Pediatric Spinal Muscular Atrophy (SMA) China Registry
Pediatric Patients With Spinal Muscular Atrophy in China: A Nationwide Registry
1 other identifier
observational
600
1 country
25
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for all trials
Started Nov 2021
Longer than P75 for all trials
25 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
September 8, 2021
CompletedFirst Posted
Study publicly available on registry
September 13, 2021
CompletedStudy Start
First participant enrolled
November 18, 2021
CompletedPrimary Completion
Last participant's last visit for primary outcome
September 2, 2027
ExpectedStudy Completion
Last participant's last visit for all outcomes
September 2, 2027
September 3, 2025
September 1, 2025
5.8 years
September 8, 2021
September 2, 2025
Conditions
Outcome Measures
Primary Outcomes (1)
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
Other Outcomes (2)
Effectiveness of DMT Treatments
Up to 60 months
Safety of DMT Treatments
Up to 60 months
Study Arms (2)
DMT Treated Participants
Participants with SMA who received prior treatment with DMTs including nusinersen will be followed prospectively for up to 60 months and the available data is collected retrospectively.
Untreated Participants
Participants with SMA who received no treatment will be followed prospectively for up to 60 months.
Eligibility Criteria
Pediatric participants with 5q-SMA will be enrolled to obtain information on both the natural history of the disease and effectiveness and safety of DMTs.
You may qualify if:
- 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.
You may not qualify if:
- Unable or unwilling to provide informed consent.
- Other types of SMA (non 5q-SMA).
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Biogenlead
Study Sites (25)
Anhui Children's Hospital
Hefei, Anhui, 230006, China
Beijing Children's Hospital, Capital Medical University
Beijing, Beijing Municipality, 100045, China
Children's Hospital of Chongqing Medical University
Chongqing, Chongqing Municipality, 400014, China
Fujian Medical University Union Hospital
Fuzhou, Fujian, 350001, China
Xiamen Maternal and Child Health Hospital
Xiamen, Fujian, 361003, China
The First Affiliated Hospital, Sun Yat-sen University
Guangzhou, Guangdong, 510080, China
Shenzhen Children's Hospital
Shenzhen, Guangdong, 518026, China
Maternity and Child Health Care of Guangxi Zhuang Autonomous Region
Nanning, Guangxi Zhuang, 530002, China
The Second Hospital of Hebei Medical University
Shijiazhuang, Hebei, 050000, China
The Third Affiliated Hospital of Zhengzhou University
Zhengzhou, Henan, 450052, China
Hunan Children's Hospital
Changsha, Hu'nan, 410007, China
Xiangya Hospital, Central South University
Changsha, Hu'nan, 410008, China
Wuhan Children's Hospital, Tongji Medical College Huazhong University of Science & Technology
Wuhan, Hubei, 430010, China
The Affiliated Hospital of Inner Mongolia Medical University
Hohhot, Inner Mongolia, 010050, China
Xuzhou Central Hospital
Xuzhou, Jiangsu, 221009, China
Jiangxi Provincial Children's Hospital
Nanchang, Jiangxi, 330006, China
The First Hospital of Jilin University
Changchun, Jilin, 130021, China
Children's Hospital Affiliated to Shandong University
Jinan, Jinan Shandong, 250022, China
Dalian Women and Children's Medical Group
Dalian, Liaoning, 116037, China
Shengjing Hospital of China Medical University
Shenyang, Liaoning, 110022, China
People's Hospital of Ningxia Hui Autonomous Region
Yinchuan, Ningxia Hui, 750002, China
Children's Hospital of Fudan University
Shanghai, Shanghai Municipality, 201102, China
The Second Affiliated Hospital of Xi'An Jiaotong University
Xi’an, Shanxi, 710004, China
West China Second University Hospital, Sichuan University
Chengdu, Sichuan, 610041, China
The Children's Hospital, Zhejiang University School of Medicine
Hangzhou, Zhejiang, 310052, China
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Medical Director
Biogen
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Target Duration
- 60 Months
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
September 8, 2021
First Posted
September 13, 2021
Study Start
November 18, 2021
Primary Completion (Estimated)
September 2, 2027
Study Completion (Estimated)
September 2, 2027
Last Updated
September 3, 2025
Record last verified: 2025-09
Data Sharing
- IPD Sharing
- Will share
In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/