Non-drug Treatment Study in Parkinson's Disease Patients and Healthy Volunteers to Collect Biological Samples in Order to Assess ex Vivo the Activity of Candidate Therapies and Develop Novel Analytical Methods for Biomarkers

Study Overview
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Brief Summary
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This is a non-drug study in patients with Parkinson's Disease (PD) and healthy volunteers to process and analyze their blood and optional cerebrospinal fluid (CSF) samples ex vivo, or outside of their body, to further develop researchers knowledge on LRRK2 biomarkers. Participants were enrolled either in Cohort A or in Cohort B. A blood sample was collected in each participant within a maximum of two study visits within 14 days, and an option to complete both visits on the same day.

Detailed Description
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This study will allow researchers to assess the potency of investigational LRRK2 inhibitors, outside of participant bodies. It will enroll Parkinson's disease patients (LRRK2 G2019S symptomatic carriers and idiopathic) and healthy volunteers meeting eligibility criteria. The study will consist of a maximum of two study visits, a selection visit to check eligibility criteria, and a sample collection visit. The two visits can take place on the same day or can occur within 14 days of each other. Participants will be assigned to Cohort A or Cohort B, each include a blood sample collection, and an optional cerebrospinal fluid (CSF) collection in Cohort A. No investigational product, study drug, will be administered to study participants.

Official Title
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An Open-label, Phase 0 Clinical Study in Parkinson's Disease Patients and Healthy Volunteers for the Establishment of Biomarker Analytical Methods and ex Vivo Assessment of the Potency of LRRK2 Inhibitors Using Human Biological Samples.

Conditions
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Parkinson Disease

Intervention / Treatment
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* Other: Blood sample collection and an optional cerebrospinal fluid (CSF) collection
* Other: Blood sample collection


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Women of childbearing potential (WOCBP) must have a negative pregnancy test Parkinson's disease (PD) patients must be at least 40 years old Healthy volunteers must be at least 30 years old PD patients must have a PD diagnosis, according to the 2015 Movement Disorder Society criteria, with bradykinesia, and at least one of the following: muscular rigidity, or resting tremor with no other suspected cause of Parkinsonism The LRRK2 G2019S symptomatic carrier PD patients must have a documented G2019S mutation on the leucine-rich repeat kinase 2 gene (LRRK2) Exclusion Criteria: Pregnant or lactating women Have participated in an investigational drug trial within 30 days Are SARS-CoV-2, COVID-19, positive within 15 days prior to blood sample collection Have another active disease that the investigator believes could interfere with study results

Ages Eligible for Study
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Minimum Age: 30 Years
Maximum Age: 85 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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Accepts Healthy Volunteers

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Cohort A<br>Cohort A will include LRRK2 G2019S symptomatic carriers, idiopathic PD patients, and healthy volunteers. It will involve a blood sample collection and an optional cerebrospinal fluid (CSF) collection. | Other: Blood sample collection and an optional cerebrospinal fluid (CSF) collection<br>* No study drug will be administered to study participants.<br>|
| Experimental: Cohort B<br>Cohort B will include LRRK2 G2019S carriers, idiopathic PD patients, and heathy volunteers. It will involve only a blood sample collection. | Other: Blood sample collection<br>* No study drug will be administered to study participants.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Assess the potency of LRRK2 inhibitors using target engagement biomarker (cohort B) |  | Study collection visit Day 1 |