Adult Spinal Muscular Atrophy (SMA) China Registry
- Conditions
- Muscular Atrophy, Spinal
- Registration Number
- NCT05618379
- Lead Sponsor
- Biogen
- Brief Summary
The primary objective of the study is to describe the natural history and utilization of disease modifying therapy (DMT) among adult Chinese participants with SMA linked to chromosome 5q (5q-SMA).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 200
-Genetically confirmed 5q-SMA.
Key
-Other types of SMA (non 5q-SMA).
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Number of Participants Characterized by Utilization of Disease Modifying Therapies (DMTs) of 5q-SMA Observed Among Adult Chinese Participants Up to 60 Months The DMT utilization characteristics will include the categories of the evolution of the DMT treated patient's condition over the time of data collection from the registry, to describe the utilization of DMTs of adult SMA patients in a real-life situation.
Number of Participants Characterized by Natural History of 5q-SMA Observed Among Adult Chinese Participants Up to 60 Months The natural history characteristics will include the categories of the evolution of the patient's condition over the time of data collection from the registry, to describe the natural history of the disease in a real-life situation.
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (12)
The First People's Hospital of Yunnan Province
🇨🇳Kunming, Yunnan, China
Huashan Hospital, Fudan University
🇨🇳Shanghai, Shanghai, China
Peking Union Medical College Hospital
🇨🇳Beijing, Beijing, China
The First Affiliated Hospital, Sun Yat-sen University
🇨🇳Guangzhou, Guangdong, China
The First Affiliated Hospital of Fujian Medical University
🇨🇳Fuzhou, Fujian, China
The First Affiliated Hospital of Soochow University
🇨🇳Suzhou, Jiangsu, China
The Second Hospital of Hebei Medical University
🇨🇳Shijiazhuang, Hebei, China
Peking University Shenzhen Hospital
🇨🇳Shenzhen, Guangdong, China
Xiangya Hospital of Central South University
🇨🇳Changsha, Hunan, China
The First Affiliated Hospital of Nanchang University
🇨🇳Nanchang, Jiangxi, China
Qilu Hospital of Shandong University
🇨🇳Jinan, Shandong, China
West China Hospital, Sichuan University
🇨🇳Chengdu, Sichuan, China