A Natural History Study of Patients With G2019S LRRK2 Parkinson's Disease

Terminated

• Conditions: Patients With Parkinson's Disease (PD) Caused by the p.Gly2019Ser (G2019S) Pathogenic Mutation of the Leucine-Rich Repeat Kinase 2 (LRRK2) Gene
• Interventions: Other: Observational

• Registration Number: NCT05349019
• Lead Sponsor: Escape Bio, Inc.

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2022-04-14
• Study First Submitted QC: 2022-04-26
• Study First Posted: 2022-04-27
• Study Start: 2022-05-02
• Status Verified: 2022-08
• Primary Completion: 2022-08-05
• Study Completion: 2022-08-05
• Last Update Submitted: 2023-01-16
• Last Update Posted: 2023-01-18

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 22

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Part A/Sub Cohort A3	Observational	Control group age and sex matched to the PD participants in Part B of the study
Part A/Sub Cohort A1	Observational	15 healthy participants 18-35 years of age
Part B	Observational	60 participants with a confirmed diagnosis of Parkinson's disease and a heterozygous G2019S mutation in the LRRK2 gene
Part A/Sub Cohort A2	Observational	15 healthy participants 65-80 years of age

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
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 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 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.

Trial Locations

Locations (1)

Science 37; 🇺🇸 Culver City, California, United States