Amyotrophic Lateral SclerosisPhase 4NCT07223723
A Study to Learn More About the Long-Term Safety of Tofersen (Qalsody) in Chinese Participants With SOD-1 Amyotrophic Lateral Sclerosis (ALS)
A Phase 4 study testing Tofersen in people with Amyotrophic Lateral Sclerosis, run by Biogen at 1 site in China.
Trial summary
In this study, researchers will learn more about the safety of tofersen, also known as Qalsody®. This is a drug available for doctors to prescribe for people with a certain type of amyotrophic lateral sclerosis, also known as ALS. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- Tofersen
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteChina
- Study dates
- 11 Dec 2025to 26 May 2027
- Phase
- Phase 4
- 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
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age ≥ 18 years at the time of informed consent.
- Must have diagnosis of SOD1-ALS.
- If taking riluzole, participant must be on a stable dose for ≥ 30 days prior to Day 1 and expected to remain at that dose until the final study visit.
- If taking edaravone, participant must have initiated edaravone ≥ 60 days (2 treatment cycles) prior to Day 1 and expected to remain at that dose until the final study visit, unless the Investigator determines that edaravone should be discontinued for medical reasons, in which case it may not be restarted during the study.
- All women of childbearing potential must practice effective contraception during the study.
Who cannot join
- Hypersensitivity to the active substance or any of the excipients of tofersen injection.
- Current or past administration of tofersen injection, in either a commercial or a clinical study setting.
- Prior or current treatment with small interfering ribonucleic acid (RNA), stem cell therapy, or gene therapy.
- Treatment with another investigational drug, biological agent, or device within 1 month or 5 half-lives of study agent, whichever is longer, prior the Baseline Visit.
- Participants who are pregnant or currently breastfeeding, and those intending to become pregnant during the study.
- Note: Other protocol-defined Inclusion/Exclusion criteria may apply.
Trial locations and contacts
1 site in 1 country.
China1 site
Peking University Third Hospital
Beijing, Beijing Municipality, 100083
About this study
Official title: A Multicenter, Open-Label, Postmarketing Surveillance Study of Tofersen (BIIB067) in Adults With Amyotrophic Lateral Sclerosis (ALS) Associated With a Mutation in the Superoxide Dismutase 1 (SOD1) Gene in China
In this study, researchers will learn more about the safety of tofersen, also known as Qalsody®. This is a drug available for doctors to prescribe for people with a certain type of amyotrophic lateral sclerosis, also known as ALS. This type is in people who have a mutation in the superoxide dismutase 1 gene, also known as SOD-1.
This is known as a "postmarketing" study. In this kind of the study, the goal is to learn more about how a drug works after it has been approved for use in the general public. Tofersen was approved in China in September 2024. The main goal of this study is to collect long-term safety information in Chinese participants with SOD-1 ALS.
Read more
The main question researchers want to answer in this study is:
- How many participants have adverse events (AEs) and serious adverse events (SAEs)?
An AE is a health problem that may or may not be caused by a drug during the study. An AE is considered serious when it results in death, is life-threatening, causes lasting problems, or requires hospital care.
Researchers will also learn more about :
- How the body processes tofersen.
- How much tofersen is found in the cerebrospinal fluid (CSF), or the fluid that surrounds the brain and the spinal cord.
This study will be done as follows:
- Participants will be screened to check if they can join the study. The screening period will be up to 4 weeks.
- After joining the study, participants will receive the first 3 doses of 100 milligrams (mg) of tofersen about 14 days apart. This will be given through an intrathecal (IT) injection. This means it will be given into the fluid surrounding the spine.
- After that, participants will receive 10 more doses every 28 days through IT injections. Participants will have up to 13 total doses of tofersen in this study.
- Participants will have up to 15 visits to their study research center. Each participant will be in the study for up to 52 weeks (1 year).
Read the detailed description
The primary objective of this study is to evaluate the safety of tofersen in participants with SOD1-ALS in post marketing setting in China.
The secondary objective of this study is to evaluate the pharmacokinetics (PK) of tofersen in participants with SOD1-ALS in the postmarketing setting in China.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- Tofersen
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TofersenDrug
Also called BIIB067, QALSODY
Administered by IT injection.
