Amyotrophic Lateral Sclerosis Associated With a SOD1 Gene MutationPhase 3ATLASNCT04856982
Study to Learn If Tofersen (BIIB067) Can Delay ALS Symptoms and About Its Safety in Adults With a SOD1 Gene Mutation Who Have Not Yet Developed ALS Symptoms
A Phase 3 study testing Tofersen in people with Amyotrophic Lateral Sclerosis Associated With a SOD1 Gene Mutation, run by Biogen at 32 sites in 14 countries.
Trial summary
In this study, researchers will learn more about the effects of a drug called tofersen, also known as BIIB067 and QALSODY®, in adults with amyotrophic lateral sclerosis (ALS) who have a superoxide dismutase 1 (SOD1) gene mutation. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Amyotrophic Lateral Sclerosis Associated With a SOD1 Gene Mutation
- What is being tested?
- Tofersen
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 158 participants
- Where
- 32 sitesin 14 countries
- Study dates
- 17 May 2021to 30 Apr 2028
- Phase
- Phase 3
- 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
Key Part A Inclusion Criteria:
- Participants should have a protocol-defined rapidly progressive SOD1 mutation, confirmed by a central reader, or a SOD1 mutation that is approved for inclusion by an external mutation adjudication committee.
- Participants with plasma NfL level less than the protocol-defined threshold.
- Participants who are clinically presymptomatic for ALS (i.e., must not have clinically manifest ALS).
Key Part A Exclusion Criteria:
- History or positive test result at screening for human immunodeficiency virus (HIV). The requirement for testing at Screening may be omitted if it is not permitted by local regulations.
- Current hepatitis C infection [defined as positive Hepatitis C Virus (HCV) antibody and detectable HCV ribonucleic acid (RNA)]. Participants with positive HCV antibody and undetectable HCV RNA are eligible to participate in the study (United States Centers for Disease Control and Prevention).
- Current hepatitis B infection (defined as positive for hepatitis B surface antigen (HBsAg) and/or anti-Hepatitis B Core antibody (HBc)). Participants with immunity to hepatitis B from previous natural infection (defined as negative HBsAg, positive anti-HBc, and positive anti-hepatitis B surface antibody (HBs) or vaccination (defined as negative HBsAg, negative anti-HBc, and positive anti- HBs) are eligible to participate in the study.
- History of systemic hypersensitivity reaction to tofersen, the excipients contained in the formulation, and if appropriate, any diagnostic agents to be administered during the study.
- History of confounding neuromuscular or neurological disorder that is expected to have a progressive (i.e., worsening) course during the study, and/or is expected to be associated with elevations in NF, in the opinion of the Investigator.
- Presence of risk for increased or uncontrolled bleeding and/or risk of bleeding that if not managed optimally could place a participant at an increased risk for intraoperative or postoperative bleeding.
- Significant cognitive impairment, clinical dementia, or unstable psychiatric illness, including psychosis, suicidal ideation, suicide attempt, or untreated major depression ≤ 90 days of screening, which in the opinion of the Investigator would interfere with the study procedures.
- Treatment with riluzole, edaravone, and/or sodium phenylbutyrate/taurursodiol (also known as ursodoxicoltaurine). If the participant has been on riluzole, edaravone, and/or sodium phenylbutyrate/taurursodiol, the medication(s) must be discontinued for at least 5 half-lives prior to Screening.
- Use of off-label treatments for ALS.
- Treatment with another investigational drug (including investigational drugs for ALS through compassionate use programs), biological agent, or device within 1 month or 5 half-lives of study agent, whichever is longer. Specifically, no prior treatment with small interfering RNA, stem cell therapy, or gene therapy is allowed.
- Anticipated need, in the opinion of the Investigator, for administration of any antiplatelet or anticoagulant medication (e.g., clopidogrel) that cannot be safely continued or held for a Lumbar Puncture (LP) procedure, if necessary, according to local or institutional guidelines and/or Investigator determination.
- Current enrollment or a plan to enroll in any interventional clinical study in which an investigational treatment, biological agent, device, or approved therapy for investigational use. Participation in a noninterventional study focused on ALS natural history may be allowed at the discretion of the Investigator.
NOTE: Other protocol defined Inclusion/Exclusion criteria will apply.
Trial locations and contacts
32 sites in 14 countries.
