Personalized Parkinson Project PSP Cohort

Brain Diseases Clinical Trial

— PPP-PSP  

Official title:

Personalized Parkinson Project PSP Cohort

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05501431
• Other study ID #: NL75356.091.20
• Status: Recruiting
• Start date: May 1, 2021
• Est. completion date: June 2024

Study information

• Verified date: August 2022
• Source: Radboud University Medical Center
• Contact: Marjan Meinders, PhD
• Phone: +31243613450
• Email: marjan.meinders[@]radboudumc.nl
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

There is an urgent need for the development of digital progression biomakers, which are sensitive to detect small, but potentially clinically relevant changes in the disease course. Digital biomarkers are based on (i) continuously collected real-time data, during the patient's day to day activities; and (ii) task-based assessment. In this study the investigators are interested in developing algorithms for the detection of disease progression in PSP patients in key clinical parameters: bradykinesia, gait, rising from a chair and falls, based on (i) sensor data obtained by means of passive monitoring during daily living; and (ii) sensor data collected during the Virtual Motor Exam.

Description:

Rationale In light of the advent of a wide range of new disease modifying treatments, and acknowledging that existing clinical outcome measures may well be insufficiently sensitive to detecting small but potentially clinically relevant changes in the disease course, there is an urgent need for development of digital progression biomarkers, not only in the field of Parkinson's disease, but also in the field of the various forms of atypical parkinsonism, including Progressive Supranuclear Palsy (PSP).

Study Design Prospective, longitudinal, single-center cohort study.

Study Objectives The primary objective is to develop algorithms for the detection of disease progression in PSP patients in key clinical parameters: bradykinesia, gait, rising from a chair and falls, based on (1) sensor data obtained by means of passive monitoring during daily living; and (2) sensor data collected during the Virtual Motor Exam.

The second objective of this study is to create a longitudinal dataset describing the clinical and functional characteristics of a representative PSP cohort to allow researchers to investigate important unanswered questions in PSP.

Study population 50 patients with possible or probable PSP, according to established international criteria (i.e., MDS-PSP criteria); diagnosis confirmed by consensus review of a videotaped neurological examination by two neurologists with deep expertise in movement disorders. In addition, we will include 50 age and gender matched healthy controls.

Main study parameters/endpoints The primary study endpoints are the annual changes in the newly developed digital measurements, extracted from the Verily Study Watch. Secondary endpoints include standardized evaluations for motor, cognition, neuropsychological, quality of life and ADL assessments. Additionally, whole blood for DNA and serum will be collected in the PSP subjects, to use for e.g. genotyping and phenotyping.

Nature and extent of the burden and risks associated with participation, benefit and group relatedness For data collection, the study assessor will visit each participant at home twice within an interval of 1 year ± 60 days. All study assessments are routine exams done in standard clinical practice and are generally well tolerated, which takes up to 2 hours each time. PSP subjects will also be asked to consent to a blood draw once. During their 1-year participation, each participant will wear the Verily Study Watch daily for up to 23 hours. This small, unobtrusive electronic device is easily worn and poses no significant safety risk. All participants will be asked to complete a set scheduled tasks twice a day, once a week, which altogether takes 15-20 minutes of their time.

As data collection is not performed for immediate diagnostic or therapeutic purposes, there will be no direct benefits for the subjects enrolled in this study. Patients may indirectly benefit from the study, as their data contribute to providing novel etiological insights for improving the use of existing treatments, developing of new therapeutic approaches, and increasing the precision of personalized disease management.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 100
• Est. completion date: June 2024
• Est. primary completion date: December 2023
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 50 Years and older

Eligibility

Inclusion Criteria (PSP cohort):

• Subject has possible or probable PSP, according to the established international criteria defined by the Movement Disorders Society (MDS-PSP criteria, Höglinger et al, 2017); diagnosis is confirmed based on by consensus review process. In addition to the diagnosis of the own treating neurologist, the diagnosis always needs to be confirmed by at least one neurologist with deep expertise in movement disorders. The videotaped neurological examination as obtained during visit 1 will be used by the expert neurologist.

• Subject is an adult, at least 50 years of age.

• Subject can read and understand Dutch.

• Subject has completed the Informed Consent, as approved by the Institutional Review Board (IRB).

• Subject is willing, competent, and able to comply with all aspects of the protocol.

• Subject is able to walk at least 5 steps with or without minimal assistance (ie, stabilization of 1 arm or use of a cane or walker).

Inclusion Criteria (HC cohort):

• Subject has no neurological or musculoskeletal disorder or any other condition that impairs movement, gait, or balance, in the opinion of the Investigator.

