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Clinical Trials in China / NCT05042921
Active, not recruiting Observational

Pediatric Spinal Muscular Atrophy (SMA) China Registry

NCT05042921 · tracked via the Priya Life Science China tracker
Sponsor
Biogen
Phase
Observational
Started
2021-11-18
Last updated
2026-09-11

Condition(s) studied

Muscular Atrophy, Spinal

Study 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.

Eligibility

Sex
ALL
Min age
—
Max age
18 Years
Healthy volunteers
No
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.

Primary outcome measure(s)

  • 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

Trial sites (25)

FacilityCityRegionStatus
Anhui Children's Hospital Hefei Anhui
Beijing Children's Hospital, Capital Medical University Beijing Beijing Municipality
Children's Hospital of Chongqing Medical University Chongqing Chongqing Municipality
Fujian Medical University Union Hospital Fuzhou Fujian
Xiamen Maternal and Child Health Hospital Xiamen Fujian
The First Affiliated Hospital, Sun Yat-sen University Guangzhou Guangdong
Shenzhen Children's Hospital Shenzhen Guangdong
Maternity and Child Health Care of Guangxi Zhuang Autonomous Region Nanning Guangxi Zhuang
The Second Hospital of Hebei Medical University Shijiazhuang Hebei
The Third Affiliated Hospital of Zhengzhou University Zhengzhou Henan
Hunan Children's Hospital Changsha Hu'nan
Xiangya Hospital, Central South University Changsha Hu'nan
Wuhan Children's Hospital, Tongji Medical College Huazhong University of Science & Technology Wuhan Hubei
The Affiliated Hospital of Inner Mongolia Medical University Hohhot Inner Mongolia
Xuzhou Central Hospital Xuzhou Jiangsu
Jiangxi Provincial Children's Hospital Nanchang Jiangxi
The First Hospital of Jilin University Changchun Jilin
Children's Hospital Affiliated to Shandong University Jinan Jinan Shandong
Dalian Women and Children's Medical Group Dalian Liaoning
Shengjing Hospital of China Medical University Shenyang Liaoning
People's Hospital of Ningxia Hui Autonomous Region Yinchuan Ningxia Hui
Children's Hospital of Fudan University Shanghai Shanghai Municipality
The Second Affiliated Hospital of Xi'An Jiaotong University Xi’an Shanxi
West China Second University Hospital, Sichuan University Chengdu Sichuan
The Children's Hospital, Zhejiang University School of Medicine Hangzhou Zhejiang

More Biogen trials in China

Adult Spinal Muscular Atrophy (SMA) China Registry Active, not recruiting
A Study to Learn About the Effects of Felzartamab Infusions on Adults With Immunoglobulin A Nephropathy (IgAN) Recruiting
A Study to Learn About the Safety of Litifilimab (BIIB059) Injections and Whether They Can Improve Symptoms of Adult Participants Who Have Systemic Lupus Erythematosus Active, not recruiting
A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety in Participants Aged 15 to 60 Years With Spinal Muscular Atrophy (SMA) Who Are Either New to SMA Treatment or Were Previously Treated With Risdiplam Recruiting
A Study to Learn More About the Long-Term Safety of Litifilimab (BIIB059) Injections and it's Effects on Symptoms in Adults With Systemic Lupus Erythematosus (SLE) Enrolling by invitation

Other trials for the same condition

Adult Spinal Muscular Atrophy (SMA) China Registry Active, not recruiting
A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety When Given Before Symptoms Appear in Babies With Genetically Diagnosed Spinal Muscular Atrophy (SMA) Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05042921 on ClinicalTrials.gov ↗ ← All trials in China