Pediatric Spinal Muscular Atrophy (SMA) China Registry
- Conditions
- Muscular Atrophy, Spinal
- Registration Number
- NCT05042921
- Lead Sponsor
- Biogen
- Brief Summary
The primary objective of the study is to describe the natural history and utilization of disease modifying therapy (DMT) treatments among pediatric Chinese participants with spinal muscular atrophy linked to chromosome 5q (5q-SMA). The study will examine SMA natural history and DMT outcomes in a real-world setting both prospectively and retrospectively.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 600
- Ability of the participant and/or his/her legally authorized representative (e.g., parent or legal guardian), as appropriate and applicable, to understand the purpose and risks of the study, to provide informed consent, and to authorize the use of confidential health information in accordance with national and local privacy regulations.
- Genetically confirmed 5q-SMA.
- Age < 18 years at enrollment.
Key
- Unable or unwilling to provide informed consent.
- 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 Natural History and Utilization of Disease Modifying Therapy (DMT) Treatments Among Pediatric Chinese Participants With Spinal Muscular Atrophy Linked to Chromosome 5q (5q-SMA) Up to 60 months
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (25)
Anhui Children's Hospital
🇨🇳Hefei, Anhui, China
Beijing Children's Hospital, Capital Medical University
🇨🇳Beijing, Beijing Municipality, China
Children's Hospital of Chongqing Medical University
🇨🇳Chongqing, Chongqing Municipality, China
Fujian Medical University Union Hospital
🇨🇳Fuzhou, Fujian, China
Xiamen Maternal and Child Health Hospital
🇨🇳Xiamen, Fujian, China
The First Affiliated Hospital, Sun Yat-sen University
🇨🇳Guangzhou, Guangdong, China
Shenzhen Children's Hospital
🇨🇳Shenzhen, Guangdong, China
Maternity and Child Health Care of Guangxi Zhuang Autonomous Region
🇨🇳Nanning, Guangxi Zhuang, China
The Second Hospital of Hebei Medical University
🇨🇳Shijiazhuang, Hebei, China
The Third Affiliated Hospital of Zhengzhou University
🇨🇳Zhengzhou, Henan, China
Scroll for more (15 remaining)Anhui Children's Hospital🇨🇳Hefei, Anhui, China