A Pilot Biomarker Study Assessing Alpha-synuclein Aggregates Across Biofluid Reservoirs in Patients With Synucleinopathies
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 入组人数
- 8
- 试验地点
- 1
- 主要终点
- Compare levels of misfolded alpha-synuclein aggregates in participants with PD, MSA, RBD, NPH and controls
研究概览
简要总结
This will be an observational study looking at clinical and biomarker characteristics in patients with Parkinson's Disease (PD), Multiple System Atrophy (MSA), Rapid Eye Movement Sleep Behavior Disorder (RBD), Normal Pressure Hydrocephalus and matched controls. Saliva, plasma, serum, urine, and cerebrospinal fluid (CSF) samples will be collected from participants.
详细描述
This is an observational study looking at clinical and biomarker characteristics in patients with Parkinson's disease (PD), Multiple System Atrophy (MSA), Rapid Eye Movement Sleep Behavior Disorder (RBD), Normal Pressure Hydrocephalus (NPH) and matched controls. Saliva, plasma, serum, urine, and cerebrospinal fluid samples will be collected from participants. Samples will be assessed for levels of misfolded alpha-synuclein aggregates. Clinical characteristics will also be assessed.
Misfolded alpha-synuclein aggregates have the potential to serve as an early biomarker for PD and MSA, increasing the ability to diagnose and treat individuals with these diseases earlier. This study examines the effectiveness of using a novel technique for distinguishing between different parkinsonian disorders by measuring small misfolded α-synuclein aggregates in different biofluids.
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Other
入排标准
- 年龄范围
- 50 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Diagnosis of idiopathic PD as confirmed by a movement disorder specialist
- •Age of onset of motor symptoms between 50 - 75
- •Well-established response to dopaminergic agents and/or amantadine
- •Ability to complete questionnaires
- •Ability to provide informed consent
- •Willingness to go off parkinsonian medication for 12 hours prior to "off" assessment
- •Medical record includes a brain MRI taken within the past 12 months showing no evidence of a tumor or abscess
排除标准
- •Symptomatic (secondary) parkinsonism (ie. drug induced)
- •Atypical parkinsonian variants
- •History of cancer (except basal or squamous cell skin cancer) within 5 years
- •Known liver disease
- •Hematological disorders
- •History of stereotactic or ablative brain surgery
- •Treatment with an investigational drug or device within the last 30 days
- •Inability to comply with or tolerate study procedures
- •Conditions precluding the safe performance of lumbar puncture (LP): use of anticoagulants and hematologic abnormalities (INR>1.3;platelet count <80,000)
- •MSA Subjects:
- •Inclusion Criteria:
- •Age of onset of motor symptoms between 50-75
- •Diagnosis of probable or possible MSA as confirmed by a movement disorders specialist
- •Ability to complete questionnaires
- •Ability to provide informed consent
- •Willingness to go off parkinsonian medications for 12 hours prior to "off" assessment
- •Medical record indicates a brain MRI taken within the past 12 months showing no evidence of a tumor or abscess
- •Exclusion Criteria
- •Symptomatic (secondary) parkinsonism (ie. drug induced)
- •History of cancer (except basal or squamous cell skin cancer) within 5 years
- •Known liver disease
- •Hematological disorders
- •History of stereotactic or ablative brain surgery
- •Treatment with an investigational drug or device within the last 30 days
- •Inability to comply with or tolerate study procedures
- •Conditions precluding the safe performance of lumbar puncture (LP): use of anticoagulants and hematologic abnormalities (INR>1.3;platelet count <80,000)
- •For RBD Subjects:
- •Inclusion Criteria:
- •Diagnosis of RBD using current consensus criteria
- •Ability to provide informed consent
- •Ability to complete questionnaires
- •Medical record indicates a brain MRI taken within the past 12 months showing no evidence of a tumor or abscess
- •Exclusion Criteria
- •Signs for symptoms suggestive of parkinsonian disorder
- •History of cancer (except basal or squamous cell skin cancer) within 5 years
- •Known liver disease
- •Hematological disorders
- •History of stereotactic or ablative brain surgery
- •Treatment with an investigational drug or device within the last 30 days
- •Inability to comply with or tolerate study procedures
- •Conditions precluding the safe performance of lumbar puncture (LP): use of anticoagulants and hematologic abnormalities (INR>1.3;platelet count <80,000)
- •Inclusion Criteria:
- •Scheduled to undergo an LP to evaluate diagnosis of NPH at Stony Brook Neurological Associates
- •Ability to complete questionnaires
- •Ability to provide informed consent
- •Exclusion Criteria:
- •History of cancer (except basal or squamous cell skin cancer) within 5 years
- •Known liver disease
- •Hematological disorders
- •History of stereotactic or ablative brain surgery
- 另有 18 项未显示
结局指标
主要结局
Compare levels of misfolded alpha-synuclein aggregates in participants with PD, MSA, RBD, NPH and controls
时间窗: 3 weeks
Levels of misfolded alpha-synuclein in CSF, serum, plasma, saliva, and urine will be quantified using the protein misfolding cyclic amplification (PMCA) technology
次要结局
- Investigate the relationship between levels of misfolded alpha-synuclein aggregates across different biofluid reservoirs, including CSF, serum, plasma, saliva, and urine(3 weeks)
- Investigate the relationship between levels of misfolded alpha-synuclein aggregates and disease severity in PD and MSA(3 weeks)
研究者
Carine Maurer
Assistant Professor
Stony Brook University