Prospective Multicenter Registry Study of Multiple System Atrophy in China (MSA Registry S)
Clinical Features and Natural History of Multiple System Atrophy: A Prospective Multicenter Registry Study in China
Multiple system atrophy is a rare, rapidly progressive neurodegenerative disease characterized by variable combinations of parkinsonism, cerebellar ataxia, and autonomic dysfunction. Existing natural history studies from North America, Europe, and Japan suggest that clinical phenotypes and disease progression may differ across populations. However, comprehensive multicenter prospective data from Chinese patients with multiple system atrophy remain limited.
This prospective multicenter registry study aims to describe the clinical characteristics, longitudinal progression, and outcomes of Chinese patients with multiple system atrophy, to identify factors associated with disease progression and prognosis, and to establish a longitudinal cohort for future biomarker validation and clinical trial design.
Visão geral do estudo
Status
Descrição detalhada
Multiple system atrophy is an adult-onset, progressive neurodegenerative disorder characterized by parkinsonism, cerebellar ataxia, autonomic dysfunction, and variable non-motor manifestations. The disease is pathologically associated with alpha-synuclein accumulation and neuronal and glial degeneration in multiple brain regions. Due to its rarity, clinical heterogeneity, rapid progression, and poor prognosis, large-scale prospective studies are needed to better define its natural history and to support future therapeutic development.
This study is a prospective, observational, multicenter registry study conducted in China. Eligible participants will include patients with clinically established or clinically probable multiple system atrophy according to the 2022 Movement Disorder Society diagnostic criteria. Parkinson disease patients and healthy or non-neurodegenerative controls may also be enrolled for comparative analyses.
Data will be collected through in-person visits, medical record review, standardized clinical scales, neurological examinations, autonomic function testing, neuroimaging, laboratory tests, and biospecimen collection. Longitudinal follow-up will be performed at prespecified time points, including alternating in-person and telephone-based assessments when applicable. Clinical scales may include the Unified Multiple System Atrophy Rating Scale, Movement Disorder Society-sponsored Unified Parkinson's Disease Rating Scale, non-motor symptom scales, autonomic symptom scales, and disability measures. Neuroimaging, autonomic function tests, electrophysiological or oculomotor evaluations, and biospecimen-based analyses may be performed according to the study protocol and local clinical practice.
The main objectives are to characterize the clinical features and longitudinal disease course of Chinese patients with multiple system atrophy, compare clinical characteristics between MSA-P and MSA-C subtypes, identify clinical and paraclinical factors associated with disease progression and prognosis, and establish a longitudinal platform for subsequent biomarker validation and clinical trial design.
Tipo de estudo
Inscrição (Estimado)
Contactos e Locais
Contato de estudo
- Nome: Yunchuang Sun, MD
- E-mail: sychuang0805@163.com
Locais de estudo
-
-
Beijing Municipality
-
Beijing, Beijing Municipality, China, 100034
- Recrutamento
- Peking University First Hospital
-
Contato:
- Yang Zhao
- Número de telefone: (+86)18610320188
- E-mail: zhaoyang2019@pku.edu.cn
-
-
Critérios de participação
Critérios de elegibilidade
Idades elegíveis para estudo
- Adulto
- Adulto mais velho
Aceita Voluntários Saudáveis
Método de amostragem
População do estudo
Descrição
Inclusion Criteria
- Patients with clinically established or clinically probable multiple system atrophy according to the 2022 Movement Disorder Society diagnostic criteria; or
- Patients with clinically established or clinically probable Parkinson disease according to the Movement Disorder Society diagnostic criteria; or
- Healthy controls or controls without hereditary or neurodegenerative diseases who voluntarily agree to participate.
- Age between 40 and 75 years.
- Ability to provide informed consent or availability of a legally authorized representative when applicable.
Exclusion Criteria
- Parkinsonism that cannot be classified as Parkinson disease or multiple system atrophy at the time of evaluation.
- Clinical suspicion or diagnosis of other atypical parkinsonian syndromes, including progressive supranuclear palsy, dementia with Lewy bodies, or corticobasal syndrome.
- Secondary parkinsonism due to intracranial space-occupying lesions, normal pressure hydrocephalus, drug-induced parkinsonism, or other identifiable causes.
- Comorbid diseases that may substantially affect autonomic function, such as diabetic peripheral neuropathy or amyloidosis.
- Refusal to participate in the study or refusal to undergo routine clinical evaluations for parkinsonian syndromes.
- Psychiatric or behavioral abnormalities that preclude reliable clinical data collection or scale-based assessment.
Plano de estudo
Como o estudo é projetado?
Detalhes do projeto
Coortes e Intervenções
Grupo / Coorte |
|---|
|
MSA
|
|
PD
|
|
HC
Healthy control / Control without neurodegenerative diseases
|
O que o estudo está medindo?
Medidas de resultados primários
Medida de resultado |
Descrição da medida |
Prazo |
|---|---|---|
|
Change in disease severity
Prazo: Baseline to up to 36 months after enrollment.
|
Change in disease severity as measured by the Unified Multiple System Atrophy Rating Scale over longitudinal follow-up.
|
Baseline to up to 36 months after enrollment.
|
Colaboradores e Investigadores
Patrocinador
Investigadores
- Investigador principal: Zhaoxia Wang, MD, Department of Neurology, Peking University First Hospital
Datas de registro do estudo
Datas Principais do Estudo
Início do estudo (Real)
Conclusão Primária (Estimado)
Conclusão do estudo (Estimado)
Datas de inscrição no estudo
Enviado pela primeira vez
Enviado pela primeira vez que atendeu aos critérios de CQ
Primeira postagem (Real)
Atualizações de registro de estudo
Última Atualização Postada (Real)
Última atualização enviada que atendeu aos critérios de controle de qualidade
Última verificação
Mais Informações
Termos relacionados a este estudo
Palavras-chave
Termos MeSH relevantes adicionais
- Sinucleinopatias
- Doenças Cerebrais
- Doenças do Sistema Nervoso Central
- Doenças do Sistema Nervoso
- Processos Patológicos
- Atributos da doença
- Doenças Neurodegenerativas
- Distúrbios do Movimento
- Distúrbios parkinsonianos
- Doenças dos Gânglios da Base
- Disautonomias primárias
- Doenças do Sistema Nervoso Autônomo
- Condições Patológicas, Sinais e Sintomas
- Progressão da doença
- Doença de Parkinson
- Atrofia de Múltiplos Sistemas
- Doença de Parkinson 4, Corpo Autossômico Dominante de Lewy
Outros números de identificação do estudo
- MSARegistryStudy-20250302
Plano para dados de participantes individuais (IPD)
Planeja compartilhar dados de participantes individuais (IPD)?
Informações sobre medicamentos e dispositivos, documentos de estudo
Estuda um medicamento regulamentado pela FDA dos EUA
Estuda um produto de dispositivo regulamentado pela FDA dos EUA
Essas informações foram obtidas diretamente do site clinicaltrials.gov sem nenhuma alteração. Se você tiver alguma solicitação para alterar, remover ou atualizar os detalhes do seu estudo, entre em contato com register@clinicaltrials.gov. Assim que uma alteração for implementada em clinicaltrials.gov, ela também será atualizada automaticamente em nosso site .