Adult Spinal Muscular Atrophy (SMA) China Registry

NCT ID: NCT05618379

Last Updated: 2025-07-11

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Total Enrollment

200 participants

Study Classification

OBSERVATIONAL

Study Start Date

2023-01-06

Study Completion Date

2028-06-01

Brief Summary

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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).

Detailed Description

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Conditions

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Muscular Atrophy, Spinal

Study Design

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Observational Model Type

OTHER

Study Time Perspective

PROSPECTIVE

Study Groups

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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.

No interventions assigned to this group

Untreated Participants

Participants with SMA who received no treatment will be followed prospectively for up to 60 months.

No interventions assigned to this group

Eligibility Criteria

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Inclusion Criteria

-Genetically confirmed 5q-SMA.

Exclusion Criteria

-Other types of SMA (non 5q-SMA).
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Biogen

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Medical Director

Role: STUDY_DIRECTOR

Biogen

Locations

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Peking Union Medical College Hospital

Beijing, Beijing Municipality, China

Site Status

The First Affiliated Hospital of Fujian Medical University

Fuzhou, Fujian, China

Site Status

The First Affiliated Hospital, Sun Yat-sen University

Guangzhou, Guangdong, China

Site Status

Peking University Shenzhen Hospital

Shenzhen, Guangdong, China

Site Status

The Second Hospital of Hebei Medical University

Shijiazhuang, Hebei, China

Site Status

Xiangya Hospital of Central South University

Changsha, Hunan, China

Site Status

The First Affiliated Hospital of Soochow University

Suzhou, Jiangsu, China

Site Status

The First Affiliated Hospital of Nanchang University

Nanchang, Jiangxi, China

Site Status

Qilu Hospital of Shandong University

Jinan, Shandong, China

Site Status

Huashan Hospital, Fudan University

Shanghai, Shanghai Municipality, China

Site Status

West China Hospital, Sichuan University

Chengdu, Sichuan, China

Site Status

The First People's Hospital of Yunnan Province

Kunming, Yun'nan, China

Site Status

Countries

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China

Other Identifiers

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CN-SMG-11772

Identifier Type: -

Identifier Source: org_study_id

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