TRACK-MSA: A Longitudinal Study to Define Outcome Measures in Multiple System Atrophy

NCT04450992 | Completed

• 1 other identifier: S19-01846
• Study Type: observational
• Target: 29
• Locations: 1 country
• Sites: 1

Brief Summary

TRACK-MSA is an observational, non-interventional, longitudinal natural history study to define changes in clinical, neurological, blood, CSF, and neuroimaging biomarkers in patients with multiple system atrophy (MSA) comparing baseline to 6-month and 1-year assessments. The study will enroll 50 patients with MSA-P or MSA-C at 2 or more participating sites.

Conditions

Multiple System Atrophy

Outcome Measures

Primary Outcomes (4)

• Brain MRI imaging data

  Diffusion-based MRI data

  5 years

• neurofilament light chain (NfL) in serum and/or plasma

  Blood samples for measure measurement of NfL

  5 years

• Cerebral spinal fluid (CSF) measures

  measurement of alpha synuclein, aggregated alpha synuclein Tau/phosphorylated Tau, neurofilament light chain, neurofilament heavy chain, and catecholamines in CSF

  5 years

• Quantitative movement assessment

  The quantitative movement assessment comprises a battery of tests performed with non-invasive motion sensors, to capture disease features associated with striatonigral degeneration; others are aimed at capturing features associated with olivopontocerebellar degeneration observed in MSA patients.

  5 years

Eligibility Criteria

• Age: 40 Years - 80 Years
• Sex: all
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Multiple system atrophy

You may qualify if:

• \- 1. Ability to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use confidential health information in accordance with applicable privacy regulations 2. Diagnosis of probable or possible multiple system atrophy (MSA-P or MSA-C) according to current consensus diagnostic criteria \[8\].
• \. Aged 40-80 with anticipated survival of at least 3 years (in the opinion of the Investigator) 4. Able to walk for, at least, 10 meters with or without assistance 5. Willingness and ability to participate in all study procedures. 6. Ability to tolerate and no contraindications for brain MRI. 7. Ability to tolerate and no contraindications for CSF collection. Participants with contraindication to the CSF procedure will be given the option to participate in the study and opt out the CSF collection. An effort will be made to ensure that at least 50% of patients enrolled in the study undergo CSF collection.

You may not qualify if:

• \. Presence of supranuclear gaze palsy. 2. Known presence of hyposmia (i.e., reduced ability to smell and detect odors).
• \. Presence of cognitive dysfunction (defined as MoCA score \<20). 4. Severe-to-complete dependence on caregivers (score \>3 on UMSARS Part IV, Global Disability), severe impairment of swallowing (score ≥3 on UMSARS Part I, Question 2), or frequent falls (score ≥3 on UMSARS Part I, Question 8) at Baseline.
• \. Family history or a known genetic cause of ataxia or parkinsonism. 6. Clinically significant neuropathy. 7. Hallucinations not induced by drugs. 8. Unstable psychiatric illness, including psychosis, suicidal ideation, or untreated major depression within 90 days before Baseline, as determined by the Investigator.
• \. History or Baseline MRI results showing evidence of structural abnormalities that could contribute to the participant's clinical state other than findings typical of MSA, or any finding that might pose a risk to the participant.
• \. Any contraindications to having a brain MRI (e.g., pacemaker; MRI-incompatible aneurysm clips, artificial heart valves, or other metal foreign body; claustrophobia that cannot be medically managed without requiring general anesthesia, etc.).
• \. Transient ischemic attack or stroke, or any unexplained loss of consciousness within 1 year before Baseline.
• \. History of any brain surgery for MSA (e.g., pallidotomy, deep brain stimulation, or fetal tissue transplant) or a history of focused ultrasound treatment or neuromodulation procedures, including but not limited to transcranial magnetic stimulation (TMS) and transcranial direct or alternating current stimulation (tDCS/tACS) that have been performed within 90 days of Baseline.
• Infection Risk 13. History of human immunodeficiency virus or hepatitis C virus antibody. 14. Chronic, recurrent, or serious infection (e.g., pneumonia, septicemia, recurrent urinary tract infection), as determined by the Investigator, within 8 weeks before Day 1.
• Cardiovascular 15. History of unstable angina, myocardial infarction, chronic heart failure (New York Heart Association Class 3 or 4), or clinically significant conduction abnormalities (e.g., unstable atrial fibrillation) within 1 year before Baseline.
• \. Chronic, sustained, uncontrolled supine hypertension (unrelated to pharmacological treatment of orthostatic hypotension) defined by an average of three SBP readings of \>180 mmHg or DBP readings of \>110 mmHg at baseline.
• \. In participants treated pharmacologically for orthostatic hypotension, any documented sitting or standing SBP reading ≥180 mmHg or DBP reading ≥110 mmHg within the 3 months before Day 1 or on Day 1).
• \. Severe orthostatic hypotension despite optimal medical management (defined as a score of ≥3 on UMSARS Part I, Question 9).
• Oncology 19. History of, or ongoing, malignant disease, including solid tumors and hematologic malignancies (with the exception of basal cell carcinomas and squamous cell carcinomas that have been completely excised and considered cured at least 12 months prior to Day -1). Participants with cancers in remission for greater than 5 years prior to Day -1 may be included.
• Metabolic 20. Poorly controlled diabetes mellitus, as defined by having dosage adjustment of diabetic medication within 3 months before dosing (Day 1) or glycated hemoglobin value ≥8% at Baseline.
• Hypersensitivity 21. Clinically significant allergies, as determined by the Investigator, to anesthetics that will be used for the LP per institutional practice, or iodine.

Sponsors & Collaborators

• NYU Langone Health: lead

Study Sites (1)

NYU Langone Medical Center

New York, New York, 10016, United States

Location

Biospecimen

Retention: SAMPLES WITHOUT DNA

Biomarker analysis will be focused on measuring the levels of neurofilament light chain (NfL) in serum and/or plasma, which is one of the most promising blood biomarkers in MSA. Analysis of CSF may include but is not limited to the following biomarkers: total alpha synuclein, aggregated alpha synuclein Tau/phosphorylated Tau, neurofilament light chain, neurofilament heavy chain, and catecholamines (dopamine, norepinephrine, DOPAC, DHPG).

MeSH Terms

Conditions

Multiple System Atrophy

Condition Hierarchy (Ancestors)

Primary Dysautonomias; Autonomic Nervous System Diseases; Nervous System Diseases; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases

Study Officials

• Jose-Alberto Palma, MD PhD

  NYU School of Medicine

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 25, 2020
• First Posted: June 30, 2020
• Study Start: June 17, 2020
• Primary Completion: June 19, 2024
• Study Completion: June 19, 2024
• Last Updated: July 31, 2024

Record last verified: 2024-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)