32 sites in 14 countries
United States12 sites
The Emory Clinic
Atlanta, Georgia, 30322-4200
Austin Neuromuscular Center
Austin, Texas, 78756
Johns Hopkins Hospital
Baltimore, Maryland, 21287
Massachusetts General Hospital, MA
Charlestown, Massachusetts, 02129
Northwestern Medicine
Chicago, Illinois, 60611
Holy Cross Hospital
Fort Lauderdale, Florida, 33308
University of California San Diego Medical Center
La Jolla, California, 92093-0949
University of Miami School of Medicine
Miami, Florida, 33136
Columbia University Medical center
New York, New York, 10032
California Pacific Medical Center Research Institute
San Francisco, California, 94107
HonorHealth Neurology
Scottsdale, Arizona, 85258
Washington University School of Medicine
St Louis, Missouri, 63110
Canada3 sites
University of Calgary
Calgary, Alberta, T2N4Z6
Genge Partners
Montreal, Quebec, H4A 3T2
Sunnybrook Health Sciences Centre
Toronto, Ontario, M4N 3M5
Brazil2 sites
Hospital Sao Paulo
São Paulo, São Paulo, 04037-002
PSEG Centro de Pesquisa Clinica
São Paulo, 04038-002
Germany2 sites
Medizinische Hochschule Hannover
Hanover, Lower Saxony, 30625
Universitaetsklinikum Ulm
Ulm, Baden-Wurttemberg, 89081
Japan2 sites
University of Tokyo Hospital
Bunkyō City, Tokyo-To, 113-8655
Kagoshima University Hospital
Kagoshima, Kagoshima-ken, 890-8520
Poland2 sites
NeuroProtect Sp. z o.o.
Warsaw, Masovian Voivodeship, 01-684
Centrum Medyczne NeuroProtect
Warsaw, 01-684
Sweden2 sites
University Hospital of Umea
Umeå, Västerbotten County, 90185
Norrlands Universitetssjukhus
Umeå, 90185
Australia1 site
Macquarie University Hospital
Macquarie Park, New South Wales, 2109
Italy1 site
Azienda Ospedaliero-Universitaria Città della Salute e della Scienza di Torino
Torino, 10124
United Kingdom1 site
University of Sheffield
Sheffield, South Yorkshire, S10 2RX
About this study
Official title: A Phase 3 Randomized, Placebo-Controlled Trial With a Longitudinal Natural History Run-In and Open-Label Extension to Evaluate BIIB067 Initiated in Clinically Presymptomatic Adults With a Confirmed Superoxide Dismutase 1 Mutation
In this study, researchers will learn more about the effects of a drug called tofersen, also known as BIIB067 and QALSODY®, in adults with amyotrophic lateral sclerosis (ALS) who have a superoxide dismutase 1 (SOD1) gene mutation.
One of the causes of ALS is a change (or mutation) in the SOD1 gene, which causes abnormal SOD1 protein to be made. This abnormal protein can cause the motor neurons in people with ALS to break down and die. When neurons are damaged, they release a protein called neurofilament light chain, also known as NfL. Previous studies have shown that NfL levels are higher in people with SOD1-ALS, even before any ALS symptoms appear.
Read more
In this study, researchers will look at the effect of tofersen treatment in the earlier stages of the disease, even before ALS symptoms appear. Researchers believe that giving tofersen to patients as early as possible may slow the onset of ALS symptoms and the progression of the disease.
This study will have 4 parts. In Part A, participants will have their health monitored and NfL levels tested on a monthly basis, but they will not receive any study drug. Tofersen or placebo will be given to participants in Parts B, while all participants will receive tofersen in Parts C and D.
The main question that researchers want to answer is:
- How long does it take for ALS symptoms to appear?
Researchers will also learn more about:
- How many participants develop ALS symptoms within 24 months of the start of Part B
- Changes in participants' ability to perform their daily activities
- Changes in participants' ability to breathe
- How many participants die or need long-term breathing support
- Changes in NfL levels in the blood
- Changes in SOD1 protein levels in the fluid around the brain and spinal cord
- How many participants have adverse events or serious adverse events. Adverse events are health problems that may or may not be caused by the study drug.
The study will be done as follows:
- Participants will first be screened to check if they can join the study. The screening period will be up to 6 weeks. Participants will join the study only after reading and signing an informed consent form, also known as an ICF.
- In Part A, participants with presymptomatic ALS will not get any study drugs. Researchers will check the levels of NfL in their blood. Participants will be closely checked for any signs or symptoms of ALS.
- Once a participant's NfL levels rise to a certain amount, they will move onto Part B.
