Muscular Atrophy, SpinalNCT05042921
Pediatric Spinal Muscular Atrophy (SMA) China Registry
An observational study of Muscular Atrophy, Spinal, run by Biogen at 25 sites in China.
Trial summary
The primary objective of the study is to describe the natural history and utilization of disease modifying therapy (DMT) treatments among pediatric Chinese participants with spinal muscular atrophy linked to chromosome 5q (5q-SMA). Read more about this study
- Who is it for?
- Ages Up to 18 years
- Conditions the study is for
- Muscular Atrophy, Spinal
- How many people
- 600 participants
- Where
- 25 sitesChina
- Study dates
- 18 Nov 2021to 2 Sep 2027
- How long will I take part?
- 60 Months
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Pediatric participants with 5q-SMA will be enrolled to obtain information on both the natural history of the disease and effectiveness and safety of DMTs.
Who may be able to join
- Ability of the participant and/or his/her legally authorized representative (e.g., parent or legal guardian), as appropriate and applicable, to understand the purpose and risks of the study, to provide informed consent, and to authorize the use of confidential health information in accordance with national and local privacy regulations.
- Genetically confirmed 5q-SMA.
- Age < 18 years at enrollment.
Who cannot join
- Unable or unwilling to provide informed consent.
- Other types of SMA (non 5q-SMA).
- NOTE: Other protocol defined inclusion/ exclusion criteria may apply.
Trial locations and contacts
25 sites in 1 country.
25 sites in 1 country
China25 sites
Beijing Children's Hospital, Capital Medical University
Beijing, Beijing Municipality, 100045
The First Hospital of Jilin University
Changchun, Jilin, 130021
Hunan Children's Hospital
Changsha, Hu'nan, 410007
Xiangya Hospital, Central South University
Changsha, Hu'nan, 410008
West China Second University Hospital, Sichuan University
Chengdu, Sichuan, 610041
Children's Hospital of Chongqing Medical University
Chongqing, Chongqing Municipality, 400014
Dalian Women and Children's Medical Group
Dalian, Liaoning, 116037
Fujian Medical University Union Hospital
Fuzhou, Fujian, 350001
The First Affiliated Hospital, Sun Yat-sen University
Guangzhou, Guangdong, 510080
The Children's Hospital, Zhejiang University School of Medicine
Hangzhou, Zhejiang, 310052
Anhui Children's Hospital
Hefei, Anhui, 230006
The Affiliated Hospital of Inner Mongolia Medical University
Hohhot, Inner Mongolia, 010050
Children's Hospital Affiliated to Shandong University
Jinan, Jinan Shandong, 250022
Jiangxi Provincial Children's Hospital
Nanchang, Jiangxi, 330006
Maternity and Child Health Care of Guangxi Zhuang Autonomous Region
Nanning, Guangxi Zhuang, 530002
Children's Hospital of Fudan University
Shanghai, Shanghai Municipality, 201102
Shengjing Hospital of China Medical University
Shenyang, Liaoning, 110022
Shenzhen Children's Hospital
Shenzhen, Guangdong, 518026
The Second Hospital of Hebei Medical University
Shijiazhuang, Hebei, 050000
Wuhan Children's Hospital, Tongji Medical College Huazhong University of Science & Technology
Wuhan, Hubei, 430010
The Second Affiliated Hospital of Xi'An Jiaotong University
Xi’an, Shanxi, 710004
Xiamen Maternal and Child Health Hospital
Xiamen, Fujian, 361003
Xuzhou Central Hospital
Xuzhou, Jiangsu, 221009
People's Hospital of Ningxia Hui Autonomous Region
Yinchuan, Ningxia Hui, 750002
The Third Affiliated Hospital of Zhengzhou University
Zhengzhou, Henan, 450052
About this study
Official title: Pediatric 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) treatments among pediatric Chinese participants with spinal muscular atrophy linked to chromosome 5q (5q-SMA). The study will examine SMA natural history and DMT outcomes in a real-world setting both prospectively and retrospectively.
- Conditions
- Muscular Atrophy, Spinal
- Medical subject headings
- Muscular Atrophy, Spinal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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 being measured
The primary outcome is the main question the study is designed to answer.
Natural History and Utilization of Disease Modifying Therapy (DMT) Treatments Among Pediatric Chinese Participants With Spinal Muscular Atrophy Linked to Chromosome 5q (5q-SMA)
Measured: Up to 60 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Sep 2021
- Study start18 Nov 2021
- Primary completion2 Sep 2027 (estimated)
- Study completion2 Sep 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Muscular Atrophy, SpinalStudies of the same condition, open and under way.
- More trials run by BiogenThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 600 participants (actual)
- Target duration
- 60 Months
Sponsor and investigators
- Lead sponsor
- Biogen
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Biogen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05042921
- Sponsor's protocol ID
- CN-SMG-11774
- Status last verified
- Sep 2026
- First submitted
- 8 Sep 2021
- Last updated
- 11 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- Yes: In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/
Study at a glance
From the registry record, last updated by the sponsor on 11 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pediatric Patients With Spinal Muscular Atrophy in China: A Nationwide Registry
- ClinicalTrials.gov ID
- NCT05042921
- Acronym / sponsor ID
- CN-SMG-11774
- Conditions
- Muscular Atrophy, Spinal
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 600 participants (actual)
- Lead sponsor
- BiogenIndustry (pharma, biotech, devices)
- Study start
- 18 Nov 2021 (actual)
- Primary completion
- 2 Sep 2027 (estimated)
- Study completion
- 2 Sep 2027 (estimated)
- First posted
- 13 Sep 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The primary objective of the study is to describe the natural history and utilization of disease modifying therapy (DMT) treatments among pediatric Chinese participants with spinal muscular atrophy linked to chromosome 5q (5q-SMA). The study will examine SMA natural history and DMT outcomes in a real-world setting both prospectively and retrospectively.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 60 Months
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 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. |
Who may qualify
- Ages
- Up to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Pediatric participants with 5q-SMA will be enrolled to obtain information on both the natural history of the disease and effectiveness and safety of DMTs.
Key inclusion criteria (3 listed)
- Ability of the participant and/or his/her legally authorized representative (e.g., parent or legal guardian), as appropriate and applicable, to understand the purpose and risks of the study, to provide informed consent, and to authorize the use of confidential health information in accordance with national and local privacy regulations.
- Genetically confirmed 5q-SMA.
- Age < 18 years at enrollment.
Key exclusion criteria (3 listed)
- Unable or unwilling to provide informed consent.
- Other types of SMA (non 5q-SMA).
- NOTE: Other protocol defined inclusion/ exclusion criteria may apply.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Natural History and Utilization of Disease Modifying Therapy (DMT) Treatments Among Pediatric Chinese Participants With Spinal Muscular Atrophy Linked to Chromosome 5q (5q-SMA)
Time frame: Up to 60 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Sep 2026.
- Individual participant dataSharing planned: Yes. In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Director study director
Biogen
25 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Muscular Atrophy, Spinal, run by Biogen at 25 sites in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 11 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 25 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Natural History and Utilization of Disease Modifying Therapy (DMT) Treatments Among Pediatric Chinese Participants With Spinal Muscular Atrophy Linked to Chromosome 5q (5q-SMA) (time frame: Up to 60 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Sep 2026.