Neurodegenerative DiseasesNCT05486806
Longitudinal Tracking of Patients Diagnosed With Neurodegenerative Movement Disorders
An observational study of Neurodegenerative Diseases and sca3, run by Brigham and Women's Hospital at 1 site in United States.
Trial summary
The purpose of this protocol is to create an active natural history cohort of patients with degenerative movement disorders, tracked in a clinical setting with clinical rating scales and neuroimaging. The overarching rationale is that neurodegenerative diseases may be heterogeneous, complex disorders. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Neurodegenerative Diseases, sca3, MSA - Multiple System Atrophy
- What is being tested?
- 18F-PBR06
- Type of treatment
- Surgery or procedure
- How many people
- 50 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Mar 2023to 15 Nov 2024
- 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 18 years and older
- All sexes
- Adult, Older adult
Study population: individuals with a clinical diagnosis of neurodegenerative movement disorder by consensus criteria (MSA, ataxias, synuclein duplication, atypical parkinsonism, etc)
Who may be able to join
- Clinical diagnosis of neurodegenerative movement disorder by consensus criteria including: MSA, ataxias, synuclein duplication, atypical parkinsonism
- Male and female subjects aged 18 and up
Who cannot join
- Individuals with a known alternate neurologic disorder including: idiopathic PD, DLB, PSP, ALS, Alzheimer's, prion disease, frontotemporal dementia, seizure disorder, stroke, or brain tumor
- Individuals with a previous head injury (with 15 minutes or greater loss of consciousness within the past 20 years)
- Individuals with substance abuse, or substance abuse disorder
- Brain MRI indicative of a significant abnormality (i.e. prior hemorrhage or infarct greater than 1 cm3, 3 or more lacunar infarcts, cerebral contusion, encephalomalacia, aneurysm, vascular malformation, subdural hematoma, hydrocephalus, space- occupying lesion).
- Individuals with bipolar disease and schizophrenia
- Concurrent medical conditions that contraindicate study procedures
Show all 12Show fewer
- Women who are pregnant, nursing, or seeking to become pregnant
- Individuals with claustrophobia
- Non-MRI compatible implanted devices
- Corticosteroid treatment in the past four weeks
- Low affinity binders to TSPO
- Significant cognitive impairment (MoCA score ≤ 23) or poor understanding of study design
Trial locations and contacts
1 site in 1 country.
Vikram Khurana, MD PhD contact
United States1 site, 1 recruiting
- Recruiting
Brigham and Women's Hospital
Boston, Massachusetts, 02115
Diego Rodriguez, MD
About this study
The purpose of this protocol is to create an active natural history cohort of patients with degenerative movement disorders, tracked in a clinical setting with clinical rating scales and neuroimaging. The overarching rationale is that neurodegenerative diseases may be heterogeneous, complex disorders. A new way of performing clinical trials in these patients may be in order and this protocol aims to build a longitudinally tracked clinical trial-ready cohort of patients. The purpose of this protocol is to establish an active natural history cohort of patients with neurodegenerative movement disorders who are deeply phenotyped and "clinical trial ready" across Mass General Brigham.
After a thorough clinical diagnostic evaluation (this may include clinically indicated testing, for example MRI, FDG-PET, MIBG scan, polysomnography, genetic testing, autonomic function tests, inflammatory tests, skin biopsy) the investigators aim to achieve this through:
Read more
- Longitudinal tracking of clinical progression through use of clinical scales including at the present time: UMSARS, BARS, MoCA and UPSIT, PROM, MDS-NMS, UPDRS, and SARA
- Longitudinal tracking of disease progression through use of neuroimaging including at the present time: TSPO-PET and 3D MRI (see section 1.3)
This is a pilot study designed to track patients with neurodegenerative movement disorders across Mass General Brigham through MRI and PET imaging modalities and clinical measures. Figure 5 represents the study design in detail. In short, subjects will be asked to visit Mass General Brigham every 6-9 months over the course of 18 months for imaging and clinical evaluation.