- In Part B, participants will be randomly assigned to get either tofersen or a placebo. Neither participants nor researchers will know who gets tofersen or the placebo during Part B. A placebo looks like the study drug but contains no medicine.
- Participants will get the first 3 doses of tofersen once every 14 days. Afterwards, they will continue getting maintenance doses every 28 days.
- All doses will be given through an intrathecal (IT) injection done through a lumbar puncture procedure. This involves injecting the drug into the fluid surrounding the spinal cord in the lower back.
- Participants who develop ALS symptoms during Part B will move to Part C to receive tofersen.
- Participants who develop ALS symptoms during Part A will move to Part D to receive tofersen.
- In both Parts C and D, participants will know they are receiving tofersen.
- Throughout the study, doctors will measure any changes to the participants' function and quality of life using different questionnaires. Doctors will also take blood and urine samples. They will also check the participants' overall health and ask how they are feeling.
- Participants will have a follow-up visit 28 days after their last dose of study drug.
- Participants will be in this study for up to about 6.5 years.
Read the detailed description
The primary objective of this study is to evaluate the efficacy of tofersen in presymptomatic adult carriers of a superoxide dismutase 1 (SOD1) mutation with elevated neurofilament (NF). The secondary objectives of this study are to evaluate the safety and tolerability of tofersen and to evaluate the effect of tofersen on pharmacodynamics (PD)/treatment response biomarkers when initiated prior to versus at the time of emergence of clinically manifest ALS.
- Being tested
- Tofersen, Placebo
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
PlaceboDrug
Administered as specified in the treatment arm
| Study group | Role | What they receive |
|---|---|---|
| Part A: Natural History Run-in | No intervention | Participants enrolled in Part A will undergo blood draws approximately once every 28 days to assess NfL levels. |
| Part B: Randomized, Double-Blind, Placebo-Controlled | Experimental | Participants from Part A who meet the protocol-defined NfL threshold and remain presymptomatic may be eligible to participate in Part B. During Part B, participants will receive tofersen 100 milligrams (mg) or placebo via IT injection on Days 1, 15, 29, and every 28 days thereafter for up to approximately 5.6 years. Receives: Drug: Tofersen; Drug: Placebo |
| Part C: Open-Label Extension | Experimental | Participants from Part B who develop clinically manifest ALS may be eligible to participate in Part C. During Part C, participants who received placebo in Part B will receive tofersen 100 mg via IT injection on Days 1, 15, 29, and every 28 days thereafter up to the final maintenance dose visit. Participants who received tofersen during Part B will receive tofersen 100 mg on Days 1, 29, and every 28 days thereafter up to the final maintenance dose visit, with a dose of placebo on Day 15 to maintain the study blind. The combined duration of Part B and Part C is up to approximately 5.6 years. Receives: Drug: Tofersen; Drug: Placebo |
| Part D: Open-Label Treatment | Experimental | Participants from Part A who develop clinically manifest ALS prior to randomization in Part B may be eligible to participate in Part D. During Part D, participants will receive tofersen 100 mg via IT injection on Days 1, 15, 29, and every 28 days thereafter for up to 2 years. Receives: Drug: Tofersen |
What is being measured
The primary outcome is the main question the study is designed to answer.
Part B: Time to Emergence of Clinically Manifest ALS
Measured: Up to 5.6 years
Secondary outcomes (8)
Parts B and C: Percentage of Participants with Emergence of Clinically Manifest ALS Within 24 Months of Part B Baseline
Measured: Up to 24 months
Parts B and C: Change in ALS Functional Rating Scale (ALSFRS-R) Total Score
The ALSFRS-R measures 4 functional domains: respiratory, bulbar function, gross motor skills, and fine motor skills. There are 12 questions, each scored from 0 to 4, for a total possible score of 48, with higher scores representing better function.
Measured: Up to 5.6 years
Parts B and C: Change from Baseline in Percent Predicted Slow Vital Capacity (SVC)
Measured: Up to 5.6 years
Parts B and C: Percentage of Participants with Outcome as Death or Permanent Ventilation Based on Time to Death or Permanent Ventilation Analysis
Permanent ventilation is defined as ≥22 hours of invasive or non-invasive mechanical ventilation per day for ≥21 consecutive days.
