AT-HOME PD: Assessing Tele-Health Outcomes in Multiyear Extensions of PD Trials

Sponsor

Massachusetts General Hospital (Other)

Overall Status

Completed

CT.gov ID

NCT03538262

Collaborator

Northwestern University (Other), Sage Bionetworks (Other), University of Rochester (Other), National Institute of Neurological Disorders and Stroke (NINDS) (NIH), Michael J. Fox Foundation for Parkinson's Research (Other), The Parkinson Study Group (Other)

270

Enrollment

Location

41.6

Actual Duration (Months)

6.5

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Parkinson Disease

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.

Study Design

Study Type:

Observational

Actual Enrollment :

270 participants

Observational Model:

Cohort

Time Perspective:

Prospective

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

Actual Study Start Date :

Oct 1, 2018

Actual Primary Completion Date :

Mar 21, 2022

Actual Study Completion Date :

Mar 21, 2022

Arms and Interventions

Arm	Intervention/Treatment
former phase 3 PD trial participants The AT-HOME PD cohort enrolled upon completion of STEADY-PD3 or during completion of SURE-PD3; enrolling 2 to 6 years after diagnosis, and on standard dopaminergic therapy for 0 to 3 years. Former STEADY-PD3 participants had been randomized (1:1) to 3 years of isradipine or placebo treatment; SURE-PD3 participants had been randomized (1:1) to 2 years of inosine or placebo treatment.

Arm

Intervention/Treatment

former phase 3 PD trial participants

The AT-HOME PD cohort enrolled upon completion of STEADY-PD3 or during completion of SURE-PD3; enrolling 2 to 6 years after diagnosis, and on standard dopaminergic therapy for 0 to 3 years. Former STEADY-PD3 participants had been randomized (1:1) to 3 years of isradipine or placebo treatment; SURE-PD3 participants had been randomized (1:1) to 2 years of inosine or placebo treatment.

Outcome Measures

Primary Outcome Measures

Tele-visit modified MDS-UPDRS parts 1-3 (total score) [Baseline (0 months)]
Participants will be asked to complete (from home or other preferred environment) an hour-long Tele-visit that includes the Movement Disorders Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS), comprising MDS-UPDRS part 1a and (modified) part 3, as well as completion or confirmation of patient-reported component parts 1b and 2.
Change in Tele-visit modified MDS-UPDRS parts 1-3 (total score) [Two years (0, 12, and 24 months)]
Participants will be asked to complete (from home or other preferred environment) an hour-long Tele-visit that includes the Movement Disorders Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS), comprising MDS-UPDRS part 1a and (modified) part 3, as well as completion or confirmation of patient-reported component parts 1b and 2.
Change in Tele-visit MDS-UPDRS part 2 (score) [Two years (0, 12, and 24 months)]
Participants will be asked to complete (from home or other preferred environment) an hour-long Tele-visit that includes completion or confirmation of part 2 of the MDS-UPDRS.
Change in Smartphone tapping (score) [Two years (0, 3, 6, 9, 12 , 15, 18, 21, and 24 months)]
Participants will undergo a series of 10 tapping assessments every three months using a smartphone app.
Change in Fox Insight MDS-UPDRS part 2 (score) [Two years (0, 6, 12, 18, and 24 months)]
Participants will be asked to complete a set of standard questionnaires every 3 months through a companion study called Fox Insight. This online observational study, conducted by The Michael J. Fox Foundation, seeks to better understand experiences of daily living in PD and how they change with disease progression. The MDS-UPDRS (part 2) questionnaire is completed every 6 months (at every other Fox Insight survey set).

Secondary Outcome Measures

Tele-visit MDS-UPDRS part 1a (score) [Two years (0, 12, and 24 months)]
Tele-visit MDS-UPDRS part 1b (score) [Two years (0, 12, and 24 months)]
Tele-visit Modified MDS-UPDRS part 3 (score) [Two years (0, 12, and 24 months)]
Tele-visit Montreal Cognitive Assessment (MoCA; score) [Two years (0, 12, and 24 months)]
Tele-visit Schwab and England (S&E; score) [Two years (0, 12, and 24 months)]
Tele-visit Clinician Global Impression (CGI; score) [Two years (0, 12, and 24 months)]
Smartphone cognitive assessment (score) [Two years (0, 3, 6, 9, 12, 15, 18, 21, and 24 months)]
Fox Insight Parkinson's Disease Questionnaire - 8 item version (PDQ-8; score) [Two years (0, 3, 6, 9, 12, 15, 18, 21, and 24 months)]
Fox Insight EuroQol Five Dimensions Questionnaire (EQ-5D; score) [Two years (0, 6, 12, 18, and 24 months)]
Fox Insight Non-motor Symptoms Questionnaire (NMS-QUEST; score) [Two years (0, 3, 6, 9, 12, 15, 18, 21, and 24 months)]
Fox Insight REM (Rapid Eye Movement) Behavior Disorder (RBD; score) [Two years (0, 3, and 15 months)]
Fox Insight Parkinson's Daily Activities Questionnaire -15 (PDAQ-15; score) [Two years (0, 3, 9, 15, and 21 months)]
Fox Insight Geriatric Depression Scale (GDS; score) [Two years (0, 12, and 24 months)]

Eligibility Criteria

Criteria

Ages Eligible for Study:

N/A and Older

Sexes Eligible for Study:

All

Inclusion Criteria:

Enrollment in STEADY-PD3 or SURE-PD3 studies
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
Internet-enabled device that will support participation in tele-visits
Have created or willing to create a Global Unique Identifier (GUID)
Willing and able to provide informed consent
English fluency
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:

Inability to carry out study activities as determined by study staff

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	CHeT Telemedicine (Site 363)	Rochester	New York	United States	14642

Sponsors and Collaborators

Massachusetts General Hospital
Northwestern University
Sage Bionetworks
University of Rochester
National Institute of Neurological Disorders and Stroke (NINDS)
Michael J. Fox Foundation for Parkinson's Research
The Parkinson Study Group

Investigators

Principal Investigator: Michael A Schwarzschild, MD, PhD, Massachusetts General Hospital
Principal Investigator: Tanya Simuni, MD, Northwestern University
Principal Investigator: E. Ray Dorsey, MD, MBA, University of Rochester
Principal Investigator: Larsson Omberg, PhD, Sage Bionetworks