Limitations of the Unified Multiple System Atrophy Rating Scale as outcome measure for clinical trials and a roadmap for improvement.
Jose-Alberto Palma, Patricio Millar Vernetti, Miguel A Perez...
https://pubmed.ncbi.nlm.nih.gov/33554315/Actively Recruiting
Led by NYU Langone Health · Updated on 2026-06-10
800
Participants Needed
8
Research Sites
N/A
Total Duration
Synucleinopathies are rare neurodegenerative diseases characterized by worsening neurological problems and abnormal buildup of the protein alpha-synuclein in the nervous system. They usually begin after age 50 and often cause slow movement, coordination issues, or mild cognitive decline. Conditions such as Parkinson disease, dementia with Lewy bodies, and multiple system atrophy fall under synucleinopathies. In some cases, symptoms start with autonomic nerve problems like unexplained constipation or low blood pressure upon standing before affecting the brain. This observational study follows people diagnosed with synucleinopathies or those in an early pre-motor phase showing only autonomic symptoms or REM sleep behavior disorder. Participants will have regular visits at specialized centers to document their clinical features and collect biological markers. The goal is to understand how these diseases progress, identify markers that predict brain involvement, and define the natural history of multiple system atrophy. Participants will undergo thorough evaluations including clinical assessments and biological testing over time. Researchers will monitor symptoms, neurological function, and autonomic signs to track disease progression. The study aims to create a detailed database of patients with autonomic disorders and identify biomarkers that may predict disease spread. Follow-up will help clarify which patients develop motor or cognitive problems and which remain stable, aiding future clinical trials and treatments.
CONDITIONS
Natural History Study of Synucleinopathies
H
Horacio Kaufmann, MD
G
Grace Nkrumah
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NYU Langone Health
Lead Sponsor
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Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
5
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Jose-Alberto Palma, Patricio Millar Vernetti, Miguel A Perez...
https://pubmed.ncbi.nlm.nih.gov/33554315/Lucy Norcliffe-Kaufmann, Horacio Kaufmann, Jose-Alberto Palma...
https://pubmed.ncbi.nlm.nih.gov/29405350/NYU Langone Health
Lead Sponsor