Measured: Up to 5.6 years
Parts B and C: Percentage of Participants with Outcome as Deaths Based on Time to Death Analysis
Measured: Up to 5.6 years
Parts B, C and D: Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) during the Treatment Period
Measured: Parts B and C: Up to 5.6 years and Part D: Up to 2 years
Parts B, C and D: Change from Baseline in Plasma NfL Concentrations
Measured: Parts B and C: Up to 5.6 years and Part D: Up to 2 years
Parts B, C and D: Change in Total Cerebrospinal Fluid (CSF) SOD1 Concentrations
Measured: Parts B and C: Up to 5.6 years and Part D: Up to 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 Apr 2021
- Study start17 May 2021
- Primary completion7 Aug 2027 (estimated)
- Study completion30 Apr 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Amyotrophic Lateral Sclerosis Associated With a SOD1 Gene MutationStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Investigator, Outcomes assessor
- Enrolment
- 158 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
- NCT04856982
- Sponsor's protocol ID
- 233AS303
- Other IDs
- 2023-505641-12, 2023
- Acronym
- ATLAS
- Status last verified
- Oct 2026
- First submitted
- 20 Apr 2021
- Last updated
- 20 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- 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/
- Expanded access
- Available
Publications
Benatar M, Wuu J, Andersen PM, Bucelli RC, Andrews JA, Otto M, Farahany NA, Harrington EA, Chen W, Mitchell AA, Ferguson T, Chew S, Gedney L, Oakley S, Heo J, Chary S, Fanning L, Graham D, Sun P, Liu Y, Wong J, Fradette S. Design of a Randomized, Placebo-Controlled, Phase 3 Trial of Tofersen Initiated in Clinically Presymptomatic SOD1 Variant Carriers: the ATLAS Study. Neurotherapeutics. 2022 Jul;19(4):1248-1258. doi: 10.1007/s13311-022-01237-4. Epub 2022 May 18.
PubMed 35585374
Study at a glance
From the registry record, last updated by the sponsor on 20 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3 Randomized, Placebo-Controlled Trial With a Longitudinal Natural History Run-In and Open-Label Extension to Evaluate BIIB067 Initiated in Clinically Presymptomatic Adults With a Confirmed Superoxide Dismutase 1 Mutation
- ClinicalTrials.gov ID
- NCT04856982
- Acronym / sponsor ID
- ATLAS / 233AS303
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 158 participants (actual)
- Lead sponsor
- BiogenIndustry (pharma, biotech, devices)
- Study start
- 17 May 2021 (actual)
- Primary completion
- 7 Aug 2027 (estimated)
- Study completion
- 30 Apr 2028 (estimated)
- First posted
- 23 Apr 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.
In this study, researchers will learn more about the effects of a drug called tofersen, also known as BIIB067 and QALSODY®, in adults with amyotrophic lateral sclerosis (ALS) who have a superoxide dismutase 1 (SOD1) gene mutation.
One of the causes of ALS is a change (or mutation) in the SOD1 gene, which causes abnormal SOD1 protein to be made. This abnormal protein can cause the motor neurons in people with ALS to break down and die. When neurons are damaged, they release a protein called neurofilament light chain, also known as NfL. Previous studies have shown that NfL levels are higher in people with SOD1-ALS, even before any ALS symptoms appear.
In this study, researchers will look at the effect of tofersen treatment in the earlier stages of the disease, even before ALS symptoms appear. Researchers believe that giving tofersen to patients as early as possible may slow the onset of ALS symptoms and the progression of the disease.
This study will have 4 parts. In Part A, participants will have their health monitored and NfL levels tested on a monthly basis, but they will not receive any study drug. Tofersen or placebo will be given to participants in Parts B, while all participants will receive tofersen in Parts C and D.
The main question that researchers want to answer is:
- How long does it take for ALS symptoms to appear?
Researchers will also learn more about:
- How many participants develop ALS symptoms within 24 months of the start of Part B
- Changes in participants' ability to perform their daily activities
- Changes in participants' ability to breathe
- How many participants die or need long-term breathing support
- Changes in NfL levels in the blood
- Changes in SOD1 protein levels in the fluid around the brain and spinal cord
- How many participants have adverse events or serious adverse events. Adverse events are health problems that may or may not be caused by the study drug.
The study will be done as follows:
- Participants will first be screened to check if they can join the study. The screening period will be up to 6 weeks. Participants will join the study only after reading and signing an informed consent form, also known as an ICF.
- In Part A, participants with presymptomatic ALS will not get any study drugs. Researchers will check the levels of NfL in their blood. Participants will be closely checked for any signs or symptoms of ALS.
- Once a participant's NfL levels rise to a certain amount, they will move onto Part B.
