Longitudinal Tracking of Patients Diagnosed With Neurodegenerative Movement Disorders

NCT05486806 | Unknown FDA Drug

• 1 other identifier: 2022P001295
• Study Type: observational
• Target: 50
• Locations: 1 country
• Sites: 1

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. 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. 2.Longitudinal tracking of disease progression through use of neuroimaging including at the present time: TSPO-PET and 3D MRI (see section 1.3)

Conditions

Neurodegenerative Diseases; sca3; MSA - Multiple System Atrophy; Ataxia; Synucleinopathies

Outcome Measures

Primary Outcomes (1)

• 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

Study Arms (1)

participants

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

Drug: 18F-PBR06; Radiation: 18F-PBR06

Interventions

18F-PBR06 DRUG

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

participants

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

individuals with a clinical diagnosis of neurodegenerative movement disorder by consensus criteria (MSA, ataxias, synuclein duplication, atypical parkinsonism, etc)

You may qualify if:

• Clinical diagnosis of neurodegenerative movement disorder by consensus criteria including: MSA, ataxias, synuclein duplication, atypical parkinsonism
• Male and female subjects aged 18 and up

You may not qualify if:

• 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
• 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

Sponsors & Collaborators

• Brigham and Women's Hospital: lead

Study Sites (1)

Brigham and Women's Hospital

Boston, Massachusetts, 02115, United States

RECRUITING

MeSH Terms

Conditions

Neurodegenerative Diseases; Muscular Disorders, Atrophic; Shy-Drager Syndrome; Ataxia; Synucleinopathies

Interventions

N-fluoroacetyl-N-(2,5-dimethoxybenzyl)-2-phenoxyaniline

Condition Hierarchy (Ancestors)

Nervous System Diseases; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Multiple System Atrophy; Primary Dysautonomias; Autonomic Nervous System Diseases; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Movement Disorders; Hypotension; Vascular Diseases; Cardiovascular Diseases; Dyskinesias; Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Vikram Khurana, MD PhD

  Brigham and Women's Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Diego Rodriguez, MD

CONTACT

507-491-0272; drodriguez29@bwh.harvard.edu

Vikram Khurana, MD PhD

CONTACT

vkhurana@bwh.harvard.edu

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Chief, Division of Movement Disorders

Study Record Dates

• First Submitted: August 2, 2022
• First Posted: August 4, 2022
• Study Start: March 1, 2023
• Primary Completion: September 15, 2024
• Study Completion: November 15, 2024
• Last Updated: May 22, 2023

Record last verified: 2023-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)