Patients With Parkinson's Disease (PD) Caused by the p.Gly2019Ser (G2019S) Pathogenic Mutation of the Leucine-Rich Repeat Kinase 2 (LRRK2) Gene
Conditions
Keywords
G2019S LRRK2 mutation
Brief summary
To characterize using a participant centered decentralized (at home) study featuring wearable technology and telemedicine to study disease change over time in patients with PD caused by the G2019S mutation in the LRRK2 gene and to identify a clinical endpoint(s) for disease modifying experimental therapy trials.
Interventions
OTHERObservational
This is an observational study to investigate the natural history of G2019S LRRK2 PD using traditional and digital methodology (digital phenotyping). Study conduct will be decentralized (at home as much as possible) consisting of two parts: Part A with Healthy Volunteers (HV) participants and Part B in patients with G2019S LRRK2 PD.
Sponsors
Momentum Pharma
Science 37
Escape Bio, Inc.
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
Yes
Inclusion criteria
Part A Inclusion Criteria (Healthy Volunteers) Individuals eligible to participate in Part A of this study will meet all the following criteria: 1. Willing and able to provide informed consent after the nature of the study has been explained 2. 18-35 years of age inclusive at Screening (sub cohort A1) or 65-80 years of age inclusive at Screening (sub cohort A2) 3. Willing and able to comply with all study procedures 4. Sub cohort A3 participants must match the age ± 0.5 years and sex of match participant in Part B 5. Participant must be healthy as per the investigator's assessment with no underlying clinically significant diseases (participants with hypertension, diabetes, hypercholesterolemia, and other chronic diseases associated with aging but not thought to interfere with the performance of the mTUG or other study measures may be allowed after consultation with the Medical Monitor) 6. Participant must be able to use the Technology as described in the protocol 7. Have access to a minimum indoor space of 6 meters by 2 meters (or approximately 20 feet by 7 feet) to carry out the mTUG 8. Agree to retain the same level of activity throughout the study and not have plans to stop/start or increase or decrease any exercise programs 9. Have reliable access to working WiFi internet or willingness to use a provided cellular internet connection device 10. Agree to refrain from all alcohol and cannabinoid products within 24 hours prior to performing the mTUG assessment (i.e., three non consecutive days during study Week two, and Weeks two and three of the run in period) Part A
Exclusion criteria
(Healthy Volunteers) Individuals who meet any of the following
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Compare the change over 12 months in the modified Timed Up and Go (mTUG) test | 12 months | To compare the change over 12 months in the modified Timed Up and Go (mTUG) test, or subitems of the mTUG, recorded by video and a smartphone sensor application between participants with PD caused by the G2019S pathogenic mutation of the LRRK2 gene, and age and sex matched healthy control cohort |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Evaluate the change over 12 months in linear velocity parameter in ambulation and gait. | 12 months | To evaluate the change over 12 months in parameter ambulation and gait including linear velocity measured in meters/second. |
| Evaluate the change over 12 months in stride length parameter in ambulation and gait. | 12 months | To evaluate the change over 12 months in parameter ambulation and gait including stride length measured in meter. |
| Evaluate the change over 12 months in cadence parameter in ambulation and gait. | 12 months | To evaluate the change over 12 months in parameter ambulation and gait including cadence measured in steps per minute. |
| Evaluate the change over 12 months in double support parameter in ambulation and gait. | 12 months | To evaluate the change over 12 months in parameter ambulation and gait including double support time as measured in milliseconds in a real world setting using a digital insole device. |
Countries
United States
Outcome results
None listed