• Subject is an adult, at least 50 years of age.

• Subject can read and understand Dutch.

• Subject has completed the Informed Consent, as approved by the IRB.

• Subject is willing, competent, and able to comply with all aspects of the protocol.

• Subject is able to walk at least 5 steps with or without minimal assistance (i.e., stabilization of 1 arm or use of a cane or walker).

Exclusion Criteria (PSP and HC cohorts):

• Subject has an additional condition or situation that, in the opinion of the Investigator, makes the subject inappropriate for participation in the study.

• For Study Watch: subject is allergic to nickel.

Related Conditions & MeSH terms

• Brain Diseases
• Movement Disorders
• Neurodegenerative Diseases
• Progressive Supranuclear Palsy
• Supranuclear Palsy, Progressive

Intervention

Device:
• Verily Study Watch
The Verily Study Watch is a smartwatch, collecting physiological and/or environmental information. The sensors collect data that include movement and activity, pulse rate, skin impedance, electrocardiogram (ECG), environmental temperature, and ambient light level.

Locations

Country	Name	City	State
Netherlands	Radboud University Medical Center	Nijmegen

Sponsors (3)

Lead Sponsor	Collaborator
Radboud University Medical Center	UCB Pharma, Verily Life Sciences LLC

Country where clinical trial is conducted

Netherlands, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Annual change in digital biomarkers for gait	Identify (a combined set of) gait-related features extracted from wearable sensor data, that are relevant for patients and that are sensitive to disease progression in PSP patients. Which outcome and which Unit of Measure for gait will be selected cannot be defined on forehand, as this is part of the analytical approach.	From baseline till one year follow-up
Primary	Annual change in digital biomarkers for bradykinesia	Identify (a combined set of) bradykinesia-related features extracted from wearable sensor data, that are relevant for patients and that are sensitive to disease progression in PSP patients. Which outcome and which Unit of Measure for bradykinesia will be selected cannot be defined on forehand, as this is part of the analytical approach.	From baseline till one year follow-up
Primary	Annual change in digital biomarkers for falls	Identify (a combined set of) falls-related features extracted from wearable sensor data, that are relevant for patients and that are sensitive to disease progression in PSP patients. Which outcome and which Unit of Measure for falls will be selected cannot be defined on forehand, as this is part of the analytical approach.	From baseline till one year follow-up
Primary	Annual change in digital biomarkers for rising from a chair	Identify (a combined set of) features related to rising from a chair extracted from wearable sensor data, that are relevant for patients and that are sensitive to disease progression in PSP patients. Which outcome and which Unit of Measure for rising from a chair will be selected cannot be defined on forehand, as this is part of the analytical approach.	From baseline till one year follow-up
Secondary	Annual change in digital biomarker for motor performance	Standardized evaluation, by means of 8 tasks, included in the Virtual Motor Examination. Which outcome and which Unit of Measure for motor performance will be selected cannot be defined on forehand, as this is part of the analytical approach.	Baseline till one year follow-up
Secondary	Annual change in cognition	Cognition will be assessed by means of the Mini-Mental State Examination version 2 (MMSE-2), on a 0-30-point scale.	Baseline till one year follow-up
Secondary	Annual change in D-KEFS trail making test	D-KEFS ( Delis-Kaplan Executive Function System) trail making test is a neuropsychological test for executive functioning, with scores in seconds to perform the task.	Baseline till one year follow-up
Secondary	Annual change in semantic fluency	The semantic fluency test is a neuropychological assessment, measuring executive function and semantic memory. The score entails the number of correct and unique words produced within a 60 seconds timeframe.	Baseline till one year follow-up
Secondary	Annual change in quality of life	Quality of life is captured by means of a general scale, i.e., EQ-5D-5L. The EQ-5D-5L consists of 2 parts: the EQ-5D descriptive system and the EQ visual analogue scale (EQ VAS).; The descriptive system comprises five dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has 5 levels: no problems, slight problems, moderate problems, severe problems and extreme problems. The EQ VAS records the patient's self-rated health on a vertical visual analogue scale, where the endpoints are labelled 'The best health you can imagine' and 'The worst health you can imagine'. The VAS can be used as a quantitative measure of health outcome that reflect the patient's own judgement.	Baseline till one year follow-up
Secondary	Annual change in Activities of Daily Living (ADL) performance	Modified Schwab & England Activities of Daily Living is a method of assessing the capabilities of people with impaired mobility. The scale uses percentages to represent how much effort and dependence on others people need to complete daily chores.	Baseline till one year follow-up