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Abstract Details

A Virtual Cohort Study of LRRK2 G2019S Carriers: A Novel Model for Long-Term Observation
Movement Disorders
P2 - Poster Session 2 (5:30 PM-6:30 PM)
10-012
The objective of this study is to establish the feasibility of engaging, following, and prospectively characterizing a nationwide cohort of 23andMe-identified LRRK2 G2019S carriers with and without manifest Parkinson’s disease (PD).  We will 1) assess our ability to recruit and retain a national cohort into a 36-month virtual study, 2) prospectively characterize the cohort and compare its demographics and disease characteristics to those of traditionally-established LRRK2 G2019S cohorts, and 3) assess the value of this model in creating a clinical trial-ready cohort.
LRRK2 is a promising target for the development of disease-modifying therapeutics for PD but new clinical research methods are needed to speed development.  The traditional multi-center research study is dependent upon a massive infrastructure, requires a huge investment of resources, and imposes barriers to participation.  Virtual studies use technology to conduct remote assessments and may improve efficiency, reduce costs, and reduce barriers to participation. Collaboration with the direct-to-consumer personal genomics company, 23andMe, which has genotyped over 3 million individuals, will allow us to move beyond traditional site-based recruitment to reach a larger number of individuals. The characterization and engagement of a large, geographically-dispersed cohort could address critical gaps in knowledge regarding LRRK2 that will inform the design of clinical trials. 

We will enroll 300 23andMe-identified LRRK2 G2019S carriers (250 without PD, 50 with PD) in this 36-month virtual, prospective, observational study.  Participants will complete annual virtual research visits conducted by a centralized research team.  Assessments will include standard patient-reported outcome measures and clinician-reported outcomes measures (modified for remote assessment).

 

This study is funded under the University of Rochester Udall Center (NINDS

1P50NS108676-01) and will launch in the first quarter of 2019.  

 
We will use novel research methods to prospectively characterize and engage a large cohort of LRRK2 G2019S carriers.
Authors/Disclosures
Ruth Schneider, MD, FAAN (University of Rochester)
PRESENTER 		Dr. Schneider has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Escape Bio. The institution of Dr. Schneider has received research support from The Michael J. Fox Foundation. The institution of Dr. Schneider has received research support from NIH. The institution of Dr. Schneider has received research support from Acadia Pharmaceuticals. The institution of Dr. Schneider has received research support from CHDI. The institution of Dr. Schneider has received research support from Biohaven Pharmaceuticals. The institution of Dr. Schneider has received research support from Parkinson Study Group. The institution of Dr. Schneider has received research support from The Michael J. Fox Foundation. The institution of Dr. Schneider has received research support from Bial. Dr. Schneider has received personal compensation in the range of $500-$4,999 for serving as a Telemedicine Evaluator with Parkinson's Foundation.
No disclosure on file
E. Ray Dorsey, MD, MBA (University of Rochester) Dr. Dorsey has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Seelos Therapeutics. Dr. Dorsey has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Eden McCallum, LLP. Dr. Dorsey has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Genentech/Roche. Dr. Dorsey has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biohaven Pharmaceuticals. Dr. Dorsey has received personal compensation in the range of $500-$4,999 for serving as a Consultant for WebMD. Dr. Dorsey has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for BioSensics. Dr. Dorsey has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for Mediflix. Dr. Dorsey has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Cerevance. Dr. Dorsey has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Kairos Cinema, LLC. Dr. Dorsey has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Mitsubishi Tanabe Pharma America Inc.. Dr. Dorsey has received personal compensation in the range of $500-$4,999 for serving as a Consultant for HanAll. Dr. Dorsey has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Novartis. Dr. Dorsey has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi. Dr. Dorsey has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Advarra. Dr. Dorsey has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Medrhythms. Dr. Dorsey has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for ANA. Dr. Dorsey has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for HMP 好色先生, LLP. Dr. Dorsey has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for ADIVO Associates France. Dr. Dorsey has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for University of Toronto. Dr. Dorsey has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Philadelphia Neurological Society. Dr. Dorsey has stock in Included Health. Dr. Dorsey has stock in Mediflix. Dr. Dorsey has stock in SemCap. Dr. Dorsey has stock in Synapticure. The institution of Dr. Dorsey has received research support from NIH/NINDS. The institution of Dr. Dorsey has received research support from Burroughs Wellcome . The institution of Dr. Dorsey has received research support from Michael J. Fox Foundation - Fox Insight. The institution of Dr. Dorsey has received research support from Michael J. Fox Foundation - Emerald. The institution of Dr. Dorsey has received research support from B. Thomas Golisano Foundation. The institution of Dr. Dorsey has received research support from Emerald Innovations, Inc.. The institution of Dr. Dorsey has received research support from Photopharmics Inc.. The institution of Dr. Dorsey has received research support from Averitas Pharma Inc.. The institution of Dr. Dorsey has received research support from DOD/Army Medical Research Acquisition Activity. The institution of Dr. Dorsey has received research support from NIH/NINDS.
Robert G. Holloway, MD, MPH, FAAN (University of Rochester Medical Center) Dr. Holloway has nothing to disclose.