Amyotrophic Lateral SclerosisNCT07259980
A Study to Learn More About the Long-Term Safety of Tofersen (Qalsody) in Participants With Superoxide Dismutase 1 (SOD-1) Amyotrophic Lateral Sclerosis (ALS)
An observational study of Amyotrophic Lateral Sclerosis, run by Biogen at 1 site in United States.
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 participant 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
- How many people
- 125 participants
- Where
- 1 siteUnited States
- Study dates
- 2 Jan 2026to 30 Dec 2033
- How long will I take part?
- 7 Years
- 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
- No healthy volunteers
- Adult, Older adult
Study population: Participants with SOD1-ALS enrolled in TRICALS network's Precision-ALS programme with data from participating clinical centers across multiple European countries or at ALS/MND NHC with data from participating clinical centers in the United States (tofersen users and tofersen non-users).
Who may be able to join
- Participants with an ALS diagnosis and a confirmed SOD1 mutation from contributing registry networks will be considered for inclusion in the study.
Who cannot join
- Data collected while a person with SOD1-ALS is participating in an interventional clinical trial (with tofersen or any other investigational medicinal product) will be excluded.
- NOTE: Other protocol- defined Inclusion/Exclusion criteria may apply.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Global Biogen Clinical Trial Center contact
United States1 site, 1 recruiting
- Recruiting
Mass General Hospital -MGH
Boston, Massachusetts, 02114
617-643-2935
Alex Sherman
About this study
Official title: An Observational Registry-Based Study to Evaluate the Long-Term Safety of Tofersen in People With SOD1-ALS
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 participant with a certain type of amyotrophic lateral sclerosis, also known as ALS. This type is in participant who have a mutation in the superoxide dismutase 1 gene, also known as SOD-1.
This is known as an "observational" study, which collects health information about study participants without changing their medical care. Participants for this study will be found using 2 different groups of study research centers that help provide clinical care for participant with ALS. These groups are in Europe and the United States and are called:
Read more
- the Precision-ALS programme
- the ALS/Motor Neuron Disease (MND) Natural History Consortium (NHC)
The main goal of this study is to collect safety information in participants with SOD-1 ALS who were in either of the groups.
The main question researchers want to answer in this study is:
- What are the characteristics of the participants in this study?
- How many participants had serious adverse events (SAEs), including ones that affect the brain, spinal cord, or nerves?
An adverse event is a health problem that may or may not be caused by a drug during the study. An adverse event 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 many participants develop other health conditions or become pregnant, including how the pregnancy turned out
- Why and when participants stopped treatment
This study will be done as follows:
- Participants will be screened to check if they can join the study.
- Data from the participants' regular visits to their clinic will be collected based on which study research center they are in.
- Each participant will be in the study until they decide to leave or until death. Currently, the study is planned to last at least 7 years.
Read the detailed description
The primary objectives of this study are to describe demographic and clinical characteristics of participants with superoxide dismutase 1-amyotropic lateral sclerosis (SOD1-ALS); to describe the frequency of SAEs among participants with SOD1-ALS, including serious neurologic events previously reported in clinical trial participants (e.g., myelitis, radiculitis, aseptic meningitis, increased intracranial pressure, and/or papilloedema).
The secondary objectives of this study are to describe the frequency of new comorbid conditions, pregnancy and pregnancy outcome among participants with SOD1-ALS; to describe the frequency of treatment discontinuation among participants with SOD1-ALS treated with tofersen.
NHC registry has started the data collection while the Treatment Research Initiative to Cure ALS (TRICALS) is yet to start.
- 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 as specified in the treatment arm.
| Study group | Role | What they receive |
|---|---|---|
| Tofersen | Data for participants with SOD1-ALS will be collected via ALS disease registries, the TRICALS network's Precision-ALS programme and ALS/MND NHC. Receives: Drug: Tofersen |
What is being measured
The primary outcome is the main question the study is designed to answer.
Baseline Demographic: Age
Measured: At Baseline
Baseline Demographic: Participant Sex
Measured: At Baseline
Baseline Demographic: Race/Ethnicity
Measured: At Baseline
Baseline Demographic: Weight
Measured: At Baseline
Baseline Demographic: Height
Measured: At Baseline
Baseline Demographic: Body Mass Index (BMI)
Measured: At Baseline
Baseline Demographic: Family History of Amyotrophic Lateral Sclerosis (ALS)
Measured: At Baseline
Clinical Characteristics: Age at Diagnosis and Symptom Onset
Measured: At Baseline
Clinical Characteristics: Revised El Escorial Classification
Measured: At Baseline
Clinical Characteristics: Classification of SOD1-ALS Clinical Phenotypes
Measured: At Baseline
Clinical Characteristics: SOD1 Mutation Type
Measured: At Baseline
Clinical Characteristics: Medical History
Measured: At Baseline
Clinical Characteristics: Concomitant Medications
Measured: At Baseline
Clinical Characteristics: Disease History
Measured: At Baseline
Clinical Characteristics: Pregnancy Status
Measured: At Baseline
Number of Participants With Serious Adverse Events
Measured: Up to 7 years
Secondary outcomes (4)
Number of Participants With New Comorbid Conditions
Measured: Up to 7 years
Number of Participants With Pregnancy and Pregnancy Outcomes
Measured: Up to 7 years
Number of Participants With Reported Treatment Discontinuation
Measured: Up to 7 years
Number of Participants With Reported Reasons for Treatment Discontinuation
Measured: Up to 7 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Dec 2025
- Study start2 Jan 2026
- Primary completion30 Dec 2033 (estimated)
- Study completion30 Dec 2033 (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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 125 participants (estimated)
- Target duration
- 7 Years
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
- NCT07259980
- Sponsor's protocol ID
- 233AS401
- Status last verified
- Apr 2026
- First submitted
- 31 Oct 2025
- Last updated
- 9 Apr 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- 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 9 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Observational Registry-Based Study to Evaluate the Long-Term Safety of Tofersen in People With SOD1-ALS
- ClinicalTrials.gov ID
- NCT07259980
- Acronym / sponsor ID
- 233AS401
- Conditions
- Amyotrophic Lateral Sclerosis
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 125 participants (estimated)
- Lead sponsor
- BiogenIndustry (pharma, biotech, devices)
- Study start
- 2 Jan 2026 (actual)
- Primary completion
- 30 Dec 2033 (estimated)
- Study completion
- 30 Dec 2033 (estimated)
- First posted
- 2 Dec 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 participant with a certain type of amyotrophic lateral sclerosis, also known as ALS. This type is in participant who have a mutation in the superoxide dismutase 1 gene, also known as SOD-1.
