NCT05042921Active, not recruiting
Pediatric Spinal Muscular Atrophy (SMA) China Registry
Pediatric Patients With Spinal Muscular Atrophy in China: A Nationwide Registry
Source: ClinicalTrials.gov · Last updated 11 Sept 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Active, not recruiting
- Study type
- Observational
- Enrollment
- 600 (actual)
- First submitted
- 8 Sept 2021
- Start date
- 18 Nov 2021
- Primary completion date
- 2 Sept 2027
- Lead sponsor
- Biogen
- Diseases
- Spinal muscular atrophy
- NCT number
- NCT05042921
What you commit to
- Study type: Observational
- Participants enrolled: 600
- From 18 Nov 2021 to the primary completion date, 2 Sept 2027
- 25 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Other
- Unable or unwilling to provide informed consent.
- Other types of SMA (non 5q-SMA).
- NOTE: Other protocol defined inclusion/ exclusion criteria may apply.
Who the study is looking for
- 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.
Age in the registry: - to 18 years · Sex: All · Does not accept healthy volunteers
Sites (25)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Beijing Children's Hospital, Capital Medical University Coordinator of this site? Confirm its status | Beijing | China | Unknown status |
| The First Hospital of Jilin University Coordinator of this site? Confirm its status | Changchun | China | Unknown status |
| Hunan Children's Hospital Coordinator of this site? Confirm its status | Changsha | China | Unknown status |
| Xiangya Hospital Central South University Coordinator of this site? Confirm its status | Changsha | China | Unknown status |
| West China Second University Hospital, Sichuan University Coordinator of this site? Confirm its status | Chengdu | China | Unknown status |
| Children's Hospital of Chongqing Medical University Coordinator of this site? Confirm its status | Chongqing | China | Unknown status |
| Dalian Women and Children's Medical Group Coordinator of this site? Confirm its status | Dalian | China | Unknown status |
| Fujian Medical University Union Hospital Coordinator of this site? Confirm its status | Fuzhou | China | Unknown status |
| The First Affiliated Hospital, Sun Yat-sen University Coordinator of this site? Confirm its status | Guangzhou | China | Unknown status |
| The Children's Hospital, Zhejiang University School of Medicine Coordinator of this site? Confirm its status | Hangzhou | China | Unknown status |
| Anhui Children's Hospital Coordinator of this site? Confirm its status | Hefei | China | Unknown status |
| The Affiliated Hospital of Inner Mongolia Medical University Coordinator of this site? Confirm its status | Hohhot | China | Unknown status |
| Children's Hospital Affiliated to Shandong University Coordinator of this site? Confirm its status | Jinan | China | Unknown status |
| Jiangxi Provincial Children's Hospital Coordinator of this site? Confirm its status | Nanchang | China | Unknown status |
| Maternity and Child Health Care of Guangxi Zhuang Autonomous Region Coordinator of this site? Confirm its status | Nanning | China | Unknown status |
| Children's Hospital Of Fudan University Coordinator of this site? Confirm its status | Shanghai | China | Unknown status |
| Shengjing Hospital Of China Medical University Coordinator of this site? Confirm its status | Shenyang | China | Unknown status |
| Shenzhen Children's Hospital Coordinator of this site? Confirm its status | Shenzhen | China | Unknown status |
| The Second Hospital of Hebei Medical University Coordinator of this site? Confirm its status | Shijiazhuang | China | Unknown status |
| Wuhan Children's Hospital, Tongji Medical College, Huazhong University of Science & Technology Coordinator of this site? Confirm its status | Wuhan | China | Unknown status |
| Xiamen Maternal and Child Health Hospital Coordinator of this site? Confirm its status | Xiamen | China | Unknown status |
| The Second Affiliated Hospital of Xi'an Jiaotong University Coordinator of this site? Confirm its status | Xi'an | China | Unknown status |
| Xuzhou Central Hospital Coordinator of this site? Confirm its status | Xuzhou | China | Unknown status |
| People's Hospital of Ningxia Hui Autonomous Region Coordinator of this site? Confirm its status | Yinchuan | China | Unknown status |
| The Third Affiliated Hospital of Zhengzhou University Coordinator of this site? Confirm its status | Zhengzhou | China | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.