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The primary objective of the study is to describe the natural history and utilization of disease modifying therapy (DMT) among adult Chinese participants with SMA linked to chromosome 5q (5q-SMA).
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| 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. |
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| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants Characterized by Natural History of 5q-SMA Observed Among Adult Chinese Participants | The natural history characteristics will include the categories of the evolution of the patient's condition over the time of data collection from the registry, to describe the natural history of the disease in a real-life situation. | Up to 60 Months |
| Number of Participants Characterized by Utilization of Disease Modifying Therapies (DMTs) of 5q-SMA Observed Among Adult Chinese Participants | The DMT utilization characteristics will include the categories of the evolution of the DMT treated patient's condition over the time of data collection from the registry, to describe the utilization of DMTs of adult SMA patients in a real-life situation. | Up to 60 Months |
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| Measure | Description | Time Frame |
|---|---|---|
| Time to Mortality | Time interval between baseline and death. | Up to 60 Months |
| Number of Participants With Clinical Characteristics | Clinical characteristics will include categories of motor function, motor measures, pulmonary function, clinical observations, nutrition, electrophysiology, biomarkers and categories of patient reported measures |
Key Inclusion Criteria:
-Genetically confirmed 5q-SMA.
Key Exclusion Criteria:
-Other types of SMA (non 5q-SMA).
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply
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Adult participants with 5q-SMA will be enrolled to obtain information on both the natural history of the disease and effectiveness and safety of DMTs.
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Biogen | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Peking Union Medical College Hospital | Beijing | Beijing Municipality | 100730 | China | ||
| The First Affiliated Hospital of Fujian Medical University |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 41761140 | Derived | Yao X, Dai Y, Zhu W, Zhao Y, Shang H, Meng Q, Liu Y, Yu L, Hong D, Chen J, Shen L, Chen W, Chen K, Zhang L, Mai M, Cui L. Administration practice and adherence of nusinersen in adults with 5q-spinal muscular atrophy in China: an ambispective multicenter study. BMC Neurol. 2026 Feb 28;26(1):176. doi: 10.1186/s12883-026-04730-x. | |
| 41461996 |
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In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/
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| 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 |
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| Up to 60 Months |
| Number of Participants with Hospitalization | Up to 60 Months |
| Number of Participants With Comorbidities | Up to 60 Months |
| Number of Participants With Adverse Events (AE) and Serious Adverse Events (SAEs) | An AE is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. SAE is any untoward medical occurrence that at any dose results in death, in the view of the investigator, places the participant at immediate risk of death (a life-threatening event), requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, results in a congenital anomaly/birth defect or is a medically important event. | Up to 60 Months |
| Fuzhou |
| Fujian |
| 350005 |
| China |
| The First Affiliated Hospital, Sun Yat-sen University | Guangzhou | Guangdong | 510080 | China |
| Peking University Shenzhen Hospital | Shenzhen | Guangdong | 518000 | China |
| The Second Hospital of Hebei Medical University | Shijiazhuang | Hebei | 050000 | China |
| Xiangya Hospital of Central South University | Changsha | Hunan | 410008 | China |
| The First Affiliated Hospital of Soochow University | Suzhou | Jiangsu | 215006 | China |
| The First Affiliated Hospital of Nanchang University | Nanchang | Jiangxi | 330006 | China |
| Qilu Hospital of Shandong University | Jinan | Shandong | 250012 | China |
| Huashan Hospital, Fudan University | Shanghai | Shanghai Municipality | 200040 | China |
| West China Hospital, Sichuan University | Chengdu | Sichuan | 610041 | China |
| The First People's Hospital of Yunnan Province | Kunming | Yun'nan | 650032 | China |
| Dai Y, Yao X, Zhu W, Zhao Y, Shang H, Chen J, Meng Q, Liu Y, Chen W, Yu L, Shen L, Hong D, Chen K, Zhang L, Mai M, Cui L. Effectiveness and Safety of Nusinersen Among Adults with 5q-Spinal Muscular Atrophy: A Multicenter Disease Registry in China. Adv Ther. 2026 Feb;43(2):848-865. doi: 10.1007/s12325-025-03475-2. Epub 2025 Dec 29. |
| D019636 | Neurodegenerative Diseases |
| D009468 | Neuromuscular Diseases |