1. PPMI Beyond 2018

2. Study Goals beyond 2018
Continue to follow original cohort focusing on long-term outcomes
Cognition
Falls
Functional status
Fully enroll and follow genetic cohorts
Possibly expand prodromal cohort
Pursue sub-studies when there are good opportunities
Other biologicals
Wearables and other new clinical assessment technologies

3. PPMI Amendment 13 Extend visits through 2023
Subjects will now have 5-13 years of follow-up dependent on cohort
Modified schedule of activities for visits post 2019
Genetic Cohort and Prodromal (RBD & hyposmics) subjects to complete 5 years of visits on current SOA and then switch to modified SOA following month 60 visit

4. Modified Schedule of Activities
Annual in person visits
No imaging
Lumbar puncture every other year
New assessments:
Determination of Falls
NeuroQOL
Boston Naming Test
Trails A & B
Lexical fluency

5. Modified Schedule of Activities
Phone calls at 6 month point in between annual visits
Confirmation of vital status
Update contact information
Clinical Diagnosis review

6. Phenoconversion visits
If a subject is diagnosed with PD- a phenoconversion visit (PV) should be performed
PV visit is similar to an annual visit in the modified SOA
If it is determined a subject has phenoconverted between annual visits, subject should be brought in within 45 days of site’s awareness for a PV visit

7. Other Changes in Amendment 13
Healthy Control cohort-change in SOA: month 72 and 96 now in person visits
Visits with Genetic Registry subjects will be discontinued with Genetic Registry subjects continuing follow up through FOUND in PPMI
Sites will complete one final phone visit with Genetic Registry subjects by phone before discontinuing visits

8. Enrollment in Genetic cohorts
Current
LRRK2
affected
141
unaffected
190
GBA 73
129
Alpha-synuclein 19 6
Recruitment goal is 300 patients with a genetic mutation and 300 unaffected carriers

9. Publicly available data
Analysis
Principal Investigator
Matrix
Cohort(s)
Visit(s)
NeuroX genotyping
Andrew Singleton, PhD
DNA
Original BL
Immunochip genotyping
Whole exome sequencing
CSF total aSyn
(Covance/BioLegend immunoassay)
Peggy Taylor, PhD
CSF
BL, 3 mo, 6 mo, and 12 mo
CSF Aβ, tau, and p-tau181
(Innogenetics INNO-BIA AlzBio3 immunoassay)
Les Shaw, PhD
αSyn transcripts
Clemens Scherzer, MD
RNA
PD and HC
ApoA1 and EGF
Alice Chen-Plotkin, MD, MSc
Plasma
BL, 6 mo, and 12 mo
IGF-1
Maria Teresa Pellecchia, PhD
Serum
mRNA transcripts
Judith Potashkin, PhD
SNCA genotyping
Matthew Farrer, PhD
Martin Rabey
BL, 12 mo, 24 mo, and 36 mo

10. Ancillary study update
Type/Description
Status
Florbetaben PET imaging
Imaging
Ongoing data collection
VMAT-2 imaging
Resting state MRI
Gait device
Motor
FOUND
Customizable remote follow-up of participants
Ongoing enrollment; anticipated increased activity in 2018
iPSC collection
Biologic
Ongoing enrollment; samples available for request

11. Wearables: Key Goals Assess prodromal PD cohorts Assess progression
Assess drug response
Comparison with in-clinic assessments

12. Wearable Plans for 2018 and beyond
Verily
Watch and bed sensor
Passive collection of Data
US based
Shared data
Roche
Phone App
Passive and Active collection of Data
EU based
Shared Data

13. Study Goals for 2018 and beyond
Summary:
Study Goals for 2018 and beyond
Assess long-term PD progression outcomes focusing on:
Progression of identified PD subsets
Dopa non-responsive milestones
Assess predictability of biomarkers identified at earlier stage
Continued acquisition of fluid /imaging biomarkers
Follow up of new cohorts
Comparison of PD and Genetic cohorts
Assess progression during prodromal PD
Integration of new markers