Muscular Atrophy, SpinalSTANDARDNCT04317794
A Study of the Safety of Nusinersen (Spinraza®) and Its Effects on Motor Function in Koreans Living With SMA Treated During Their Usual Medical Care
An observational study of Muscular Atrophy, Spinal, run by Biogen at 16 sites in South Korea.
Trial summary
In this study, researchers will learn more about the effects of nusinersen, also known as Spinraza®, in Korean participants with spinal muscular atrophy, also known as SMA. This is a drug available for doctors to prescribe for people with SMA. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Muscular Atrophy, Spinal
- What is being tested?
- Nusinersen Sodium Injection
- Type of treatment
- Injection or infusion
- How many people
- 74 participants
- Where
- 16 sitesSouth Korea
- Study dates
- 2 Jul 2019to 5 Feb 2027
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Participants who are receiving or about to initiate treatment with commercial Spinraza in Korea according to the local marketing authorization (including documentation of SMA diagnosis), agree to the collection and use of data as specified in the protocol, and meet the eligibility criteria.
Who may be able to join
- Currently receiving or about to initiate treatment with commercial Spinraza in the postmarketing setting
- Genetic documentation of 5q-linked SMA
Who cannot join
- Hypersensitivity to the active substance or any of the excipients of Spinraza
- Ongoing participation or participation within 6 months or 5 half-lives of the agent (whichever is longer) of enrollment in other interventional clinical trials for the treatment of SMA
- Inability to comply with study requirements
- NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
Trial locations and contacts
16 sites in 1 country.
16 sites in 1 country
South Korea16 sites
Pusan Natioanl University Hospital
Busan
Samsung Changwon Hospital
Changwon
Chungbuk National University Hospital
Cheongju-si
Kyungpook National University Hospital
Daegu
Yeungnam University Hospital
Daegu
Chungnam National University Hospital
Daejeon
Chonnam National University Hospital
Gwangju
Chonbuk National University Hospital
Jeonju
Severance Hospital
Seoul, 03722
Asan Medical Center
Seoul
Korea university Anam Hospital
Seoul
Korea University Guro Hospital
Seoul
Samsung Medical Center
Seoul
Seoul Natioanl University Hospital
Seoul
Severance Hospital, Yonsei University Health System
Seoul
Pusan National University Yangsan Hospital
Yangsan
About this study
Official title: A Multicenter, Observational, Postmarketing Surveillance Study of Spinraza Injection (Nusinersen Sodium) When Used in Routine Medical Practice in Korea
In this study, researchers will learn more about the effects of nusinersen, also known as Spinraza®, in Korean participants with spinal muscular atrophy, also known as SMA. This is a drug available for doctors to prescribe for people with SMA. This is known as an "observational" study, which collects health information about study participants without changing their medical care.
In Korea, some medicines for rare diseases are allowed to be reviewed for up to 10 years when there are no other treatment options. This study was set up to support that review by collecting more information about nusinersen. In this study, researchers wanted to learn more about its safety and how it may help patients receiving it as part of their routine medical care in Korea.
Read more
The main goal of the study is to learn about the long-term safety of nusinersen in Korean participants with SMA. The main question researchers want to answer are:
- How many participants have adverse events and serious adverse events during the study?
Researchers will also learn more about:
- How does nusinersen affect the participants' muscle function and ability to move?
- How long participants live after joining the study?
- How many participants need help with their breathing during this study?
This study will be done as follows:
- Participants will join the study after signing an informed consent form, also known as an ICF.
- During the study, health information from the participants' regular visits to their doctor will be collected about every 4 months.
- During each visit, doctors will measure the participants' motor function with different tests. They will also check the participants' overall health and ask how they are feeling.
- Each participant will be in the study for up to 2 years.
Read the detailed description
The primary objective is to evaluate the safety of nusinersen sodium injection in the postmarketing setting in Korea. The secondary objective is to evaluate the effectiveness of nusinersen sodium injection in the postmarketing setting in Korea.
- Conditions
- Muscular Atrophy, Spinal
- Being tested
- Nusinersen Sodium Injection
- 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.
Nusinersen Sodium InjectionDrug
Also called BIIB058, ISIS 396443, Spinraza
Administered as specified in the treatment arm.
| Study group | Role | What they receive |
|---|---|---|
| All Participants | Participants who were prescribed with nusinersen sodium injection (12 mg or 50/28 mg regimens) in Korea according to local marketing authorization. Receives: Drug: Nusinersen Sodium Injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
An AE is any untoward medical occurrence in a patient or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An SAE is any untoward medical occurrence that at any dose results in death, places the participant at immediate risk of death, 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.
Measured: Up to End of Treatment (2 Years)
Secondary outcomes (4)
Change from Baseline in Hammersmith Infant Neurological Examination (HINE) Section 2 Scores
Section 2 of the HINE is used to assess motor milestones of the participants. It is composed of 8 motor milestone categories: voluntary grasp, ability to kick in supine position, head control, rolling, sitting, crawling, standing, and walking.
Measured: Baseline, up to End of Treatment (2 years)
Change from Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE)
The HFMSE is a tool used to assess motor function in children with SMA. The original 20 item Hammersmith Functional Motor Scale (HFMS) was expanded to include 13 additional items to improve sensitivity for the higher functioning ambulant population. Participants are asked to complete a specific movement and are then graded on the quality and execution of that movement. Higher scores indicate higher levels of motor ability where 0=unable, 1=performed with some assistance, and 2=unaided. The overall score is the sum of the scores for all activities.
