Longitudinal Exploratory Prospective Evaluation of Clinical Evolution in Patients With Motor Predominant Parkinson Disease.
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 发起方
- BrainEver
- 入组人数
- 3
- 试验地点
- 2
- 主要终点
- Change from baseline in Movement Disorders Society Unified Parkinson's Disease Rating Scale Part 3 (MDS-UPDRS Part III) "off" medications
研究概览
简要总结
The objective of this study is the longitudinal prospective clinical evaluation in patients with motor predominant PD; it will assess the individual disease progression (change) of the clinical and imaging parameters measured at 6-month interval over a minimum of 12 months.
详细描述
The absence of clear PD subtypes defining a common path and rate of disease progression, combined with the absence of validated biomarkers indicative of disease progression and a strong placebo effect, constitute a challenge for clinical trials assessing new potential disease-modifying therapies in PD.
The present cohort would constitute a well-defined population of patients, with individual progression assessment, suitable to evaluate new disease-modifying therapies in a subsequent Phase1/2 clinical trial.
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
- — 至 70 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Idiopathic Parkinson's Disease (According to British Brain Bank Criteria); genetic forms of PD are not excluded
- •Male or female adult, aged 70 or less
- •Modified Hoehn and Yahr stage 2 to 3 in OFF state
- •Disease duration, i.e. time from first motor symptoms between 2 to 8 (included) years ; first motor symptoms to be taken into consideration being the cardinal motor symptoms of PD (bradykinesia, tremor and rigidity)
- •Univoqual response to L-DOPA (of at least 50% in MDS-UPDRS Part III motor score)
- •Presence of fluctuations and/or dyskinesia
- •MDS-UPDRS total motor score ≥15 in OFF state
- •L-Dopa treatment stable for at least 4 weeks
- •Covered by healthcare insurance
- •Written informed consent form signed
排除标准
- •Scan Without Evidence of Dopamine Deficit (SWEDD) (DaTSCAN)
- •Atypical parkinsonism syndrome
- •Dementia as detected by a score < 21/30 at the Montreal Cognitive Assessment Screening (MoCA)
- •Psychiatric disorders including major depression with suicidal thoughts as evaluated by a psychiatrist or a neurologist at the selection period
- •Any medical or psychological problems which may interfere with a smooth conduction of the study protocol (e.g. cancer with a limited life expectancy)
- •History of significant brain or vascular disease (tumor, epilepsy, stroke ...)
- •Any contraindication for undergoing MRI of the head
- •Prior brain surgical procedures with or without implementation of an intra-cerebral device
- •Drug or alcohol addiction
- •Pregnancy or breastfeeding
- •Patient with reproductive potential who do not agree to use an accepted effective method of contraception - investigator's judgment- during the study period
- •Illiteracy or insufficient language skills (French) to complete the questionnaires
- •Patient deprived of liberty by a judicial or administrative decision, or who is under a measure of legal protection (e.g. guardianship or curatorship).
- •Simultaneous participation in another clinical trial with the administration of investigational drug(s)
结局指标
主要结局
Change from baseline in Movement Disorders Society Unified Parkinson's Disease Rating Scale Part 3 (MDS-UPDRS Part III) "off" medications
时间窗: baseline, 6, 12, 18, 24 and 30 months after baseline ]
An examiner will measure MDS-UPDRS part 3 motor scores "off" medications. MDS-UPDRS Part III is a motor examination consisting of 18 summed items where the investigator rates each motor symptom based on a scale of 0 - 4, higher values indicating worse function.
次要结局
- Change from Baseline of brain MRI(12 and 24 months)