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.
试验速览
- 阶段
- 早期 1 期
- 状态
- 已完成
- 发起方
- Servier
- 入组人数
- 45
- 试验地点
- 8
- 主要终点
- Assess the potency of LRRK2 inhibitors using target engagement biomarker (cohort B)
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
盲法说明
Analysis is done ex-vivo and arm assignment is based on if the participant is symptomatic or not.
入排标准
- 年龄范围
- 30 Years 至 85 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •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)
排除标准
- •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
结局指标
主要结局
Assess the potency of LRRK2 inhibitors using target engagement biomarker (cohort B)
时间窗: Study collection visit Day 1
次要结局
未报告次要终点