Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
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.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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. |
Not provided
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
Not provided
| Measure | Description | Time Frame |
|---|---|---|
| Effectiveness of DMT Treatments | Effectiveness will be assessed using following measures: time to mortality, motor function, motor measures, pulmonary function, scoliosis, hospitalizations and comorbidities, clinical observations, patient reported outcomes, wheelchair use, nutrition and electrophysiology and biomarkers. | Up to 60 months |
Key Inclusion Criteria:
Key Exclusion Criteria:
NOTE: Other protocol defined inclusion/ exclusion criteria may apply.
Not provided
Not provided
Not provided
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.
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Biogen | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Anhui Children's Hospital | Hefei | Anhui | 230006 | China | ||
| Beijing Children's Hospital, Capital Medical University |
In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/
Not provided
Not provided
Not provided
Not provided
| ID | Term |
|---|---|
| D009134 | Muscular Atrophy, Spinal |
| ID | Term |
|---|---|
| D013118 | Spinal Cord Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D016472 | Motor Neuron Disease |
Not provided
Not provided
Not provided
Not provided
Not provided
| Safety of DMT Treatments |
Safety will be assessed by adverse events (AEs) and serious AEs. |
| Up to 60 months |
| 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 |
| D019636 | Neurodegenerative Diseases |
| D009468 | Neuromuscular Diseases |