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NCT Number: NCT05618379

Adult Spinal Muscular Atrophy (SMA) China 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).

Active, Not Recruiting

This study is active but is not currently recruiting participants.

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Key information

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Peking Union Medical College Hospital, Beijing, Beijing Municipality, China

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Who can participate

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

Treatment and study plan

Primary outcomes

  1. Number of Participants Characterized by Natural History of 5q-SMA Observed Among Adult Chinese Participants

    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.

  2. Number of Participants Characterized by Utilization of Disease Modifying Therapies (DMTs) of 5q-SMA Observed Among Adult Chinese Participants

    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.

Other outcomes

  1. Time to Mortality

    Time frame: Up to 60 Months

    Time interval between baseline and death.

  2. Number of Participants With Clinical Characteristics

    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

  3. Number of Participants with Hospitalization

    Time frame: Up to 60 Months

  4. Number of Participants With Comorbidities

    Time frame: Up to 60 Months

  5. Number of Participants With Adverse Events (AE) and Serious Adverse Events (SAEs)

    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.

Sponsors and collaborators

Lead sponsor

Biogen

Industry

Registry information

Official study title

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

Important dates

Study start
2023
Primary completion
2028
Study completion
2028
First posted
Nov 16, 2022
Registry last updated
Jul 11, 2025

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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