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/33554315Actively Recruiting
Led by NYU Langone Health · Updated on 2025-06-11
800
Participants Needed
8
Research Sites
N/A
Total Duration
Synucleinopathies are rare neurodegenerative diseases that involve abnormal buildup of the protein alpha-synuclein in the nervous system, often leading to worsening neurological problems. These diseases usually start in late adulthood, typically after age 50, and can cause movement slowing, coordination issues, or mild cognitive problems. Common diagnoses include Parkinson disease (PD), dementia with Lewy bodies (DLB), and multiple system atrophy (MSA). In some cases, symptoms begin with autonomic nerve issues such as unexplained constipation or urinary problems before brain involvement develops. This study aims to understand how these diseases progress and identify biological markers that predict their evolution. The study is observational and follows patients diagnosed with synucleinopathies, including those in the early "pre-motor" stage showing mainly autonomic impairments or REM sleep behavior disorder (RBD). Participants will be monitored over time through a series of follow-up visits at participating centers. The study will document clinical features, track disease markers, and define the natural history of MSA, which is noted as the most aggressive form. Different patient groups include those with RBD, MSA, pure autonomic failure, PD, and DLB. Participants will undergo clinical evaluations to observe symptoms and disease progression. Researchers will collect data on autonomic function and biomarkers to help predict which patients develop motor or cognitive problems. The primary goal is to create a detailed database of autonomic disorders over five years, with a focus on defining disease milestones and identifying predictive biomarkers. This long-term monitoring will help guide future clinical trials and improve understanding of these complex disorders.
CONDITIONS
Natural History Study of Synucleinopathies
You may qualify if you...
You will not qualify if you...
Complete this quick 3-step screening to check your eligibility
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 5 years
Participants are observed over time to understand the natural progression of synucleinopathies and to identify biomarkers predicting disease spread.
Regular follow-up visits over the study period
Total: 8 locations
1
Beth Israel Deaconess Medical Center
Boston, Massachusetts, United States
Actively Recruiting
2
University of Michigan
Ann Arbor, Michigan, United States
Actively Recruiting
3
Mayo Clinic
Rochester, Minnesota, United States
Actively Recruiting
4
NYU Medical Center
New York, New York, United States, 10016
Actively Recruiting
5
Vanderbilt Univeristy
Nashville, Tennessee, United States
Actively Recruiting
6
FLENI - Fundación para la Lucha contras las Enfermedades Neurológicas
Buenos Aires, Argentina
Actively Recruiting
7
Seoul National University Hospital
Seoul, South Korea
Actively Recruiting
8
BioCruces Research Institute - Hospital Universitario de Cruces
Bilbao, Spain
Actively Recruiting
H
Horacio Kaufmann, MD
G
Grace Nkrumah
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
5
Have more questions? Get in touch with our team for quick support
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here
Jose-Alberto Palma, Patricio Millar Vernetti, Miguel A Perez...
https://pubmed.ncbi.nlm.nih.gov/33554315Lucy Norcliffe-Kaufmann, Horacio Kaufmann, Jose-Alberto Palma...
https://pubmed.ncbi.nlm.nih.gov/29405350