Pilot Biomarker Study Assessing Alpha-synuclein Aggregates in Parkinson's Disease, Multiple System Atrophy, and Rapid Eye Movement Sleep Behavior Disorder

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

Sponsors

Lead Sponsor: Stony Brook University

Source Stony Brook University
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) and matched controls. Saliva, plasma, serum, urine, and cerebrospinal fluid (CSF) samples will be collected from participants.

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) and matched controls. Saliva, plasma, serum, urine, and optional 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.

Overall Status Recruiting
Start Date January 15, 2020
Completion Date June 2021
Primary Completion Date February 2021
Study Type Observational
Primary Outcome
Measure Time Frame
Compare levels of misfolded alpha-synuclein aggregates in participants with PD, MSA, RBD, and controls 3 weeks
Secondary Outcome
Measure Time Frame
Investigate the relationship between levels of misfolded alpha-synuclein aggregates and disease severity in PD and MSA 3 weeks
Investigate the relationship between levels of misfolded alpha-synuclein aggregates across different biofluid reservoirs, including CSF, serum, plasma, saliva, and urine 3 weeks
Enrollment 24
Condition
Intervention

Intervention Type: Other

Intervention Name: Biomarker assay

Description: 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 and controls.

Eligibility

Sampling Method: Non-Probability Sample

Criteria:

For PD subjects:

Inclusion Criteria:

- Age 50-75

- 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

- Willing to go off parkinsonian medication for 12 hours prior to "off" assessment

Exclusion Criteria:

- 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

- Pregnancy

- Inability to comply with or tolerate study procedures

- For subjects receiving optional lumbar puncture, 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 50-75

- Age of onset of motor symptoms between 50-75

- Diagnosis of probable or possible MSA as confirmed by a movement disorders specialist

- Able to complete questionnaires

- Ability to provide informed consent

- Willing to go off parkinsonian medications for 12 hours prior to "off" assessment

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

- Pregnancy

- Inability to comply with or tolerate study procedures

- For subjects receiving optional lumbar puncture, 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:

- Age 50-75

- Diagnosis of RBD using current consensus criteria

- Ability to provide informed consent

- Ability to complete questionnaires

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

- Pregnancy

- Inability to comply with or tolerate study procedures

- For subjects receiving optional lumbar puncture, 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:

- Age 50-80

- Scheduled to undergo an LP at the Stony Brook LP Clinic

- Ability to complete questionnaires

- Ability to provide informed consent

Exclusion Criteria:

- Signs or symptoms suggestive of parkinsonian disorder

- History of rapid eye movement (REM) Sleep Behavior Disorder (RBD)

- First degree relative with PD or 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

- Pregnancy

- 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)

Gender: All

Minimum Age: 50 Years

Maximum Age: 75 Years

Healthy Volunteers: Accepts Healthy Volunteers

Overall Official
Last Name Role Affiliation
Carine W. Maurer, MD, PhD Principal Investigator Stony Brook University Medical Center
Overall Contact

Last Name: Sandra L Skinner, PhD

Phone: 631-444-7513

Email: [email protected]

Location
Facility: Status: Contact: Contact Backup: Investigator: Stony Brook University Medical Center Sandra Skinner, PhD 631-444-7513 [email protected] Carine Maurer, MD,PhD Principal Investigator
Location Countries

United States

Verification Date

March 2020

Responsible Party

Type: Principal Investigator

Investigator Affiliation: Stony Brook University

Investigator Full Name: Carine Maurer

Investigator Title: Assistant Professor

Keywords
Has Expanded Access No
Condition Browse
Arm Group

Label: Parkinson's Disease

Description: Subjects who have a PD diagnosis

Label: Multiple System Atrophy

Description: Subjects who have an MSA diagnosis

Label: Age-matched controls

Description: Subjects who do not have a diagnosed neurological disorder.

Label: Rapid Eye Movement Sleep Behavior Disorder (RBD)

Description: Subjects who have a diagnosis of RBD

Patient Data Undecided
Study Design Info

Observational Model: Other

Time Perspective: Other

Source: ClinicalTrials.gov