Read the detailed description
After a thorough clinical diagnostic evaluation (this may include clinically indicated testing, for example MRI, FDG-PET, MIBG scan, polysomnography, genetic testing, autonomic function tests, inflammatory tests, skin biopsy) the investigators aim to achieve this through:
- Longitudinal tracking of clinical progression through use of clinical scales including at the present time: UMSARS, BARS, MoCA and UPSIT, PROM, MDS-NMS, UPDRS, and SARA
- Longitudinal tracking of disease progression through use of neuroimaging including at the present time: TSPO-PET and 3D MRI (see section 1.3)
This is a pilot study designed to track patients with neurodegenerative movement disorders across Mass General Brigham through MRI and PET imaging modalities and clinical measures. Figure 5 represents the study design in detail. In short, subjects will be asked to visit Mass General Brigham every 6-9 months over the course of 18 months for imaging and clinical evaluation.
- Conditions
- Neurodegenerative Diseases, sca3, MSA - Multiple System Atrophy, Ataxia, Synucleinopathies
- Being tested
- 18F-PBR06
- Medical subject headings
- Neurodegenerative Diseases, Muscular Disorders, Atrophic, Shy-Drager Syndrome, Ataxia, Synucleinopathies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
18F-PBR06Drug
TSPO-PET (translocatior protein, positron emission tomography) scan
18F-PBR06Radiation
TSPO-PET (translocatior protein, positron emission tomography) scan
| Study group | Role | What they receive |
|---|---|---|
| participants | all participants in this study must have a diagnosis of a neurodegenerative movement disorder Receives: Drug: 18F-PBR06; Radiation: 18F-PBR06 |
What is being measured
The primary outcome is the main question the study is designed to answer.
association between change in PET and clinical measures
For the longitudinal evaluation, the investigators will estimate the association between the change in the PET and the change in the clinical and morphometric outcome measures using a mixed model with both the baseline PET uptake and the rate of change in PET uptake as predictors. This approach will allow the investigators to estimate both how between and within subject changes in PET impact the outcomes of interest. The investigators will use a p<0.05, corrected for multiple comparisons.
Measured: 18 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Aug 2022
- Study start1 Mar 2023
- Primary completion15 Sep 2024 (estimated)
- Study completion15 Nov 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neurodegenerative DiseasesStudies of the same condition, open and under way.
- More trials run by Brigham and Women's HospitalThe sponsor's other registered studies.
- 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
- Cohort
- Time perspective
- Prospective
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Brigham and Women's Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Vikram Khurana, MD PhD, Brigham and Women's Hospital
- Investigators
- Vikram Khurana, MD PhD, Principal investigator, Brigham and Women's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05486806
- Sponsor's protocol ID
- 2022P001295
- Status last verified
- May 2023
- First submitted
- 2 Aug 2022
- Last updated
- 22 May 2023
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 May 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Longitudinal Tracking of Patients Diagnosed With Neurodegenerative Movement Disorders
- ClinicalTrials.gov ID
- NCT05486806
- Acronym / sponsor ID
- 2022P001295
- Conditions
- Neurodegenerative Diseases, sca3, MSA - Multiple System Atrophy, Ataxia, Synucleinopathies
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Brigham and Women's HospitalUniversity, hospital or other
- Study start
- 1 Mar 2023 (actual)
- Primary completion
- 15 Sep 2024 (estimated)
- Study completion
- 15 Nov 2024 (estimated)
- First posted
- 4 Aug 2022 (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.
The purpose of this protocol is to create an active natural history cohort of patients with degenerative movement disorders, tracked in a clinical setting with clinical rating scales and neuroimaging. The overarching rationale is that neurodegenerative diseases may be heterogeneous, complex disorders. A new way of performing clinical trials in these patients may be in order and this protocol aims to build a longitudinally tracked clinical trial-ready cohort of patients. The purpose of this protocol is to establish an active natural history cohort of patients with neurodegenerative movement disorders who are deeply phenotyped and "clinical trial ready" across Mass General Brigham.
After a thorough clinical diagnostic evaluation (this may include clinically indicated testing, for example MRI, FDG-PET, MIBG scan, polysomnography, genetic testing, autonomic function tests, inflammatory tests, skin biopsy) the investigators aim to achieve this through:
- Longitudinal tracking of clinical progression through use of clinical scales including at the present time: UMSARS, BARS, MoCA and UPSIT, PROM, MDS-NMS, UPDRS, and SARA
- Longitudinal tracking of disease progression through use of neuroimaging including at the present time: TSPO-PET and 3D MRI (see section 1.3)
This is a pilot study designed to track patients with neurodegenerative movement disorders across Mass General Brigham through MRI and PET imaging modalities and clinical measures. Figure 5 represents the study design in detail. In short, subjects will be asked to visit Mass General Brigham every 6-9 months over the course of 18 months for imaging and clinical evaluation.
