Muscular Atrophy, SpinalNCT05618379
Adult Spinal Muscular Atrophy (SMA) China Registry
An observational study of Muscular Atrophy, Spinal, run by Biogen at 12 sites in China.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Muscular Atrophy, Spinal
- How many people
- 200 participants
- Where
- 12 sitesChina
- Study dates
- 6 Jan 2023to 1 Jun 2028
- 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 18 years and older
- All sexes
- Adult, Older adult
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.
Who may be able to join
- -Genetically confirmed 5q-SMA.
Who cannot join
- -Other types of SMA (non 5q-SMA).
- NOTE: Other protocol defined Inclusion/Exclusion criteria may apply
Trial locations and contacts
12 sites in 1 country.
12 sites in 1 country
China12 sites
Peking Union Medical College Hospital
Beijing, Beijing Municipality, 100730
Xiangya Hospital of Central South University
Changsha, Hunan, 410008
West China Hospital, Sichuan University
Chengdu, Sichuan, 610041
The First Affiliated Hospital of Fujian Medical University
Fuzhou, Fujian, 350005
The First Affiliated Hospital, Sun Yat-sen University
Guangzhou, Guangdong, 510080
Qilu Hospital of Shandong University
Jinan, Shandong, 250012
The First People's Hospital of Yunnan Province
Kunming, Yun'nan, 650032
The First Affiliated Hospital of Nanchang University
Nanchang, Jiangxi, 330006
Huashan Hospital, Fudan University
Shanghai, Shanghai Municipality, 200040
Peking University Shenzhen Hospital
Shenzhen, Guangdong, 518000
The Second Hospital of Hebei Medical University
Shijiazhuang, Hebei, 050000
The First Affiliated Hospital of Soochow University
Suzhou, Jiangsu, 215006
About this study
Official title: 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).
- 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.
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.
Measured: 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.
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 posted16 Nov 2022
- Study start6 Jan 2023
- Primary completion1 Jun 2028 (estimated)
- Study completion1 Jun 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- 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
- Other
- Time perspective
- Prospective
- Enrolment
- 200 participants (estimated)
- 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
- NCT05618379
- Sponsor's protocol ID
- CN-SMG-11772
- Status last verified
- Aug 2026
- First submitted
- 20 Oct 2022
- Last updated
- 4 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/
Publications
Yao X, Dai Y, Zhu W, Zhao Y, Shang H, Meng Q, Liu Y, Yu L, Hong D, Chen J, Shen L, Chen W, Chen K, Zhang L, Mai M, Cui L. Administration practice and adherence of nusinersen in adults with 5q-spinal muscular atrophy in China: an ambispective multicenter study. BMC Neurol. 2026 Feb 28;26(1):176. doi: 10.1186/s12883-026-04730-x.
PubMed 41761140Dai 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. 2026 Feb;43(2):848-865. doi: 10.1007/s12325-025-03475-2. Epub 2025 Dec 29.
PubMed 41461996
Study at a glance
From the registry record, last updated by the sponsor on 4 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Adult Patients With Spinal Muscular Atrophy in China: A Nationwide Registry
- ClinicalTrials.gov ID
- NCT05618379
- Acronym / sponsor ID
- CN-SMG-11772
- Conditions
- Muscular Atrophy, Spinal
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 200 participants (estimated)
- Lead sponsor
- BiogenIndustry (pharma, biotech, devices)
- Study start
- 6 Jan 2023 (actual)
- Primary completion
- 1 Jun 2028 (estimated)
- Study completion
- 1 Jun 2028 (estimated)
- First posted
- 16 Nov 2022 (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) among adult Chinese participants with SMA linked to chromosome 5q (5q-SMA).
Study design
- Observational model
- Other
- 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
- 18 years and older
- Sex
- All
- Age groups
- Adult, Older adult
- 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.
Key inclusion criteria (1 listed)
- -Genetically confirmed 5q-SMA.
Key exclusion criteria (2 listed)
- -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
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.
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.
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 4 Aug 2026.
- Derived from this study: PubMed 41761140Yao X, Dai Y, Zhu W, Zhao Y, Shang H, Meng Q, Liu Y, Yu L, Hong D, Chen J, Shen L, Chen W, Chen K, Zhang L, Mai M, Cui L. Administration practice and adherence of nusinersen in adults with 5q-spinal muscular atrophy in China: an ambispective multicenter study. BMC Neurol. 2026 Feb 28;26(1):176. doi: 10.1186/s12883-026-04730-x.
- Derived from this study: PubMed 41461996Dai 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. 2026 Feb;43(2):848-865. doi: 10.1007/s12325-025-03475-2. Epub 2025 Dec 29.
- 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
12 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 12 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 1 inclusion and 2 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 4 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 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?
Number of Participants Characterized by Natural History of 5q-SMA Observed Among Adult Chinese Participants (time frame: Up to 60 Months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05618379, which also lists 2 linked publications.
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 4 Aug 2026.