Clinical trial NCT03538262: Assessing Tele-Health Outcomes in Multiyear Extensions of PD Trials
Official title: Tele-health Outcomes as Digital Biomarkers of Parkinson's Disease Progression During Extended Follow up of STEADY-PD3 and SURE-PD3 Trial Participants
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Parkinson Disease
Keywords: SURE-PD3
STEADY-PD III
Parkinson disease
PD
Parkinson Study Group (PSG)
Telemedicine
Smartphone
Disease progression
Televisits
Patient-reported outcomes
Lead sponsor: Massachusetts General Hospital
Sponsor class: OTHER
Collaborators: name: Northwestern University
name: Sage Bionetworks
name: University of Rochester
name: National Institute of Neurological Disorders and Stroke (NINDS)
name: Michael J. Fox Foundation for Parkinson's Research
name: The Parkinson Study Group
First posted: 2018-05-29
Enrollment: 226
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: All individuals with early idiopathic PD enrolled in the STEADY-PD3 (a.k.a. STEADY-PD III; NCT02168842) and SURE-PD3 (NCT02642393) studies.

Brief summary: An observational study to characterize and compare long-term clinical outcomes data collected remotely through periodic tele-visits, interactive smartphone app sessions, and web-based surveys in individuals with Parkinson's Disease (PD) who have completed the interventional phases of the STEADY-PD3 and SURE-PD3 clinical trials.

Detailed description: Telemedicine and smartphone-based remote sensor assessments represent particularly promising opportunities to streamline study conduct, reduce participant burden, and allow for the collection of data beyond the usual episodic, in-clinic assessments. Demonstrating the utility of these relatively inexpensive accessible platforms for the measurement of PD progression would also establish the infrastructure for long-term follow up of participants after completion of interventional studies.

STEADY-PD3 (a.k.a. STEADY-PD III) and SURE-PD3 were both designed as phase 3 trials of potential disease-modifying interventions in PD. In both studies, de novo PD participants were randomized in 1:1 allocation to active therapy versus placebo with longitudinal follow up for three and two years, respectively. Both studies include collection of DNA and plasma samples, which along with rigorously collected clinical data will become part of the Parkinson's Disease Biomarker Program (PDBP), providing valuable resources for biomarker development. Long-term observation of participants from these two trials, together comprising \~600 early PD subjects, is invaluable not only in characterizing any persistent or delayed benefits of either randomized treatment but also in the development of tele-health outcomes to facilitate future interventional trials in PD and of neurotherapeutics more broadly.

Challenges of traditional long-term follow up of large cohorts are the high cost of in-clinic assessment, the high dropout rates and the need to maintain multi-site infrastructure. The objective of this study is to leverage modern technology to develop, pilot and implement a 100% virtual model for long-term follow up utilizing telemedicine and smartphone platforms for quantitative monitoring of clinician- and patient-reported outcomes (PROs). This cohort may also serve to test feasibility of new technology platforms as they become available.

Eligibility criteria: Inclusion Criteria:

1. Enrollment in STEADY-PD3 or SURE-PD3 studies
2. Prior consent to be contacted by the University of Rochester (UR) or if a participant from STEADY-PD III or SURE-PD3 studies directly contacts UR to request information about study participation
3. Internet-enabled device that will support participation in tele-visits
4. Have created or willing to create a Global Unique Identifier (GUID)
5. Willing and able to provide informed consent
6. English fluency
7. For participants opting to participate in the smartphone component, possession of a suitable smartphone (iPhone or Android) with adequate data plan and cellular network access/signal or wifi access

Exclusion Criteria:

1. Inability to carry out study activities as determined by study staff

Primary outcomes:
measure: Change in Tele-visit Modified MDS-UPDRS Parts 1-3 (Total Score)
timeFrame: Two years (0, 12, and 24 months)
measure: Change in Tele-visit MDS-UPDRS Part 2 (Score)
timeFrame: Two years (0, 12, and 24 months)
measure: Change in Smartphone Tapping (Score)
timeFrame: Two years (0, 3, 6, 9, 12 , 15, 18, 21, and 24 months)
measure: Change in Fox Insight MDS-UPDRS Part 2 (Score)
timeFrame: Two years (0, 6, 12, 18, and 24 months)
Secondary outcomes:
measure: Change in Tele-visit MDS-UPDRS Part 1a (Score)
timeFrame: Two years (0, 12, and 24 months)
measure: Change in Tele-visit MDS-UPDRS Part 1b (Score)
timeFrame: Two years (0, 12, and 24 months)
measure: Change in Tele-visit Modified MDS-UPDRS Part 3 (Score)
timeFrame: Two years (0, 12, and 24 months)
measure: Change in Tele-visit Montreal Cognitive Assessment (MoCA; Score)
timeFrame: Two years (0, 12, and 24 months)
measure: Change in Tele-visit Schwab and England (S&E; Score)
timeFrame: Two years (0, 12, and 24 months)
measure: Change in Tele-visit Clinician Global Impression - Severity (CGIS; Score)
timeFrame: Two years (0, 12, and 24 months)
measure: Change in Fox Insight Parkinson's Disease Questionnaire - 8 Item Version (PDQ-8; Score)
timeFrame: Two years (0, 3, 6, 9, 12, 15, 18, 21, and 24 months)
measure: Change in Fox Insight EuroQol Five Dimensions Questionnaire (EQ-5D; Score)
timeFrame: Two years (0, 6, 12, 18, and 24 months)
measure: Change in Fox Insight Non-motor Symptoms Questionnaire (NMS-QUEST; Score)
timeFrame: Two years (0, 3, 6, 9, 12, 15, 18, 21, and 24 months)
measure: Change in Fox Insight REM (Rapid Eye Movement) Behavior Disorder (RBD; Score)
timeFrame: Two years (0, 3, 6, 9, 12, 15, 18, 21, and 24 months)
measure: Change in Fox Insight Parkinson's Daily Activities Questionnaire -15 (PDAQ-15; Score)
timeFrame: Two years (0, 3, 9, 15, and 21 months)
measure: Change in Fox Insight Geriatric Depression Scale (GDS; Score)
timeFrame: Two years (0, 12, and 24 months)
Other outcomes:
Locations:
city: Rochester
country: United States
facility: CHeT Telemedicine (Site 363)
state: New York
Study officials:
affiliation: Massachusetts General Hospital
name: Michael A Schwarzschild, MD, PhD
role: PRINCIPAL_INVESTIGATOR
affiliation: Northwestern University
name: Tanya Simuni, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: University of Rochester
name: E. Ray Dorsey, MD, MBA
role: PRINCIPAL_INVESTIGATOR
affiliation: Sage Bionetworks
name: Larsson Omberg, PhD
role: PRINCIPAL_INVESTIGATOR
References: