Adult Spinal Muscular Atrophy (SMA) China Registry

Muscular Atrophy, Spinal Clinical Trial

Official title:

Adult Patients With Spinal Muscular Atrophy in China: A Nationwide Registry

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05618379
• Other study ID #: CN-SMG-11772
• Status: Recruiting
• Start date: January 6, 2023
• Est. completion date: June 1, 2028

Study information

• Verified date: May 2023
• Source: Biogen
• Contact: US Biogen Clinical Trial Center
• Phone: 866-633-4636
• Email: clinicaltrials[@]biogen.com
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial 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).

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 200
• Est. completion date: June 1, 2028
• Est. primary completion date: June 1, 2028
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Atrophy
• Muscular Atrophy
• Muscular Atrophy, Spinal

Locations

Country	Name	City	State
China	Peking Union Medical College Hospital	Beijing	Beijing
China	West China Hospital, Sichuan University	Chengdu	Sichuan
China	The First Affiliated Hospital of Fujian Medical University	Fuzhou	Fujian
China	Qilu Hospital of Shandong University	Jinan	Shandong
China	The First People's Hospital of Yunnan Province	Kunming	Yunnan
China	The First Affiliated Hospital of Nanchang University	Nanchang	Jiangxi
China	Peking University Shenzhen Hospital	Shenzhen	Guangdong
China	The Second Hospital of Hebei Medical University	Shijiazhuang	Hebei

Sponsors (1)

Lead Sponsor	Collaborator
Biogen

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Time to Mortality	Time interval between baseline and death.	Up to 60 Months
Other	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	Up to 60 Months
Other	Number of Participants with Hospitalization		Up to 60 Months
Other	Number of Participants With Comorbidities		Up to 60 Months
Other	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
Primary	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
Primary	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