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

总结 目前的临床研究方法需要大量的研究基础设施和多个临床站点来招募 有足够的参与者。其结果是研究是昂贵的，旷日持久的，可变的， 给参与者带来负担。这种方法的局限性是众所周知的，并且在当前的情况下是不可避免的。 研究方法我们的目标是建立，扩大和评估虚拟访问的使用，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、敏捷， 无论地理位置、疾病负担或旅行能力如何，个人都可以参加。 我们将与个人基因组学公司（23andMe）合作，进行为期36个月的虚拟研究。 研究并建立全国范围内无明显PD和有明显PD的LRRK2携带者队列（n=350） （n=50）。LRRK2突变是PD的最常见的常染色体显性病因，LRRK2是一种有前途的突变。 作为疾病改善治疗剂的靶点。我们将通过以下方式对所有400个LRRK 2载体进行前瞻性表征： 虚拟研究访问与远程专家评估从一个单一的研究网站。此外，我们预计， 我们将能够成功地招募和留住目标数量的参与者，我们将达到 在先前的LRRK2队列中没有代表的个体。除了展示 该方法的优点和评估研究特定的假设，由此产生的大型队列良好， 特征化和参与的LRRK2载体将作为建立PD预防的资源， 干预试验。 实施临床研究虚拟访视和建立PD虚拟队列将使 通过减少参与障碍，让更多的人参与到临床研究中来， Udall中心从事LRRK2研究，并作为未来研究的模型，包括观察和 介入性的

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.