| Study group | Role | What they receive |
|---|---|---|
| Tofersen 100 milligrams (mg) | Experimental | Participants will receive tofersen 100 mg by intrathecal (IT) injection as 3 loading doses once every 2 weeks (Days 1, 15, and 29), followed by 10 maintenance doses once every 4 weeks (approximately every 28 days), for a maximum total of 13 doses per participant over a 44- week treatment period. Receives: Drug: Tofersen |
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)
Measured: From Screening up to end of study follow-up (up to 52 weeks)
Secondary outcomes (4)
Maximum Observed Plasma Concentration (Cmax) of Tofersen
Measured: Predose and at multiple timepoints postdose on Day 1
Time to Reach Maximum Plasma Concentration (Tmax) of Tofersen
Measured: Predose and at multiple timepoints postdose on Day 1
Area Under the Concentration-Time Curve (AUC) of Tofersen in Plasma
Measured: Predose and at multiple timepoints postdose on Day 1
Tofersen Cerebrospinal Fluid (CSF) Concentration
Measured: Up to 44 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Nov 2025
- Study start11 Dec 2025
- Primary completion26 May 2027 (estimated)
- Study completion26 May 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral SclerosisStudies 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
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Biogen
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study chair, Biogen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07223723
- Sponsor's protocol ID
- 233AS402
- Status last verified
- Aug 2026
- First submitted
- 31 Oct 2025
- Last updated
- 13 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/
Study at a glance
From the registry record, last updated by the sponsor on 13 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Open-Label, Postmarketing Surveillance Study of Tofersen (BIIB067) in Adults With Amyotrophic Lateral Sclerosis (ALS) Associated With a Mutation in the Superoxide Dismutase 1 (SOD1) Gene in China
- ClinicalTrials.gov ID
- NCT07223723
- Acronym / sponsor ID
- 233AS402
- Conditions
- Amyotrophic Lateral Sclerosis
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- BiogenIndustry (pharma, biotech, devices)
- Study start
- 11 Dec 2025 (actual)
- Primary completion
- 26 May 2027 (estimated)
- Study completion
- 26 May 2027 (estimated)
- First posted
- 3 Nov 2025 (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 safety of tofersen, also known as Qalsody®. This is a drug available for doctors to prescribe for people with a certain type of amyotrophic lateral sclerosis, also known as ALS. This type is in people who have a mutation in the superoxide dismutase 1 gene, also known as SOD-1.
This is known as a "postmarketing" study. In this kind of the study, the goal is to learn more about how a drug works after it has been approved for use in the general public. Tofersen was approved in China in September 2024. The main goal of this study is to collect long-term safety information in Chinese participants with SOD-1 ALS.
The main question researchers want to answer in this study is:
- How many participants have adverse events (AEs) and serious adverse events (SAEs)?
An AE is a health problem that may or may not be caused by a drug during the study. An AE is considered serious when it results in death, is life-threatening, causes lasting problems, or requires hospital care.
Researchers will also learn more about :
- How the body processes tofersen.
- How much tofersen is found in the cerebrospinal fluid (CSF), or the fluid that surrounds the brain and the spinal cord.
This study will be done as follows:
- Participants will be screened to check if they can join the study. The screening period will be up to 4 weeks.
- After joining the study, participants will receive the first 3 doses of 100 milligrams (mg) of tofersen about 14 days apart. This will be given through an intrathecal (IT) injection. This means it will be given into the fluid surrounding the spine.
- After that, participants will receive 10 more doses every 28 days through IT injections. Participants will have up to 13 total doses of tofersen in this study.
- Participants will have up to 15 visits to their study research center. Each participant will be in the study for up to 52 weeks (1 year).
Detailed description
The primary objective of this study is to evaluate the safety of tofersen in participants with SOD1-ALS in post marketing setting in China.
The secondary objective of this study is to evaluate the pharmacokinetics (PK) of tofersen in participants with SOD1-ALS in the postmarketing setting in China.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Tofersen 100 milligrams (mg) | Experimental | Drug: TofersenParticipants will receive tofersen 100 mg by intrathecal (IT) injection as 3 loading doses once every 2 weeks (Days 1, 15, and 29), followed by 10 maintenance doses once every 4 weeks (approximately every 28 days), for a maximum total of 13 doses per participant over a 44- week treatment period. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Age ≥ 18 years at the time of informed consent.
- Must have diagnosis of SOD1-ALS.
- If taking riluzole, participant must be on a stable dose for ≥ 30 days prior to Day 1 and expected to remain at that dose until the final study visit.
- If taking edaravone, participant must have initiated edaravone ≥ 60 days (2 treatment cycles) prior to Day 1 and expected to remain at that dose until the final study visit, unless the Investigator determines that edaravone should be discontinued for medical reasons, in which case it may not be restarted during the study.
- All women of childbearing potential must practice effective contraception during the study.
Key exclusion criteria (6 listed)
- Hypersensitivity to the active substance or any of the excipients of tofersen injection.
- Current or past administration of tofersen injection, in either a commercial or a clinical study setting.
- Prior or current treatment with small interfering ribonucleic acid (RNA), stem cell therapy, or gene therapy.
- Treatment with another investigational drug, biological agent, or device within 1 month or 5 half-lives of study agent, whichever is longer, prior the Baseline Visit.
- Participants who are pregnant or currently breastfeeding, and those intending to become pregnant during the study.
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 |
|---|---|---|
| TofersenAlso: BIIB067, QALSODYAdministered by IT injection. | Drug | Tofersen 100 milligrams (mg) |
Primary outcome
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: From Screening up to end of study follow-up (up to 52 weeks)
4 secondary outcomes
Maximum Observed Plasma Concentration (Cmax) of Tofersen
Time frame: Predose and at multiple timepoints postdose on Day 1
Time to Reach Maximum Plasma Concentration (Tmax) of Tofersen
Time frame: Predose and at multiple timepoints postdose on Day 1
Area Under the Concentration-Time Curve (AUC) of Tofersen in Plasma
Time frame: Predose and at multiple timepoints postdose on Day 1
Tofersen Cerebrospinal Fluid (CSF) Concentration
Time frame: Up to 44 weeks
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 13 Aug 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 chair
Biogen
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Tofersen in people with Amyotrophic Lateral Sclerosis, run by Biogen at 1 site 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 5 inclusion and 6 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 13 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 1 site 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.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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: From Screening up to end of study follow-up (up to 52 weeks)). 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 13 Aug 2026.