: Bradley F Boeve
• 金额: $ 786.26万
• 依托单位: MAYO CLINIC ROCHESTER
• 依托单位国家: 美国
• 财政年份: 2019
• 资助国家: 美国
• 起止时间: 2019-09-15 至
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/9802927
• 关键词: Address; Advance Directives; Affect; Alzheimer' s Disease; Alzheimer' s disease related dementia; Autopsy; Basic Science; Biological Markers; C9ORF72; California; Categories; Clinical; Clinical Data; Clinical Sciences; Clinical Trials; Clinical Trials Network; Communities; DNA; Data; Data Set; Development; Diagnosis; Disease; Disease Progression; Enrollment; Ensure; Evaluation; Family member; Fostering; Frontotemporal Dementia; Frontotemporal Lobar Degenerations; Funding; Genes; Goals; Heterogeneity; Impairment; Indiana; Infrastructure; International; Laboratories; MRI Scans; Messenger RNA; Mutation; National Institute of Neurological Disorders and Stroke; Natural History; Neuropsychology; North America; Participant; Patient Recruitments; Patients; Periodicity; Peripheral Blood Mononuclear Cell; Phenotype; Plasma; Population; Preparation; Procedures; Process; Protocols documentation; Quality Control; Questionnaires; Recommendation; Registries; Research; Research Personnel; Research Priority; Sampling; Scanning; Site; Standardization; Uncertainty; Universities; Update; Washington; arm; cohort; data sharing; design; genotyped patients; kindred; member; neuroimaging; programs; recruit; repository; success; symposium; tool

ABSTRACT – ARTFL LEFFTDS Longitudinal FTLD: CLINICAL CORE The ARTFL LEFFTDS Longitudinal Frontotemporal Lobar Degeneration (ALLFTD) protocol represents an expanded and integrated international research consortium involving seven cores and two projects; ALLFTD is designed to substantially and comprehensively increase the amount of longitudinal data, numbers of biospecimen samples and MRI scans that are publicly available for research in FTLD, to ultimately foster development of disease-modifying therapies. Previously, the Advancement of Research and Treatment in Frontotemporal Lobar Degeneration (ARTFL; U54 NS092089) and Longitudinal Evaluation of Familial Frontotemporal Dementia Subjects (LEFFTDS; U01 AG045390) protocols collectively enrolled >1100 participants at 18 sites, including >380 members of families affected by MAPT, GRN and C9orf72 mutations (surpassing the original LEFFTDS target of 300), with the remaining participants being sporadic FTLD patients enrolled through ARTFL. Detailed clinical and neuropsychological data, including (but not limited to) the National Alzheimer's Coordinating Center (NACC) Uniform Data Set Version 3 and FTLD Module has been completed, with almost 1200 data packets uploaded to NACC to date. Biofluid samples, including DNA, PBMC, mRNA, plasma and serum on the vast majority of participants, and CSF on a subset, have been collected during the ARTFL/LEFFTDS studies, transferred and processed at the National Centralized Repository for Alzheimer's Disease and Related Dementias. Over 900 MRI scans using a standardized protocol have been performed in 585 participants, and these data have undergone quality control inspection and transferal to the Laboratory of NeuroImaging. All data, samples and scans are available and have been used by investigators within and outside of the ARTFL/LEFFTDS Consortium. The ALLFTD Clinical Core will continue to build on the obvious success of the ARTFL and LEFFTDS protocols by recruiting additional participants in either the longitudinal or biofluid-focused arm. The Clinical Core will enroll and evaluate up to 1100 participants annually in the longitudinal arm, and will include 500 asymptomatic and mildly symptomatic members of kindreds with familial FTLD and 600 mildly symptomatic sporadic FTLD patients. Approximately 1000 additional FTLD patients will be recruited into the biofluid focused arm. In partnership with the FTD Disorders Registry, participants in both arms will be followed remotely, requested to complete questionnaires and provided with periodic updates and information of FTLD research opportunities. The sites in the Clinical Core will provide high quality clinical data, biofluid samples and MRI scans to be integrated for use in all cores and projects, and shared with the scientific community.

摘要 – ARTFL LEFFTDS 纵向 FTLD：临床核心 ARTFL LEFFTDS 纵向额颞叶变性 (ALLFTD) 协议代表 扩大和整合国际研究联盟，涉及七个核心和两个项目； ALLFTD 是 旨在大幅全面增加纵向数据量、 生物样本样本和 MRI 扫描可公开用于 FTLD 研究，最终促进 疾病修饰疗法的开发。此前，研究和治疗的进展 额颞叶变性（ARTFL；U54 NS092089）和家族性的纵向评估 额颞叶痴呆受试者 (LEFFTDS; U01 AG045390) 协议总共登记 >1100 名 18 个地点的参与者，包括超过 380 名受 MAPT、GRN 和 C9orf72 突变影响的家庭成员 （超过 LEFFTDS 最初的 300 名目标），其余参与者为零星 FTLD 患者 通过 ARTFL 注册。详细的临床和神经心理学数据，包括（但不限于） 国家阿尔茨海默病协调中心 (NACC) 统一数据集版本 3 和 FTLD 模块已 已完成，迄今为止已向 NACC 上传了近 1200 个数据包。生物流体样品，包括 DNA、PBMC、 已收集了绝大多数参与者的 mRNA、血浆和血清以及一小部分参与者的脑脊液 在 ARTFL/LEFFTDS 研究期间，在国家中央存储库中传输和处理 阿尔茨海默病和相关痴呆症。使用标准化协议进行了 900 多次 MRI 扫描 在 585 名参与者中进行，这些数据已经过质量控制检查并转移到 神经影像实验室。所有数据、样本和扫描均可用并已被研究人员使用 ARTFL/LEFFTDS 联盟内部和外部。 ALLFTD 临床核心将继续建立在 通过招募更多参与者，ARTFL 和 LEFFTDS 协议取得了明显的成功 纵向或生物流体聚焦臂。临床核心每年将招募和评估多达 1100 名参与者 在纵向臂中，将包括 500 名无症状和轻度症状的亲属成员 家族性 FTLD 和 600 名轻度症状散发性 FTLD 患者。大约 1000 个额外 FTLD 患者将被招募到生物流体重点组。与 FTD 疾病登记处合作， 两组参与者都将受到远程跟踪，要求填写调查问卷并提供 FTLD 研究机会的定期更新和信息。临床核心中的站点将提供 高质量的临床数据、生物流体样本和 MRI 扫描将被集成以用于所有核心和项目，以及 与科学界共享。