Research Project 2 will use remote assessments to engage large, novel populations of participants with Parkinson disease

研究项目 2 将使用远程评估来吸引大量新的帕金森病参与者

• 批准号: 10017342
• 负责人: ROBERT G. HOLLOWAY
• 金额: $ 21.02万
• 依托单位: UNIVERSITY OF ROCHESTER
• 依托单位国家: 美国
• 财政年份: 2018
• 资助国家: 美国
• 起止时间: 2018-09-30 至 2023-07-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10017342
• 关键词: Age; Ashkenazim; Characteristics; Clinical; Clinical Research; Clinical Trials; Cohort Studies; Databases; Development; Disease; Dominant Genetic Conditions; Enrollment; Exhibits; Family history of; Foundations; Future; Genomics; Geography; Human Resources; Individual; Infrastructure; Intervention; Intervention Trial; LRRK2 gene; Longitudinal Studies; Modeling; Modification; Mutation; Natural History; Parkinson Disease; Participant; Patient Self-Report; Penetrance; Persons; Population; Prevention trial; PubMed; Publishing; Research; Research Infrastructure; Research Methodology; Research Personnel; Research Project Grants; Resources; Science; Site; Technology; Therapeutic; Travel; Universities; Visit; burden of illness; clinical Diagnosis; clinical research site; cohort; cost; demographics; disease diagnosis; genetic risk factor; improved; interest; kinase inhibitor; nervous system disorder; novel; prospective; recruit; research study; therapeutic development; therapeutic target; virtual

SUMMARY Current clinical research methods require a massive research infrastructure and multiple clinical sites to recruit a sufficient number of participants. The result is research that is expensive, protracted, variable, and burdensome to participants. The limitations of this approach are well-recognized and unavoidable given current research methods. We aim to establish, expand and evaluate the use of virtual visits as a new model for national clinical research studies in Parkinson disease (PD). This approach is efficient, agile, and enables individuals to participate regardless of geography, disease burden, or ability to travel. We will collaborate with a personal genomics company (23andMe) to conduct a 36-month, virtual research study and establish a nationwide cohort of LRRK2 carriers without manifest PD (n=350) and with manifest PD (n=50). LRRK2 mutations are the most common autosomal dominant cause of PD and LRRK2 is a promising target for disease-modifying therapeutics. We will prospectively characterize all 400 LRRK2 carriers through virtual research visits with remote expert assessment from a single research site. Moreover, we anticipate that we will be able to successfully recruit and retain the targeted number of participants and that we will reach individuals who have not been represented in prior LRRK2 cohorts. In addition to demonstrating the advantages of the approach and evaluating study-specific hypotheses, the resulting large cohort of well- characterized and engaged LRRK2 carriers will serve as a resource for establishing PD prevention or intervention trials. Implementation of virtual visits for clinical research and the establishment of a PD virtual cohort will enable more people to participate in clinical research by reducing barriers to participation, provide a resource to other Udall centers engaged in LRRK2 research, and serve as a model for future studies, both observational and interventional.

摘要 目前的临床研究方法需要大规模的研究基础设施和多个临床站点来招募 有足够数量的参与者。其结果是昂贵、旷日持久、多变的研究，以及 对参与者来说是沉重的负担。这种方法的局限性是众所周知的，而且在当前情况下是不可避免的。 研究方法。我们的目标是建立、扩展和评估虚拟访问作为一种新的模式 帕金森病(PD)的国家临床研究。这种方法高效、灵活，并且能够 无论地域、疾病负担或旅行能力如何，个人都可以参与。 我们将与一家个人基因组学公司(23andMe)合作，进行为期36个月的虚拟研究 研究并建立全国范围的LRRK2携带者队列，无明显PD(n=350)和有明显PD (n=50)。LRRK2突变是帕金森病最常见的常染色体显性致病原因，LRRK2是一种很有前途的 疾病修正疗法的目标。我们将通过以下方式前瞻性地描述所有400名LRRK2携带者的特征 从单个研究站点进行远程专家评估的虚拟研究访问。此外，我们预计 我们将能够成功地招募和保留目标人数的参与者，我们将达到 在之前的LRRK2队列中没有代表的个人。除了展示 该方法的优点和评估特定研究的假设，结果是大量的井- 特化和参与的LRRK2携带者将作为建立帕金森病预防或 干预试验。 实施临床研究虚拟访问和建立PD虚拟队列将使 更多的人通过降低参与门槛来参与临床研究，为其他人提供资源 Udall中心从事LRRK2研究，并作为未来研究的模型，包括观察性和 干预性。