Observational, Postmarketing Surveillance Study of Spinraza Injection (Nusinersen Sodium)
NCT04317794
Central Nervous System Diseases, Motor Neuron Disease
Busan, South Korea
View Trial DetailsNCT Number: NCT05618379
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).
This study is active but is not currently recruiting participants.
Notify Me18 year and older
All sexes
Observational
Peking Union Medical College Hospital, Beijing, Beijing Municipality, China
Only the study team can determine whether someone qualifies for participation.
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
Time frame: 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.
Time frame: 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.
Time frame: Up to 60 Months
Time interval between baseline and death.
Time frame: 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
Time frame: Up to 60 Months
Time frame: Up to 60 Months
Time frame: 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.
Biogen
Industry
Adult Patients With Spinal Muscular Atrophy in China: A Nationwide Registry
OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.
View the official ClinicalTrials.gov record (opens in a new tab)This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.
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