Active, Not RecruitingN/AOther
Pediatric Spinal Muscular Atrophy (SMA) China Registry
Sponsored by Biogen
NCT ID
NCT05042921
Target Enrollment
600 participants
Start Date
2021-11-18
Est. Completion
2027-09-02
About This Study
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.
Conditions Studied
Eligibility
Age:N/A - 18 Years
Healthy Volunteers:No
View full eligibility criteria
Key Inclusion Criteria: * 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 Exclusion Criteria: * Unable or unwilling to provide informed consent. * Other types of SMA (non 5q-SMA). NOTE: Other protocol defined inclusion/ exclusion criteria may apply.
Study 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
+15 more locations