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A Pilot Biomarker Study Assessing Alpha-synuclein Aggregates Across Biofluid Reservoirs in Patients With Synucleinopathies

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04020198
Recruitment Status : Active, not recruiting
First Posted : July 15, 2019
Last Update Posted : May 5, 2022
Sponsor:
Information provided by (Responsible Party):
Carine Maurer, Stony Brook University

Study Description
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Brief Summary:
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.

Condition or disease Intervention/treatment
Parkinson Disease Multiple System Atrophy Rapid Eye Movement Sleep Behavior Disorder Normal Pressure Hydrocephalus Other: Biomarker assay

Detailed Description:

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.

Study Design
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Study Type : Observational
Actual Enrollment : 8 participants
Observational Model: Other
Time Perspective: Other
Official Title: A Pilot Biomarker Study Assessing Alpha-synuclein Aggregates Across Biofluid Reservoirs in Patients With Synucleinopathies
Actual Study Start Date : January 15, 2020
Estimated Primary Completion Date : February 1, 2023
Estimated Study Completion Date : June 1, 2023

Resource links provided by the National Library of Medicine


Groups and Cohorts
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Group/Cohort Intervention/treatment
Parkinson's Disease
Subjects who have a PD diagnosis
 Other: Biomarker assay
Biomarker assay will be used to quantify levels of misfolded alpha-synuclein aggregates in biofluid samples from patients with Parkinson's disease, multiple system atrophy, rapid eye movement sleep behavior disorder, normal pressure hydrocephalus and controls.
Multiple System Atrophy
Subjects who have an MSA diagnosis
 Other: Biomarker assay
Biomarker assay will be used to quantify levels of misfolded alpha-synuclein aggregates in biofluid samples from patients with Parkinson's disease, multiple system atrophy, rapid eye movement sleep behavior disorder, normal pressure hydrocephalus and controls.
Age-matched controls
Subjects who do not have a diagnosed parkinsonian disorder
 Other: Biomarker assay
Biomarker assay will be used to quantify levels of misfolded alpha-synuclein aggregates in biofluid samples from patients with Parkinson's disease, multiple system atrophy, rapid eye movement sleep behavior disorder, normal pressure hydrocephalus and controls.
Rapid Eye Movement Sleep Behavior Disorder (RBD)
Subjects who have a diagnosis of RBD
 Other: Biomarker assay
Biomarker assay will be used to quantify levels of misfolded alpha-synuclein aggregates in biofluid samples from patients with Parkinson's disease, multiple system atrophy, rapid eye movement sleep behavior disorder, normal pressure hydrocephalus and controls.
Normal Pressure Hydrocephalus
Subjects who are prescribed a lumbar puncture to treat normal pressure hydrocephalus
 Other: Biomarker assay
Biomarker assay will be used to quantify levels of misfolded alpha-synuclein aggregates in biofluid samples from patients with Parkinson's disease, multiple system atrophy, rapid eye movement sleep behavior disorder, normal pressure hydrocephalus and controls.


Outcome Measures
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Primary Outcome Measures :
  1. Compare levels of misfolded alpha-synuclein aggregates in participants with PD, MSA, RBD, NPH and controls [ Time Frame: 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


Secondary Outcome Measures :
  1. Investigate the relationship between levels of misfolded alpha-synuclein aggregates and disease severity in PD and MSA [ Time Frame: 3 weeks ]
    Levels of misfolded alpha-synuclein aggregates will be quantified using the PMCA technology. PD and MSA disease severity will be assessed using the Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) and the Unified Multiple System Atrophy Rating Scale (UMSARS), respectively. All groups will receive the MDS-UPDRS III and the RBD Questionnaire.

  2. Investigate the relationship between levels of misfolded alpha-synuclein aggregates across different biofluid reservoirs, including CSF, serum, plasma, saliva, and urine [ Time Frame: 3 weeks ]
    Levels of misfolded alpha-synuclein in the different biofluid reservoirs will be quantified using the PMCA technology


Biospecimen Retention:   Samples Without DNA
CSF, plasma, serum, urine

Eligibility Criteria
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Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   50 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Sampling Method:   Non-Probability Sample
Study Population
Subjects with PD, MSA, RBD, NPH and controls
Criteria

For PD subjects:

Inclusion Criteria:

  1. Age 50-75
  2. Diagnosis of idiopathic PD as confirmed by a movement disorder specialist
  3. Age of onset of motor symptoms between 50 - 75
  4. Well-established response to dopaminergic agents and/or amantadine
  5. Ability to complete questionnaires
  6. Ability to provide informed consent
  7. Willingness to go off parkinsonian medication for 12 hours prior to "off" assessment
  8. Medical record includes a brain MRI taken within the past 12 months showing no evidence of a tumor or abscess

Exclusion Criteria:

  1. Symptomatic (secondary) parkinsonism (ie. drug induced)
  2. Atypical parkinsonian variants
  3. History of cancer (except basal or squamous cell skin cancer) within 5 years
  4. Known liver disease
  5. Hematological disorders
  6. History of stereotactic or ablative brain surgery
  7. Treatment with an investigational drug or device within the last 30 days
  8. Pregnancy
  9. Inability to comply with or tolerate study procedures
  10. 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:

  1. Age 50-75
  2. Age of onset of motor symptoms between 50-75
  3. Diagnosis of probable or possible MSA as confirmed by a movement disorders specialist
  4. Ability to complete questionnaires
  5. Ability to provide informed consent
  6. Willingness to go off parkinsonian medications for 12 hours prior to "off" assessment
  7. Medical record indicates a brain MRI taken within the past 12 months showing no evidence of a tumor or abscess

Exclusion Criteria

  1. Symptomatic (secondary) parkinsonism (ie. drug induced)
  2. History of cancer (except basal or squamous cell skin cancer) within 5 years
  3. Known liver disease
  4. Hematological disorders
  5. History of stereotactic or ablative brain surgery
  6. Treatment with an investigational drug or device within the last 30 days
  7. Pregnancy
  8. Inability to comply with or tolerate study procedures
  9. 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:

  1. Age 50-75
  2. Diagnosis of RBD using current consensus criteria
  3. Ability to provide informed consent
  4. Ability to complete questionnaires
  5. Medical record indicates a brain MRI taken within the past 12 months showing no evidence of a tumor or abscess

Exclusion Criteria

  1. Signs for symptoms suggestive of parkinsonian disorder
  2. History of cancer (except basal or squamous cell skin cancer) within 5 years
  3. Known liver disease
  4. Hematological disorders
  5. History of stereotactic or ablative brain surgery
  6. Treatment with an investigational drug or device within the last 30 days
  7. Pregnancy
  8. Inability to comply with or tolerate study procedures
  9. Conditions precluding the safe performance of lumbar puncture (LP): use of anticoagulants and hematologic abnormalities (INR>1.3;platelet count <80,000)

For NPH:

Inclusion Criteria:

  1. Age 50-75
  2. Scheduled to undergo an LP to evaluate diagnosis of NPH at Stony Brook Neurological Associates
  3. Ability to complete questionnaires
  4. Ability to provide informed consent

Exclusion Criteria:

  1. History of cancer (except basal or squamous cell skin cancer) within 5 years
  2. Known liver disease
  3. Hematological disorders
  4. History of stereotactic or ablative brain surgery
  5. Treatment with an investigational drug or device within the last 30 days
  6. Pregnancy
  7. Inability to comply with or tolerate study procedures
  8. Conditions precluding the safe performance of lumbar puncture (LP): use of anticoagulants and hematologic abnormalities (INR>1.3;platelet count <80,000)

For Controls:

Inclusion Criteria:

  1. Age 50-75
  2. Scheduled to undergo an LP at Stony Brook Neurological Associates
  3. Ability to complete questionnaires
  4. Ability to provide informed consent

Exclusion Criteria:

  1. Signs or symptoms suggestive of parkinsonian disorder
  2. History of rapid eye movement (REM) Sleep Behavior Disorder (RBD)
  3. History of cancer (except basal or squamous cell skin cancer) within 5 years
  4. Known liver disease
  5. Hematological disorders
  6. History of stereotactic or ablative brain surgery
  7. Treatment with an investigational drug or device within the last 30 days
  8. Pregnancy
  9. Inability to comply with or tolerate study procedures
  10. Conditions precluding the safe performance of lumbar puncture (LP): use of anticoagulants and hematologic abnormalities (INR>1.3;platelet count <80,000)
Contacts and Locations
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Top of Page Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04020198


Locations
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United States, New York
Stony Brook University Medical Center
Stony Brook, New York, United States, 11794-8121
Sponsors and Collaborators
Stony Brook University
Investigators
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Principal Investigator: Carine W. Maurer, MD, PhD Stony Brook University Medical Center
More Information
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Responsible Party: Carine Maurer, Assistant Professor, Stony Brook University
ClinicalTrials.gov Identifier: NCT04020198    
Other Study ID Numbers: SNCA 1355881
First Posted: July 15, 2019    Key Record Dates
Last Update Posted: May 5, 2022
Last Verified: May 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided
Plan Description: Identifiers will be removed from participant data or biospecimens. After such removal, the information or biospecimens may be used for future research studies.

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Carine Maurer, Stony Brook University:
Alpha-synuclein
Additional relevant MeSH terms:
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Parkinson Disease
Hydrocephalus
Multiple System Atrophy
Shy-Drager Syndrome
Hydrocephalus, Normal Pressure
Synucleinopathies
REM Sleep Behavior Disorder
Mental Disorders
Parkinsonian Disorders
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
 Movement Disorders
Neurodegenerative Diseases
Primary Dysautonomias
Autonomic Nervous System Diseases
Hypotension
Vascular Diseases
Cardiovascular Diseases
Proteostasis Deficiencies
Metabolic Diseases
REM Sleep Parasomnias
Parasomnias
Sleep Wake Disorders