Assessing Skin Biomarkers for Preclinical Diagnosis of PD and Non-PD Parkinsonism
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 184
- 试验地点
- 2
- 主要终点
- Change in UMSARS measures of multiple system atrophy (MSA) severity in people with MSA
研究概览
简要总结
The purpose of this study is to determine whether identification of misfolded proteins in the skin will help to determine what sort of parkinsonism someone has. We seek to demonstrate whether someone has a synucleinopathy such as Parkinson's disease (PD), multiple system atrophy (MSA), or dementia with Lewy bodies(DLB), as opposed to a tauopathy such as progressive supranuclear palsy (PSP) or corticobasal degeneration (CBD) or no parkinsonism at all (control).
详细描述
This is a clinical research study for patients with parkinsonism, including Parkinson's disease, progressive supranuclear palsy, corticobasal degeneration, multiple system atrophy, and dementia with Lewy bodies. Parkinsonism can be difficult to diagnose, especially in the early stages of the disease. Skin punch biopsy could be a useful and way to diagnose and measure the severity of these conditions. Given that there currently is no proven way to determine that someone has a synucleinopathy such as PD and not a tauopathy, this is a novel study that may lead to better ways to diagnose people with parkinsonism. The purpose of the study is to identify changes on a skin punch biopsy, in which small samples of skin are removed and sent to the laboratory for examination. We are seeking to measure the amount of misfolded alpha-synuclein in someone's skin. Participation will last between 1 and 2 years and will involve between 2 and 4 visits. Visits will include a physical examination, questionnaires, a memory test, blood draws and saliva collection, and a single visit for skin punch biopsies. We will also be looking to enroll volunteers to serve as "controls," who do not have any neurological illness.
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
- 21 Years 至 89 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Age 21 years old and age <90 years of age at the time of the baseline visit 1
- •Age of diagnosis at least 40 years old for PD, DLB, and PSP and at least 30 years old for MSA
- •A confirmed diagnosis of PD, PSP, CBD, MSA, DLB, or healthy control
- •Montreal Cognitive Assessment (MoCA) > 10 at the outset of the study
排除标准
- •Age 90 or above
- •Allergy to local anesthetic
- •History of deep brain stimulation (DBS) or other brain surgery prior to Visit 1
- •For PD or DLB diagnoses, any other neurodegenerative or central nervous system process that would interfere with examination
- •For PD or DLB, history of negative DATscan
- •Use of investigational drugs or devices within 60 days prior to baseline visit (except for dietary supplements)
- •In control subjects, family history of a neurodegenerative disease in a first degree or second degree blood relative
- •History of schizophrenia
- •History of antipsychotic medication use or exposure in controls or history of antipsychotic medication leading to parkinsonism (drug induced parkinsonism) in the parkinsonism group
- •Blood clotting disorder
- •On multiple (more than one) antiplatelet and/or anticoagulant blood thinner medications in combination (except for aspirin if it can be safely held for 1 week)
- •Any other medical, psychiatric, or cognitive illness that in the investigator's opinion would interfere with cooperation or ability to undergo the study procedures.
结局指标
主要结局
Change in UMSARS measures of multiple system atrophy (MSA) severity in people with MSA
时间窗: Baseline, 1 year, and optional 2 year assessment
Questionnaire and examination. Lower scores are better.
Change in Schwab and England (S&E) Score
时间窗: Baseline, 1 year, and optional 2 year assessment
0% to 100% rating scale score. Higher score is better.
Change in PSPRS measures of progressive supranuclear palsy (PSP) severity in people with PSP
时间窗: Baseline, 1 year, and optional 2 year assessment
Questionnaire and examination. Lower scores are better.
Change in Hoehn and Yahr (H&Y) and modified H&Y Scores
时间窗: Baseline, 1 year, and optional 2 year assessment
Zero to 5 parkinsonism rating scale score. Lower score is better.
Amount of alpha-synuclein in the skin
时间窗: Cross-sectional at baseline
Alpha-synuclein will be measured by RT-QuIC and sPMCA
次要结局
- Change in REM Behavior Disorder Questionnaire(Baseline, 1 year, and optional 2 year assessment)
- Change in blood pressure with orthostatic posture(Baseline, 1 year, and optional 2 year assessment)
- Change in Montreal Cognitive Assessment (MoCA)(Baseline, 1 year, and optional 2 year assessment)
- Change in Epworth Sleepiness Scale (ESS)(Baseline, 1 year, and optional 2 year assessment)
- Change in Hamilton anxiety scale(Baseline, 1 year, and optional 2 year assessment)
- Change in PDQ-39(Baseline, optional 1 year assessment, and optional 2 year assessment)
- Change in Hamilton depression scale(Baseline, 1 year, and optional 2 year assessment)
- Amount of alpha-synuclein in the blood(Baseline, optional 1 year assessment, and optional 2 year assessment)
研究者
Steven Gunzler, MD
Assistant Professor, Neurology
University Hospitals Cleveland Medical Center