Measured: Baseline, up to End of Treatment (2 years)
Time to Death
Measured: Up to End of Treatment (2 years)
Number of Participants with Ventilatory Support
Measured: Up to End of Treatment (2 years)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Mar 2020
- Study start2 Jul 2019
- Primary completion5 Feb 2027 (estimated)
- Study completion5 Feb 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
- Case only
- Time perspective
- Prospective
- Enrolment
- 74 participants (actual)
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
- NCT04317794
- Sponsor's protocol ID
- 232SM403
- Acronym
- STANDARD
- Status last verified
- Oct 2026
- First submitted
- 5 Mar 2020
- Last updated
- 26 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- 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 26 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Observational, Postmarketing Surveillance Study of Spinraza Injection (Nusinersen Sodium) When Used in Routine Medical Practice in Korea
- ClinicalTrials.gov ID
- NCT04317794
- Acronym / sponsor ID
- STANDARD / 232SM403
- Conditions
- Muscular Atrophy, Spinal
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 74 participants (actual)
- Lead sponsor
- BiogenIndustry (pharma, biotech, devices)
- Study start
- 2 Jul 2019 (actual)
- Primary completion
- 5 Feb 2027 (estimated)
- Study completion
- 5 Feb 2027 (estimated)
- First posted
- 23 Mar 2020 (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.
In this study, researchers will learn more about the effects of nusinersen, also known as Spinraza®, in Korean participants with spinal muscular atrophy, also known as SMA. This is a drug available for doctors to prescribe for people with SMA. This is known as an "observational" study, which collects health information about study participants without changing their medical care.
In Korea, some medicines for rare diseases are allowed to be reviewed for up to 10 years when there are no other treatment options. This study was set up to support that review by collecting more information about nusinersen. In this study, researchers wanted to learn more about its safety and how it may help patients receiving it as part of their routine medical care in Korea.
The main goal of the study is to learn about the long-term safety of nusinersen in Korean participants with SMA. The main question researchers want to answer are:
- How many participants have adverse events and serious adverse events during the study?
Researchers will also learn more about:
- How does nusinersen affect the participants' muscle function and ability to move?
- How long participants live after joining the study?
- How many participants need help with their breathing during this study?
This study will be done as follows:
- Participants will join the study after signing an informed consent form, also known as an ICF.
- During the study, health information from the participants' regular visits to their doctor will be collected about every 4 months.
- During each visit, doctors will measure the participants' motor function with different tests. They will also check the participants' overall health and ask how they are feeling.
- Each participant will be in the study for up to 2 years.
Detailed description
The primary objective is to evaluate the safety of nusinersen sodium injection in the postmarketing setting in Korea. The secondary objective is to evaluate the effectiveness of nusinersen sodium injection in the postmarketing setting in Korea.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| All Participants | – | Drug: Nusinersen Sodium InjectionParticipants who were prescribed with nusinersen sodium injection (12 mg or 50/28 mg regimens) in Korea according to local marketing authorization. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Participants who are receiving or about to initiate treatment with commercial Spinraza in Korea according to the local marketing authorization (including documentation of SMA diagnosis), agree to the collection and use of data as specified in the protocol, and meet the eligibility criteria.
Key inclusion criteria (2 listed)
- Currently receiving or about to initiate treatment with commercial Spinraza in the postmarketing setting
- Genetic documentation of 5q-linked SMA
Key exclusion criteria (4 listed)
- Hypersensitivity to the active substance or any of the excipients of Spinraza
- Ongoing participation or participation within 6 months or 5 half-lives of the agent (whichever is longer) of enrollment in other interventional clinical trials for the treatment of SMA
- Inability to comply with study requirements
- 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.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Nusinersen Sodium InjectionAlso: BIIB058, ISIS 396443, SpinrazaAdministered as specified in the treatment arm. | Drug | All Participants |
Primary outcome
Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: Up to End of Treatment (2 Years)
An AE is any untoward medical occurrence in a patient or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An SAE is any untoward medical occurrence that at any dose results in death, places the participant at immediate risk of death, 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.
4 secondary outcomes
Change from Baseline in Hammersmith Infant Neurological Examination (HINE) Section 2 Scores
Time frame: Baseline, up to End of Treatment (2 years)
Change from Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE)
Time frame: Baseline, up to End of Treatment (2 years)
Time to Death
Time frame: Up to End of Treatment (2 years)
Number of Participants with Ventilatory Support
Time frame: Up to End of Treatment (2 years)
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 26 Oct 2026.
- Individual participant dataSharing planned: Yes. In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com
Related trials for Muscular Atrophy, Spinal
Recruiting
Evaluating Long-term Use of a Pediatric Robotic Exoskeleton (P.REX/Agilik) to Improve Gait in Children With Movement Disorders
Cerebral Palsy, Muscular Dystrophy
Recruiting
SMAdult - Monitoring of Movement on Spinal Muscular Atrophy
Spinal Amyotrophy
Active, not recruitingPhase 1
A Study to Learn About the Safety of BIIB115 Injections and How BIIB115 is Processed in the Bodies of Healthy Adult Male Volunteers and of Pediatric Participants With Spinal Muscular Atrophy Who Previously Took Onasemnogene Abeparvovec
Healthy Volunteer, Muscular Atrophy, Spinal
Recruiting
Effects of Different Stiffness Settings During Robotic Isokinetic Training in Children With SMA
Spinal Muscular Atrophy (SMA)
RecruitingPhase 3
A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety When Given Before Symptoms Appear in Babies With Genetically Diagnosed Spinal Muscular Atrophy (SMA)
Muscular Atrophy, Spinal
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
16 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 16 sites in South Korea.
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 2 inclusion and 4 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 26 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 16 sites in South Korea. 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 with Adverse Events (AEs) and Serious Adverse Events (SAEs) (time frame: Up to End of Treatment (2 Years)). 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 26 Oct 2026.