Parkinson DiseaseNCT04020198
A Pilot Biomarker Study Assessing Alpha-synuclein Aggregates Across Biofluid Reservoirs in Patients With Synucleinopathies
An observational study of Parkinson Disease and Multiple System Atrophy, run by Stony Brook University at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 50 to 75 yearsHealthy volunteers can take part
- Conditions the study is for
- Parkinson Disease, Multiple System Atrophy, Rapid Eye Movement Sleep Behavior Disorder
- What is being tested?
- Biomarker assay
- How many people
- 8 participants
- Where
- 1 siteUnited States
- Study dates
- 15 Jan 2020to 19 Oct 2022
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 50 to 75 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Study population: Subjects with PD, MSA, RBD, NPH and controls
Who may be able to join
- 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
Show all 28Show fewer
- 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
- 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
- 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
- Age 50-75
- 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
- Age 50-75
- Scheduled to undergo an LP to evaluate diagnosis of NPH at Stony Brook Neurological Associates
- Ability to complete questionnaires
- Ability to provide informed consent
- Age 50-75
- Scheduled to undergo an LP at Stony Brook Neurological Associates
- Ability to complete questionnaires
- Ability to provide informed consent
Who cannot join
- 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
Show all 50Show fewer
- 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)
- MSA Subjects:
- 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
- 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:
- 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
- Conditions precluding the safe performance of lumbar puncture (LP): use of anticoagulants and hematologic abnormalities (INR>1.3;platelet count <80,000)
- For NPH:
- 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)
- For Controls:
- Signs or symptoms suggestive of parkinsonian disorder
- History of rapid eye movement (REM) Sleep Behavior Disorder (RBD)
- 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)
Trial locations and contacts
1 site in 1 country.
United States1 site
Stony Brook University Medical Center
Stony Brook, New York, 11794-8121
About this study
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.
Read the 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.
- Conditions
- Parkinson Disease, Multiple System Atrophy, Rapid Eye Movement Sleep Behavior Disorder, Normal Pressure Hydrocephalus
- Being tested
- Biomarker assay
- Keywords
- Alpha-synuclein
- Medical subject headings
- Parkinson Disease, Multiple System Atrophy, REM Sleep Behavior Disorder, Hydrocephalus, Normal Pressure, Parkinson Disease 4, Autosomal Dominant Lewy Body
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Biomarker assayOther
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.
| Study group | Role | What they receive |
|---|---|---|
| Parkinson's Disease | Subjects who have a PD diagnosis Receives: Other: Biomarker assay | |
| Multiple System Atrophy | Subjects who have an MSA diagnosis Receives: Other: Biomarker assay | |
| Age-matched controls | Subjects who do not have a diagnosed parkinsonian disorder Receives: Other: Biomarker assay | |
| Rapid Eye Movement Sleep Behavior Disorder (RBD) | Subjects who have a diagnosis of RBD Receives: Other: Biomarker assay | |
| Normal Pressure Hydrocephalus | Subjects who are prescribed a lumbar puncture to treat normal pressure hydrocephalus Receives: Other: Biomarker assay |
What is being measured
The primary outcome is the main question the study is designed to answer.
Compare levels of misfolded alpha-synuclein aggregates in participants with PD, MSA, RBD, NPH and controls
Levels of misfolded alpha-synuclein in CSF, serum, plasma, saliva, and urine will be quantified using the protein misfolding cyclic amplification (PMCA) technology
Measured: 3 weeks
Secondary outcomes (2)
Investigate the relationship between levels of misfolded alpha-synuclein aggregates and disease severity in PD and MSA
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.
Measured: 3 weeks
Investigate the relationship between levels of misfolded alpha-synuclein aggregates across different biofluid reservoirs, including CSF, serum, plasma, saliva, and urine
Levels of misfolded alpha-synuclein in the different biofluid reservoirs will be quantified using the PMCA technology
Measured: 3 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Jul 2019
- Study start15 Jan 2020
- Primary completion19 Oct 2022
- Study completion19 Oct 2022
Why it stopped: The assay under investigation did not show evidence of accurately detecting alpha-synuclein in specimens collected in the study other than CSF and the intention of the study was to identify less invasive ways to measure alpha-synuclein.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Parkinson DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Other
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- Stony Brook University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Carine Maurer, Stony Brook University
- Investigators
- Carine W. Maurer, MD, PhD, Principal investigator, Stony Brook University Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04020198
- Sponsor's protocol ID
- SNCA 1355881
- Status last verified
- Apr 2025
- First submitted
- 10 Jul 2019
- Last updated
- 6 Apr 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided: Identifiers will be removed from participant data or biospecimens. After such removal, the information or biospecimens may be used for future research studies.
Study at a glance
From the registry record, last updated by the sponsor on 6 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Biomarker Study Assessing Alpha-synuclein Aggregates Across Biofluid Reservoirs in Patients With Synucleinopathies
- ClinicalTrials.gov ID
- NCT04020198
- Acronym / sponsor ID
- SNCA 1355881
- Conditions
- Parkinson Disease, Multiple System Atrophy, Rapid Eye Movement Sleep Behavior Disorder, Normal Pressure Hydrocephalus
- Keywords
- Alpha-synuclein
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: The assay under investigation did not show evidence of accurately detecting alpha-synuclein in specimens collected in the study other than CSF and the intention of the study was to identify less invasive ways to measure alpha-synuclein.
- Enrolment
- 8 participants (actual)
- Lead sponsor
- Stony Brook UniversityUniversity, hospital or other
- Study start
- 15 Jan 2020 (actual)
- Primary completion
- 19 Oct 2022 (actual)
- Study completion
- 19 Oct 2022 (actual)
- First posted
- 15 Jul 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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
- Observational model
- Other
- Time perspective
- Other
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| Parkinson's Disease | – | Other: Biomarker assaySubjects who have a PD diagnosis |
| Multiple System Atrophy | – | Other: Biomarker assaySubjects who have an MSA diagnosis |
| Age-matched controls | – | Other: Biomarker assaySubjects who do not have a diagnosed parkinsonian disorder |
| Rapid Eye Movement Sleep Behavior Disorder (RBD) | – | Other: Biomarker assaySubjects who have a diagnosis of RBD |
| Normal Pressure Hydrocephalus | – | Other: Biomarker assaySubjects who are prescribed a lumbar puncture to treat normal pressure hydrocephalus |
Who may qualify
- Ages
- 50 to 75 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
- Study population
- Subjects with PD, MSA, RBD, NPH and controls
Key inclusion criteria (28 listed)
- 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
Key exclusion criteria (50 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Biomarker assayBiomarker 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. | Other | Age-matched controls, Multiple System Atrophy, Normal Pressure Hydrocephalus, Parkinson's Disease, Rapid Eye Movement Sleep Behavior Disorder (RBD) |
Primary outcome
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
2 secondary outcomes
Investigate the relationship between levels of misfolded alpha-synuclein aggregates and disease severity in PD and MSA
Time frame: 3 weeks
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
Results and safety
The study was stopped early (reason given: The assay under investigation did not show evidence of accurately detecting alpha-synuclein in specimens collected in the study other than CSF and the intention of the study was to identify less invasive ways to measure alpha-synuclein.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Apr 2025.
- Individual participant dataSharing planned: Undecided. 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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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Carine W. Maurer, MD, PhD principal investigator
Stony Brook University Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Parkinson Disease and Multiple System Atrophy, run by Stony Brook University at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 28 inclusion and 50 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 6 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was stopped early (reason given: The assay under investigation did not show evidence of accurately detecting alpha-synuclein in specimens collected in the study other than CSF and the intention of the study was to identify less invasive ways to measure alpha-synuclein.). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Compare levels of misfolded alpha-synuclein aggregates in participants with PD, MSA, RBD, NPH and controls (time frame: 3 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Apr 2025.