A Multi-Modal Remote Monitoring Platform for Frontotemporal Lobar Degeneration (FTLD) Syndromes

The primary objective of this study is to enroll an observational cohort of approximately 60 patients with PSP over the course of 24 months using a multicenter study design and to follow each of them for 12 months.

The secondary objective of this study is to develop a robust solution for multi-modal remote monitoring of motor symptoms and function in PSP that can be applied to other Frontotemporal lobar degeneration (FTLD) syndromes.

Study Overview

• Status: Recruiting
• Conditions: Corticobasal Degeneration; Frontotemporal Dementia; Frontotemporal Lobar Degeneration; Progressive Supranuclear Palsy; CBD; FTD; PSP
• Intervention / Treatment: Device: PAMSys; Device: LEGSys

Detailed Description

The goal of this project is to develop a robust multi-modal platform for remote monitoring of motor symptoms and cognitive function in FTLD syndromes using wearable sensors and mobile health technology to assess speech, motor, and cognitive functions. The solution will be validated in Progressive Supranuclear Palsy (PSP) by collecting longitudinal data from 60 PSP individuals with PSP. Enrollment will take place over a period of 24 months, though each participant will only be followed for 12 months for data collection. The participants will consist of adult volunteers from two leading CurePSP Centers of Care located at the Atypical Parkinsonism Center at Johns Hopkins Hospital in Baltimore, MD and Massachusetts General Hospital (MGH) in Boston, MA.

• Study Type: Observational
• Enrollment (Estimated): 60

Contacts and Locations

• Study Contact: Name: Mansi Sharma, BS; Phone Number: 6176432400; Email: msharma13@mgh.harvard.edu
• Study Contact Backup: Name: Anne-Marie A Wills, MD MPH; Phone Number: 16177265532; Email: awills@mgh.harvard.edu

Study Locations

• United States
  • Maryland
    • Baltimore, Maryland, United States, 21093
      • Recruiting
      • Johns Hopkins School of Medicine
      • Principal Investigator: Alex Pantelyat, MD
      • Contact: AJ Hall; Phone Number: 410-616-2813; Email: ahall52@jhmi.edu
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Recruiting
      • Massachusetts General Hospital
      • Principal Investigator: Anne-Marie A Wills, MD MPH
      • Contact: Mansi Sharma; Phone Number: 617-643-2400; Email: msharma13@mgh.harvard.edu

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

The study intends to enroll a diverse population of individuals representative of the general PSP population. There are no restrictions based upon race or ethnic origin. This study will recruit both male and female participants aged 40 years or older with a clinical diagnosis of possible or probable PSP phenotype as defined by the 2017 MDS criteria.

Description

Inclusion Criteria:

• Clinical diagnosis of possible or probable PSP as defined by the 2017 MDS criteria
• Fluent in reading and speaking English and capable of providing informed consent based on the principal investigator's judgement
• Able to walk 10 feet unassisted at the time of initial enrollment
• Must have a caregiver or study partner who is willing and able to assist with all study-related procedures

Exclusion Criteria:

• Any neurological, medical, or psychiatric condition that would preclude participation in study activities based on the investigator's judgment.
• A history of frequent falls defined as more than 5 falls per month, or who require a walker to ambulate safely at baseline will not be eligible to participate in the study.

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Feasibility	Feasibility will be defined by the number of participants who complete the 12 month study and who wear their PAMSys pendant sensors at least 16 hours/day for at least 75% of the designated sensor days (7 days per month).	12 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
PSPRS	The Progressive Supranuclear Palsy Rating Scale will be administered in-person every 3 months. The PSPRS is a 28 item scale with a total score of 0-100 where lower scores are less affected.	12 months
Cortical Basal ganglia Functional Scale (CBFS)	The Cortical Basal ganglia Functional Scale (CBFS) will be performed every 3 months. The CBFS is a 31 item scale, with a total of 124 possible points, where a lower score is less affected.	12 months
PSP Quality of Life scale (PSP QoL)	The PSP Quality of Life scale will be performed every 3 months. The PSP-QoL is a 45 item scale, with a possible scale of 0-100 with lower scores less affected.	12 months

Collaborators and Investigators

• Sponsor: Massachusetts General Hospital
• Collaborators: Johns Hopkins University; BioSensics LLC

Study record dates

Study Major Dates

• Study Start (Actual): July 28, 2023
• Primary Completion (Estimated): July 30, 2025
• Study Completion (Estimated): December 30, 2026

Study Registration Dates

• First Submitted: July 14, 2023
• First Submitted That Met QC Criteria: July 14, 2023
• First Posted (Actual): July 21, 2023

Study Record Updates

• Last Update Posted (Actual): September 7, 2023
• Last Update Submitted That Met QC Criteria: September 2, 2023
• Last Verified: September 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Mental Disorders; Metabolic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Eye Diseases; Neurologic Manifestations; Neurobehavioral Manifestations; Neurocognitive Disorders; Basal Ganglia Diseases; Movement Disorders; Neurodegenerative Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Dementia; Tauopathies; Cranial Nerve Diseases; Ocular Motility Disorders; Language Disorders; Communication Disorders; Paralysis; Speech Disorders; Ophthalmoplegia; Aphasia; Frontotemporal Dementia; Aphasia, Primary Progressive; Pick Disease of the Brain; Supranuclear Palsy, Progressive; Frontotemporal Lobar Degeneration; Corticobasal Degeneration
• Other Study ID Numbers: IRB00341607

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Deidentified data will be available upon reasonable request.
• IPD Sharing Time Frame: 12 months after completion of the study and for 24 months.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; ICF

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No