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.
