Longitudinal Tracking of Patients Diagnosed With Neurodegenerative Movement Disorders

Neurodegenerative Diseases Clinical Trial

Official title:

Longitudinal Tracking of Patients Diagnosed With Neurodegenerative Movement Disorders

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05486806
• Other study ID #: 2022P001295
• Status: Recruiting
• Start date: March 1, 2023
• Est. completion date: November 15, 2024

Study information

• Verified date: May 2023
• Source: Brigham and Women's Hospital
• Contact: Diego Rodriguez, MD
• Phone: 507-491-0272
• Email: drodriguez29[@]bwh.harvard.edu
• Is FDA regulated: No
• Study type: Observational

Clinical 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. 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 50
• Est. completion date: November 15, 2024
• Est. primary completion date: September 15, 2024
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Ataxia
• Movement Disorders
• MSA - Multiple System Atrophy
• Multiple System Atrophy
• Neurodegenerative Diseases
• sca3
• Shy-Drager Syndrome

Intervention

Drug:
• 18F-PBR06
TSPO-PET (translocatior protein, positron emission tomography) scan

Radiation:
• 18F-PBR06
TSPO-PET (translocatior protein, positron emission tomography) scan

Locations

Country	Name	City	State
United States	Brigham and Women's Hospital	Boston	Massachusetts

Sponsors (1)

Lead Sponsor	Collaborator
Brigham and Women's Hospital

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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