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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:
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.
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:
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| participants | all participants in this study must have a diagnosis of a neurodegenerative movement disorder |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 18F-PBR06 | Drug | TSPO-PET (translocatior protein, positron emission tomography) scan |
| |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 18 months |
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Inclusion Criteria:
Exclusion Criteria:
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individuals with a clinical diagnosis of neurodegenerative movement disorder by consensus criteria (MSA, ataxias, synuclein duplication, atypical parkinsonism, etc)
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Diego Rodriguez, MD | Contact | 507-491-0272 | drodriguez29@bwh.harvard.edu | |
| Vikram Khurana, MD PhD | Contact | vkhurana@bwh.harvard.edu |
| Name | Affiliation | Role |
|---|---|---|
| Vikram Khurana, MD PhD | Brigham and Women's Hospital | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Brigham and Women's Hospital | Recruiting | Boston | Massachusetts | 02115 | United States |
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| ID | Term |
|---|---|
| D019636 | Neurodegenerative Diseases |
| D020966 | Muscular Disorders, Atrophic |
| D012791 | Shy-Drager Syndrome |
| D001259 | Ataxia |
| D000080874 | Synucleinopathies |
| ID | Term |
|---|---|
| D009422 | Nervous System Diseases |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
| D009468 | Neuromuscular Diseases |
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| ID | Term |
|---|---|
| C538798 | N-fluoroacetyl-N-(2,5-dimethoxybenzyl)-2-phenoxyaniline |
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| 18F-PBR06 |
| Radiation |
TSPO-PET (translocatior protein, positron emission tomography) scan |
|
| D019578 | Multiple System Atrophy |
| D054969 | Primary Dysautonomias |
| D001342 | Autonomic Nervous System Diseases |
| D001480 | Basal Ganglia Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009069 | Movement Disorders |
| D007022 | Hypotension |
| D014652 | Vascular Diseases |
| D002318 | Cardiovascular Diseases |
| D020820 | Dyskinesias |
| D009461 | Neurologic Manifestations |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D057165 | Proteostasis Deficiencies |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |