Adult Spinal Muscular Atrophy (SMA) China Registry

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

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).

Study Overview

• Status: Recruiting
• Conditions: Muscular Atrophy, Spinal

• Study Type: Observational
• Enrollment (Anticipated): 200

Contacts and Locations

• Study Contact: Name: US Biogen Clinical Trial Center; Phone Number: 866-633-4636; Email: clinicaltrials@biogen.com
• Study Contact Backup: Name: Global Biogen Clinical Trial Center; Email: clinicaltrials@biogen.com

Study Locations

• China
  • Beijing
    • Beijing, Beijing, China, 100730
      • Recruiting
      • Peking Union Medical College Hospital
  • Fujian
    • Fuzhou, Fujian, China, 350005
      • Recruiting
      • The First Affiliated Hospital Of Fujian Medical University
  • Guangdong
    • Shenzhen, Guangdong, China, 518000
      • Recruiting
      • Peking University Shenzhen Hospital
  • Hebei
    • Shijiazhuang, Hebei, China, 050000
      • Recruiting
      • The Second Hospital of Hebei Medical University
  • Jiangxi
    • Nanchang, Jiangxi, China, 330006
      • Recruiting
      • The First Affiliated Hospital of Nanchang University
  • Shandong
    • Jinan, Shandong, China, 250012
      • Recruiting
      • Qilu Hospital of Shandong University
  • Sichuan
    • Chengdu, Sichuan, China, 610041
      • Recruiting
      • West China Hospital, Sichuan University
  • Yunnan
    • Kunming, Yunnan, China, 650032
      • Recruiting
      • The First People's Hospital of Yunnan Province

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Probability Sample

Study Population

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.

Description

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

Study Plan

How is the study designed?

Design Details

• Observational Models: Other
• Time Perspectives: Prospective

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort
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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	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

Other Outcome Measures

Outcome Measure	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	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

Collaborators and Investigators

• Sponsor: Biogen
• Investigators: Study Director: Medical Director, Biogen

Study record dates

Study Major Dates

• Study Start (Actual): January 6, 2023
• Primary Completion (Anticipated): June 1, 2028
• Study Completion (Anticipated): June 1, 2028

Study Registration Dates

• First Submitted: October 20, 2022
• First Submitted That Met QC Criteria: November 8, 2022
• First Posted (Actual): November 16, 2022

Study Record Updates

• Last Update Posted (Actual): May 17, 2023
• Last Update Submitted That Met QC Criteria: May 15, 2023
• Last Verified: May 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Central Nervous System Diseases; Nervous System Diseases; Neurologic Manifestations; Neuromuscular Diseases; Neurodegenerative Diseases; Neuromuscular Manifestations; Pathological Conditions, Anatomical; Spinal Cord Diseases; Motor Neuron Disease; Muscular Atrophy; Atrophy; Muscular Atrophy, Spinal
• Other Study ID Numbers: CN-SMG-11772

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No