Clinical trial NCT05837130: 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
Official title: 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.
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: EARLY_PHASE1
Conditions: Parkinson Disease
Lead sponsor: Servier
Sponsor class: INDUSTRY
First posted: 2023-05-01
Enrollment: 45
Sex: ALL
Minimum age (years): 30
Maximum age (years): 85
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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: 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.

Eligibility criteria: 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

Primary outcomes:
measure: Assess the potency of LRRK2 inhibitors using target engagement biomarker (cohort B)
timeFrame: Study collection visit Day 1
Secondary outcomes:
Other outcomes:
Locations:
city: La Tronche
country: France
facility: Centre Hospitalo-Universitaire Grenoble Alpes
city: Lille
country: France
facility: Centre Hospitalo-Universitaire Lille
city: Paris
country: France
facility: Hopital La Pitié Salpétrière-Institut du Cerveau et de la Moelle Epinière-CIC Neurosciences
city: Toulouse
country: France
facility: Centre Hospitalo-Universitaire Toulouse
Study officials:
References: