We are enrolling 23andMe-identified LRRK2 G2019 carriers with and without Parkinson’s disease in this 36-month virtual study. To date, we have sent out ~3800 invitations, complete 267 test visits, and completed 254 baseline visits. Enrollment in this study has not only continued, but also accelerated due to COVID-19. While the vast majority of research studies in PD have halted new enrollment, we have added 60 new research participants in the past two months. Virtual visits have been well received by participants and we are establishing a well-characterized, clinical trial-ready cohort. We expect to complete enrollment in June 2020.
The study team has endeavored to engage research participants in numerous ways. The lead study coordinators devised a name for the study. VALOR-PD stands for Virtual Assessment of LRRK2 carriers to Optimize Research of Parkinson’s Disease. We created a VALOR-PD logo and designed a VALOR-PD newsletter, which was launched in March 2020 and distributed to all enrolled participants. The newsletter unveiled the new study name and logo, provided news on study progress and updates, and featured profiles on Ms. Reni Winter-Evans, a member of the UR Udall Research Partner Advisory Board and Ms. Taylor Myers, the lead study coordinator. A follow-up newsletter will automatically be sent to participants approximately 6 months after each annual visit.
A live webinar will be held on June 16, 2020. Open to all enrolled participants, the webinar will cover study background, updates, and a Q&A. An invitation was sent to all enrolled participants on May 14th and we encouraged submitting questions in advance to ensure we address topics of interest. To date, 85 participants have registered and over 40 questions have been submitted.
The Journal of Parkinson Disease has accepted our paper, A virtual cohort study of individuals at genetic risk for Parkinson’s disease: study protocol and design, for publication.
Advances in genetics have led to the identification of disease populations that may be suitable for gene-targeted therapies. Additionally, the rise of direct-to-consumer genetic testing has enabled many individuals to learn of their possible increased risk for rare diseases, some of which may be suitable for gene-targeted therapies. However, recruiting a large and representative population for rare diseases or genetically defined sub-populations of common diseases is slow, difficult, and expensive.
Virtual observational studies can characterize the natural history of genetically defined populations over large geographic areas. If successful, this single-site study could serve as a new model for developing a well-characterized cohort ready for participation in future clinical trials of gene-directed therapies.