Longitudinal Tracking of Patients Diagnosed With Neurodegenerative Movement Disorders

NCT ID: NCT05486806

Last Updated: 2023-05-22

Basic Information

• Recruitment Status: UNKNOWN
• Total Enrollment: 50 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2023-03-01
• Study Completion Date: 2024-11-15

Brief 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. 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:

1. 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

2. 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:

1. 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

2. 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

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Study Groups

participants

all participants in this study must have a diagnosis of a neurodegenerative movement disorder

18F-PBR06

Intervention Type: DRUG

TSPO-PET (translocatior protein, positron emission tomography) scan

18F-PBR06

Intervention Type: RADIATION

TSPO-PET (translocatior protein, positron emission tomography) scan

Interventions

18F-PBR06

TSPO-PET (translocatior protein, positron emission tomography) scan

Intervention Type: DRUG

18F-PBR06

TSPO-PET (translocatior protein, positron emission tomography) scan

Intervention Type: RADIATION

Eligibility Criteria

Inclusion Criteria

1. Clinical diagnosis of neurodegenerative movement disorder by consensus criteria including: MSA, ataxias, synuclein duplication, atypical parkinsonism

2. Male and female subjects aged 18 and up

Exclusion Criteria

1. 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

2. Individuals with a previous head injury (with 15 minutes or greater loss of consciousness within the past 20 years)

3. Individuals with substance abuse, or substance abuse disorder

4. 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).

5. Individuals with bipolar disease and schizophrenia

6. Concurrent medical conditions that contraindicate study procedures

7. Women who are pregnant, nursing, or seeking to become pregnant

8. Individuals with claustrophobia

9. Non-MRI compatible implanted devices

10. Corticosteroid treatment in the past four weeks

11. Low affinity binders to TSPO

12. Significant cognitive impairment (MoCA score ≤ 23) or poor understanding of study design

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Brigham and Women's Hospital

OTHER

Sponsor Role: lead

Responsible Party

Vikram Khurana, MD PhD

Chief, Division of Movement Disorders

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Vikram Khurana, MD PhD

Role: PRINCIPAL_INVESTIGATOR

Brigham and Women's Hospital

Locations

Brigham and Women's Hospital

Boston, Massachusetts, United States

Site Status: RECRUITING

Countries

United States

Central Contacts

Diego Rodriguez, MD

Role: CONTACT

drodriguez29@bwh.harvard.edu

507-491-0272

Vikram Khurana, MD PhD

Role: CONTACT

vkhurana@bwh.harvard.edu

Facility Contacts

Diego Rodriguez, MD

Role: primary

Other Identifiers

2022P001295

Identifier Type: -

Identifier Source: org_study_id