Evaluation De La Performance Clinique D'un Nouveau Biomarqueur Sanguin Des Phases Précoces De La Maladie De Parkinson
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 70
- 试验地点
- 1
- 主要终点
- validation of the biomarker in advanced stage PD
研究概览
简要总结
The investigators have recently discovered a metabolic biomarker which predicts Parkinson's disease (PD) at the early stages in patients and in animal models. The aim of BIOPARK is to investigate how the biomarker evolves in advanced PD stage, when diagnosis confirmation is higher, an in de novo PD patients who come from a different geographical area than those of the publication (since it is known that the metabolome is largely influenced by lifestyle). They will also evaluate if the biomarker is able to distinguish patients with a parkinsonian syndrome often confused with parkinson's disease, i.e. Multiple System Atrophy (MSA).
详细描述
Parkinson's disease (PD) affects more than 7 million people worldwide and represents a growing health and socio-economic burden. It is an incurable neurodegenerative disease, and the search for biomarkers that allow reliable early diagnosis and provide new therapeutic targets is essential to find cures for PD.
In a recently published preclinical and clinical study, the investigators have identified in 2 rat models of PD and a primate model and in 2 human cohorts from biobanks significant deregulations of 6 serum metabolites: acetoacetate, betaine, beta-hydroxybutyrate, creatine, pyruvate and valine. From these 6 metabolites, they built a composite biomarker, which allowed to classify de novo parkinsonian patients against controls (healthy subjects) with an accuracy (defined as the ratio (correctly classified/total) of 82.6%. This study demonstrated for the first time that a common metabolic dysregulation occurs early in either animal models or in PD patients, thus providing an unbiased diagnostic tool as well as major hypothesis for the understanding of the pathophysiology of the disease and the development of innovative therapeutic approaches.
The goal of BIOPARK is to improve the clinical diagnosis of early PD using the blood biomarker.
To this end, the investigators will study whether the biomarker is able to differentiate between patients with PD >5 years and already treated with dopaminergic drugs (thus with a very high diagnostic confirmation) and patients suffering from other neurodegenerative diseases often confused with it, mainly Multiple Systeme Atrophy (MSA). Furthermore, the investigators hope to confirm the preliminary results on a new cohort of de novo patients.
For that aim, they will use the already optimized method for biomarker discovery, i.e. Nuclear Magnetic Resonance (NMR)-based metabolomics on patient serum.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients with de novo Parkinson's disease, without dopaminergic treatment
- •Patients with advanced Parkinson's disease (> 5years) with dopaminergic treatment
- •Patients with multiple system atrophy
排除标准
- •Patients with deep brain stimulation
- •Other neurodegenerative diseases
- •patients protected by french law (pregnant or lactating women, prisoners, ...)
结局指标
主要结局
validation of the biomarker in advanced stage PD
时间窗: 2 years
The main objective of the project is to evaluate whether classification with the biomarker is consistent with the diagnosis in a cohort of parkinsonian patients with high diagnostic confirmation, i.e. patients treated with L-Dopa for over 5 years
次要结局
- Validation of the biomarker in a new cohort of PD patients(2 years)
- Specificity of the biomarker for PD(2 years)
- Correlation with MDS-UPDRS(2 years)