Detailed description
After a thorough clinical diagnostic evaluation (this may include clinically indicated testing, for example MRI, FDG-PET, MIBG scan, polysomnography, genetic testing, autonomic function tests, inflammatory tests, skin biopsy) the investigators aim to achieve this through:
- Longitudinal tracking of clinical progression through use of clinical scales including at the present time: UMSARS, BARS, MoCA and UPSIT, PROM, MDS-NMS, UPDRS, and SARA
- Longitudinal tracking of disease progression through use of neuroimaging including at the present time: TSPO-PET and 3D MRI (see section 1.3)
This is a pilot study designed to track patients with neurodegenerative movement disorders across Mass General Brigham through MRI and PET imaging modalities and clinical measures. Figure 5 represents the study design in detail. In short, subjects will be asked to visit Mass General Brigham every 6-9 months over the course of 18 months for imaging and clinical evaluation.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| participants | – | Drug: 18F-PBR06, Radiation: 18F-PBR06all participants in this study must have a diagnosis of a neurodegenerative movement disorder |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Age groups
- Adult, Older adult
- Study population
- individuals with a clinical diagnosis of neurodegenerative movement disorder by consensus criteria (MSA, ataxias, synuclein duplication, atypical parkinsonism, etc)
Key inclusion criteria (2 listed)
- Clinical diagnosis of neurodegenerative movement disorder by consensus criteria including: MSA, ataxias, synuclein duplication, atypical parkinsonism
- Male and female subjects aged 18 and up
Key exclusion criteria (12 listed)
- Individuals with a known alternate neurologic disorder including: idiopathic PD, DLB, PSP, ALS, Alzheimer's, prion disease, frontotemporal dementia, seizure disorder, stroke, or brain tumor
- Individuals with a previous head injury (with 15 minutes or greater loss of consciousness within the past 20 years)
- Individuals with substance abuse, or substance abuse disorder
- Brain MRI indicative of a significant abnormality (i.e. prior hemorrhage or infarct greater than 1 cm3, 3 or more lacunar infarcts, cerebral contusion, encephalomalacia, aneurysm, vascular malformation, subdural hematoma, hydrocephalus, space- occupying lesion).
- Individuals with bipolar disease and schizophrenia
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 |
|---|---|---|
| 18F-PBR06TSPO-PET (translocatior protein, positron emission tomography) scan | Drug | participants |
| 18F-PBR06TSPO-PET (translocatior protein, positron emission tomography) scan | Radiation | participants |
Primary outcome
association between change in PET and clinical measures
Time frame: 18 months
For the longitudinal evaluation, the investigators will estimate the association between the change in the PET and the change in the clinical and morphometric outcome measures using a mixed model with both the baseline PET uptake and the rate of change in PET uptake as predictors. This approach will allow the investigators to estimate both how between and within subject changes in PET impact the outcomes of interest. The investigators will use a p<0.05, corrected for multiple comparisons.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 May 2023.
- Individual participant dataSharing planned: No
Related trials for Neurodegenerative Diseases
Active, not recruitingPhase 1/2
Selumetinib in Treating Young Patients With Recurrent or Refractory Low Grade Glioma
Low Grade Glioma, Recurrent Childhood Pilocytic Astrocytoma
RecruitingPhase 2
Neuroplasticity Enhancement From Cognitive Training Reinforced by Psilocybin
Alzheimer's Disease
Not yet recruiting
Acupressure as Long-term Intervention to Slow the Progression of Early/Mild Alzheimer's Disease
Alzheimer's Disease
Active, not recruitingPhase 4
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy
Muscular Atrophy, Spinal
RecruitingObservational study
Effectiveness and Safety of Disease-Modifying Therapy for Alzheimer's Disease: A Multicenter Retrospective Study
Alzheimer's Disease (AD)
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.
Vikram Khurana, MD PhD contact
Vikram Khurana, MD PhD principal investigator
Brigham and Women's Hospital
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 Neurodegenerative Diseases and sca3, run by Brigham and Women's Hospital 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 2 inclusion and 12 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: status not confirmed, as of the sponsor's last update on 22 May 2023. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Diego Rodriguez, MD (507-491-0272, drodriguez29@bwh.harvard.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
association between change in PET and clinical measures (time frame: 18 months). 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 22 May 2023.