Pediatric Spinal Muscular Atrophy (SMA) China Registry

Last updated: September 2, 2025
Sponsor: Biogen
Overall Status: Active - Not Recruiting

Phase

N/A

Condition

Myasthenia Gravis (Chronic Weakness)

Muscular Dystrophy

Spinal Muscular Atrophy

Treatment

N/A

Clinical Study ID

NCT05042921
CN-SMG-11774
  • Ages < 18
  • All Genders

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 Criteria

Inclusion

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 purposeand risks of the study, to provide informed consent, and to authorize the use ofconfidential health information in accordance with national and local privacyregulations.

  • Genetically confirmed 5q-SMA.

  • Age < 18 years at enrollment.

Exclusion

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 Design

Total Participants: 600
Study Start date:
November 18, 2021
Estimated Completion Date:
September 02, 2027

Connect with a study center

  • Anhui Children's Hospital

    Hefei, Anhui 230006
    China

    Site Not Available

  • Anhui Children's Hospital

    Hefei 1808722, Anhui 1818058 230006
    China

    Site Not Available

  • Beijing Children's Hospital, Capital Medical University

    Beijing, Beijing 100045
    China

    Site Not Available

  • Beijing Children's Hospital, Capital Medical University

    Beijing 1816670, Beijing Municipality 2038349 100045
    China

    Site Not Available

  • Children's Hospital of Chongqing Medical University

    Chongqing, Chongqing 400014
    China

    Site Not Available

  • Children's Hospital of Chongqing Medical University

    Chongqing 1814906, Chongqing Municipality 1814905 400014
    China

    Site Not Available

  • Fujian Medical University Union Hospital

    Fuzhou, Fujian 350001
    China

    Site Not Available

  • Xiamen Maternal and Child Health Hospital

    Xiamen, Fujian 361003
    China

    Site Not Available

  • Fujian Medical University Union Hospital

    Fuzhou 1810821, Fujian 1811017 350001
    China

    Site Not Available

  • Xiamen Maternal and Child Health Hospital

    Xiamen 1790645, Fujian 1811017 361003
    China

    Site Not Available

  • The First Affiliated Hospital, Sun Yat-sen University

    Guangzhou, Guangdong 510080
    China

    Site Not Available

  • Shenzhen Children's Hospital

    Shenzhen, Guangdong 518026
    China

    Site Not Available

  • The First Affiliated Hospital, Sun Yat-sen University

    Guangzhou 1809858, Guangdong 1809935 510080
    China

    Site Not Available

  • Shenzhen Children's Hospital

    Shenzhen 1795565, Guangdong 1809935 518026
    China

    Site Not Available

  • Maternity and Child Health Care of Guangxi Zhuang Autonomous Region

    Nanning, Guangxi 530002
    China

    Site Not Available

  • Maternity and Child Health Care of Guangxi Zhuang Autonomous Region

    Nanning 1799869, Guangxi Zhuang 530002
    China

    Site Not Available

  • The Second Hospital of Hebei Medical University

    Shijiazhuang, Hebei 050000
    China

    Site Not Available

  • The Second Hospital of Hebei Medical University

    Shijiazhuang 1795270, Hebei 1808773 050000
    China

    Site Not Available

  • The Third Affiliated Hospital of Zhengzhou University

    Zhengzhou, Henan 450052
    China

    Site Not Available

  • The Third Affiliated Hospital of Zhengzhou University

    Zhengzhou 1784658, Henan 1808520 450052
    China

    Site Not Available

  • Hunan Children's Hospital

    Changsha 1815577, Hu'nan 410007
    China

    Site Not Available

  • Xiangya Hospital, Central South University

    Changsha 1815577, Hu'nan 410008
    China

    Site Not Available

  • Wuhan Children's Hospital, Tongji Medical College Huazhong University of Science & Technology

    Wuhan, Hubei 430010
    China

    Site Not Available

  • Wuhan Children's Hospital, Tongji Medical College Huazhong University of Science & Technology

    Wuhan 1791247, Hubei 1806949 430010
    China

    Site Not Available

  • Hunan Children's Hospital

    Changsha, Hunan 410007
    China

    Active - Recruiting

  • Xiangya Hospital, Central South University

    Changsha, Hunan 410008
    China

    Site Not Available

  • The Affiliated Hospital of Inner Mongolia Medical University

    Hohhot, Inner Mongolia 010050
    China

    Site Not Available

  • The Affiliated Hospital of Inner Mongolia Medical University

    Hohhot 2036892, Inner Mongolia 2035607 010050
    China

    Site Not Available

  • Xuzhou Central Hospital

    Xuzhou, Jiangsu 221009
    China

    Site Not Available

  • Xuzhou Central Hospital

    Xuzhou 10630003, Jiangsu 1806260 221009
    China

    Site Not Available

  • Jiangxi Provincial Children's Hospital

    Nanchang, Jiangxi 330006
    China

    Site Not Available

  • Jiangxi Provincial Children's Hospital

    Nanchang 1800163, Jiangxi 1806222 330006
    China

    Site Not Available

  • The First Hospital of Jilin University

    Changchun, Jilin 130021
    China

    Site Not Available

  • The First Hospital of Jilin University

    Changchun 2038180, Jilin 2036500 130021
    China

    Site Not Available

  • Children's Hospital Affiliated to Shandong University

    Jinan, Jinan Shandong 250022
    China

    Site Not Available

  • Children's Hospital Affiliated to Shandong University

    Jinan 1805753, Jinan Shandong 250022
    China

    Site Not Available

  • Dalian Women and Children's Medical Group

    Dalian, Liaoning 116037
    China

    Site Not Available

  • Shengjing Hospital of China Medical University

    Shenyang, Liaoning 110022
    China

    Site Not Available

  • Dalian Women and Children's Medical Group

    Dalian 1814087, Liaoning 2036115 116037
    China

    Site Not Available

  • Shengjing Hospital of China Medical University

    Shenyang 2034937, Liaoning 2036115 110022
    China

    Site Not Available

  • People's Hospital of Ningxia Hui Autonomous Region

    YInchuan, Ningxia 750002
    China

    Site Not Available

  • People's Hospital of Ningxia Hui Autonomous Region

    Yinchuan 1786657, Ningxia Hui 750002
    China

    Site Not Available

  • Children's Hospital of Fudan University

    Shanghai, Shanghai 201102
    China

    Site Not Available

  • Children's Hospital of Fudan University

    Shanghai 1796236, Shanghai Municipality 1796231 201102
    China

    Site Not Available

  • The Second Affiliated Hospital of Xi'An Jiaotong University

    Xi'an, Shanxi 710004
    China

    Site Not Available

  • The Second Affiliated Hospital of Xi'An Jiaotong University

    Xi’an 11474004, Shanxi 1795912 710004
    China

    Site Not Available

  • West China Second University Hospital, Sichuan University

    Chengdu, Sichuan 610041
    China

    Site Not Available

  • West China Second University Hospital, Sichuan University

    Chengdu 1815286, Sichuan 1794299 610041
    China

    Site Not Available

  • The Children's Hospital, Zhejiang University School of Medicine

    Hangzhou, Zhejiang 310052
    China

    Site Not Available

  • The Children's Hospital, Zhejiang University School of Medicine

    Hangzhou 1808926, Zhejiang 1784764 310052
    China

    Site Not Available

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