NCT05618379

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).

Trial Health

75
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
200

participants targeted

Target at P75+ for all trials

Timeline
25mo left

Started Jan 2023

Longer than P75 for all trials

Geographic Reach
1 country

12 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress62%
Jan 2023Jun 2028

First Submitted

Initial submission to the registry

October 20, 2022

Completed
27 days until next milestone

First Posted

Study publicly available on registry

November 16, 2022

Completed
2 months until next milestone

Study Start

First participant enrolled

January 6, 2023

Completed
5.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 1, 2028

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

June 1, 2028

Last Updated

July 11, 2025

Status Verified

July 1, 2025

Enrollment Period

5.4 years

First QC Date

October 20, 2022

Last Update Submit

July 10, 2025

Conditions

Muscular Atrophy, Spinal

Outcome Measures

Primary Outcomes (2)

  • 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 Outcomes (5)

  • Time to Mortality

    Up to 60 Months

  • Number of Participants With Clinical Characteristics

    Up to 60 Months

  • Number of Participants with Hospitalization

    Up to 60 Months

  • +2 more other outcomes

Study Arms (2)

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.

Eligibility Criteria

Age18 Years+
Sexall
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodProbability 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.

You may qualify if:

  • Genetically confirmed 5q-SMA.

You may not qualify if:

  • Other types of SMA (non 5q-SMA).

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (12)

Peking Union Medical College Hospital

Beijing, Beijing Municipality, 100730, China

Location

The First Affiliated Hospital of Fujian Medical University

Fuzhou, Fujian, 350005, China

Location

The First Affiliated Hospital, Sun Yat-sen University

Guangzhou, Guangdong, 510080, China

Location

Peking University Shenzhen Hospital

Shenzhen, Guangdong, 518000, China

Location

The Second Hospital of Hebei Medical University

Shijiazhuang, Hebei, 050000, China

Location

Xiangya Hospital of Central South University

Changsha, Hunan, 410008, China

Location

The First Affiliated Hospital of Soochow University

Suzhou, Jiangsu, 215006, China

Location

The First Affiliated Hospital of Nanchang University

Nanchang, Jiangxi, 330006, China

Location

Qilu Hospital of Shandong University

Jinan, Shandong, 250012, China

Location

Huashan Hospital, Fudan University

Shanghai, Shanghai Municipality, 200040, China

Location

West China Hospital, Sichuan University

Chengdu, Sichuan, 610041, China

Location

The First People's Hospital of Yunnan Province

Kunming, Yun'nan, 650032, China

Location

Related Publications (1)

  • Dai Y, Yao X, Zhu W, Zhao Y, Shang H, Chen J, Meng Q, Liu Y, Chen W, Yu L, Shen L, Hong D, Chen K, Zhang L, Mai M, Cui L. Effectiveness and Safety of Nusinersen Among Adults with 5q-Spinal Muscular Atrophy: A Multicenter Disease Registry in China. Adv Ther. 2025 Dec 29. doi: 10.1007/s12325-025-03475-2. Online ahead of print.

    PMID: 41461996DERIVED

MeSH Terms

Conditions

Muscular Atrophy, Spinal

Condition Hierarchy (Ancestors)

Spinal Cord DiseasesCentral Nervous System DiseasesNervous System DiseasesMotor Neuron DiseaseNeurodegenerative DiseasesNeuromuscular Diseases

Study Officials

  • Medical Director

    Biogen

    STUDY DIRECTOR

Study Design

Study Type
observational
Observational Model
OTHER
Time Perspective
PROSPECTIVE
Target Duration
60 Months
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 20, 2022

First Posted

November 16, 2022

Study Start

January 6, 2023

Primary Completion (Estimated)

June 1, 2028

Study Completion (Estimated)

June 1, 2028

Last Updated

July 11, 2025

Record last verified: 2025-07

Data Sharing

IPD Sharing
Will share

In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/

More information

Locations

CN(12)