Adult Spinal Muscular Atrophy (SMA) China Registry
Study Details
Study Description
Brief Summary
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).
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
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Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| 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. |
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| Untreated Participants Participants with SMA who received no treatment will be followed prospectively for up to 60 months. |
Outcome Measures
Primary Outcome Measures
- Number of Participants Characterized by Natural History of 5q-SMA Observed Among Adult Chinese Participants [Up to 60 Months]
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.
- Number of Participants Characterized by Utilization of Disease Modifying Therapies (DMTs) of 5q-SMA Observed Among Adult Chinese Participants [Up to 60 Months]
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.
Other Outcome Measures
- Time to Mortality [Up to 60 Months]
Time interval between baseline and death.
- Number of Participants With Clinical Characteristics [Up to 60 Months]
Clinical characteristics will include categories of motor function, motor measures, pulmonary function, clinical observations, nutrition, electrophysiology, biomarkers and categories of patient reported measures
- 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) [Up to 60 Months]
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.
Eligibility Criteria
Criteria
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
Contacts and Locations
Locations
No locations specified.Sponsors and Collaborators
- Biogen
Investigators
- Study Director: Medical Director, Biogen
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- CN-SMG-11772