This is known as an "observational" study, which collects health information about study participants without changing their medical care. Participants for this study will be found using 2 different groups of study research centers that help provide clinical care for participant with ALS. These groups are in Europe and the United States and are called:
- the Precision-ALS programme
- the ALS/Motor Neuron Disease (MND) Natural History Consortium (NHC)
The main goal of this study is to collect safety information in participants with SOD-1 ALS who were in either of the groups.
The main question researchers want to answer in this study is:
- What are the characteristics of the participants in this study?
- How many participants had serious adverse events (SAEs), including ones that affect the brain, spinal cord, or nerves?
An adverse event is a health problem that may or may not be caused by a drug during the study. An adverse event 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 many participants develop other health conditions or become pregnant, including how the pregnancy turned out
- Why and when participants stopped treatment
This study will be done as follows:
- Participants will be screened to check if they can join the study.
- Data from the participants' regular visits to their clinic will be collected based on which study research center they are in.
- Each participant will be in the study until they decide to leave or until death. Currently, the study is planned to last at least 7 years.
Detailed description
The primary objectives of this study are to describe demographic and clinical characteristics of participants with superoxide dismutase 1-amyotropic lateral sclerosis (SOD1-ALS); to describe the frequency of SAEs among participants with SOD1-ALS, including serious neurologic events previously reported in clinical trial participants (e.g., myelitis, radiculitis, aseptic meningitis, increased intracranial pressure, and/or papilloedema).
The secondary objectives of this study are to describe the frequency of new comorbid conditions, pregnancy and pregnancy outcome among participants with SOD1-ALS; to describe the frequency of treatment discontinuation among participants with SOD1-ALS treated with tofersen.
NHC registry has started the data collection while the Treatment Research Initiative to Cure ALS (TRICALS) is yet to start.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 7 Years
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Tofersen | – | Drug: TofersenData for participants with SOD1-ALS will be collected via ALS disease registries, the TRICALS network's Precision-ALS programme and ALS/MND NHC. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Participants with SOD1-ALS enrolled in TRICALS network's Precision-ALS programme with data from participating clinical centers across multiple European countries or at ALS/MND NHC with data from participating clinical centers in the United States (tofersen users and tofersen non-users).
Key inclusion criteria (1 listed)
- Participants with an ALS diagnosis and a confirmed SOD1 mutation from contributing registry networks will be considered for inclusion in the study.
Key exclusion criteria (2 listed)
- Data collected while a person with SOD1-ALS is participating in an interventional clinical trial (with tofersen or any other investigational medicinal product) will be excluded.
- 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 |
|---|---|---|
| TofersenAlso: BIIB067, QalsodyAdministered as specified in the treatment arm. | Drug | Tofersen |
Primary outcome
Baseline Demographic: Age
Time frame: At Baseline
Baseline Demographic: Participant Sex
Time frame: At Baseline
Baseline Demographic: Race/Ethnicity
Time frame: At Baseline
Baseline Demographic: Weight
Time frame: At Baseline
Baseline Demographic: Height
Time frame: At Baseline
Baseline Demographic: Body Mass Index (BMI)
Time frame: At Baseline
Baseline Demographic: Family History of Amyotrophic Lateral Sclerosis (ALS)
Time frame: At Baseline
Clinical Characteristics: Age at Diagnosis and Symptom Onset
Time frame: At Baseline
Clinical Characteristics: Revised El Escorial Classification
Time frame: At Baseline
Clinical Characteristics: Classification of SOD1-ALS Clinical Phenotypes
Time frame: At Baseline
Clinical Characteristics: SOD1 Mutation Type
Time frame: At Baseline
Clinical Characteristics: Medical History
Time frame: At Baseline
Clinical Characteristics: Concomitant Medications
Time frame: At Baseline
Clinical Characteristics: Disease History
Time frame: At Baseline
Clinical Characteristics: Pregnancy Status
Time frame: At Baseline
Number of Participants With Serious Adverse Events
Time frame: Up to 7 years
4 secondary outcomes
Number of Participants With New Comorbid Conditions
Time frame: Up to 7 years
Number of Participants With Pregnancy and Pregnancy Outcomes
Time frame: Up to 7 years
Number of Participants With Reported Treatment Discontinuation
Time frame: Up to 7 years
Number of Participants With Reported Reasons for Treatment Discontinuation
Time frame: Up to 7 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 9 Apr 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.
Global Biogen Clinical Trial Center contact
Medical Director study director
Biogen
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Amyotrophic Lateral Sclerosis, run by Biogen at 1 site in United States.
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: looking for participants now, as of the sponsor's last update on 9 Apr 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 United States. 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?
Contact Study US Biogen Clinical Trial Center (866-633-4636, clinicaltrials@biogen.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Baseline Demographic: Age (time frame: At Baseline). The study lists 16 primary outcomes in all. 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 9 Apr 2026.