- In Part B, participants will be randomly assigned to get either tofersen or a placebo. Neither participants nor researchers will know who gets tofersen or the placebo during Part B. A placebo looks like the study drug but contains no medicine.
- Participants will get the first 3 doses of tofersen once every 14 days. Afterwards, they will continue getting maintenance doses every 28 days.
- All doses will be given through an intrathecal (IT) injection done through a lumbar puncture procedure. This involves injecting the drug into the fluid surrounding the spinal cord in the lower back.
- Participants who develop ALS symptoms during Part B will move to Part C to receive tofersen.
- Participants who develop ALS symptoms during Part A will move to Part D to receive tofersen.
- In both Parts C and D, participants will know they are receiving tofersen.
- Throughout the study, doctors will measure any changes to the participants' function and quality of life using different questionnaires. Doctors will also take blood and urine samples. They will also check the participants' overall health and ask how they are feeling.
- Participants will have a follow-up visit 28 days after their last dose of study drug.
- Participants will be in this study for up to about 6.5 years.
Detailed description
The primary objective of this study is to evaluate the efficacy of tofersen in presymptomatic adult carriers of a superoxide dismutase 1 (SOD1) mutation with elevated neurofilament (NF). The secondary objectives of this study are to evaluate the safety and tolerability of tofersen and to evaluate the effect of tofersen on pharmacodynamics (PD)/treatment response biomarkers when initiated prior to versus at the time of emergence of clinically manifest ALS.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, investigator, outcomes assessor)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Part A: Natural History Run-in | No intervention | –Participants enrolled in Part A will undergo blood draws approximately once every 28 days to assess NfL levels. |
| Part B: Randomized, Double-Blind, Placebo-Controlled | Experimental | Drug: Tofersen, Drug: PlaceboParticipants from Part A who meet the protocol-defined NfL threshold and remain presymptomatic may be eligible to participate in Part B. During Part B, participants will receive tofersen 100 milligrams (mg) or placebo via IT injection on Days 1, 15, 29, and every 28 days thereafter for up to approximately 5.6 years. |
| Part C: Open-Label Extension | Experimental | Drug: Tofersen, Drug: PlaceboParticipants from Part B who develop clinically manifest ALS may be eligible to participate in Part C. During Part C, participants who received placebo in Part B will receive tofersen 100 mg via IT injection on Days 1, 15, 29, and every 28 days thereafter up to the final maintenance dose visit. Participants who received tofersen during Part B will receive tofersen 100 mg on Days 1, 29, and every 28 days thereafter up to the final maintenance dose visit, with a dose of placebo on Day 15 to maintain the study blind. The combined duration of Part B and Part C is up to approximately 5.6 years. |
| Part D: Open-Label Treatment | Experimental | Drug: TofersenParticipants from Part A who develop clinically manifest ALS prior to randomization in Part B may be eligible to participate in Part D. During Part D, participants will receive tofersen 100 mg via IT injection on Days 1, 15, 29, and every 28 days thereafter for up to 2 years. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
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 | Part B: Randomized, Double-Blind, Placebo-Controlled, Part C: Open-Label Extension, Part D: Open-Label Treatment |
| PlaceboAdministered as specified in the treatment arm | Drug | Part B: Randomized, Double-Blind, Placebo-Controlled, Part C: Open-Label Extension |
Primary outcome
Part B: Time to Emergence of Clinically Manifest ALS
Time frame: Up to 5.6 years
8 secondary outcomes
Parts B and C: Percentage of Participants with Emergence of Clinically Manifest ALS Within 24 Months of Part B Baseline
Time frame: Up to 24 months
Parts B and C: Change in ALS Functional Rating Scale (ALSFRS-R) Total Score
Time frame: Up to 5.6 years
Parts B and C: Change from Baseline in Percent Predicted Slow Vital Capacity (SVC)
Time frame: Up to 5.6 years
Parts B and C: Percentage of Participants with Outcome as Death or Permanent Ventilation Based on Time to Death or Permanent Ventilation Analysis
Time frame: Up to 5.6 years
Parts B and C: Percentage of Participants with Outcome as Deaths Based on Time to Death Analysis
Time frame: Up to 5.6 years
Parts B, C and D: Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) during the Treatment Period
Time frame: Parts B and C: Up to 5.6 years and Part D: Up to 2 years
Parts B, C and D: Change from Baseline in Plasma NfL Concentrations
Time frame: Parts B and C: Up to 5.6 years and Part D: Up to 2 years
Parts B, C and D: Change in Total Cerebrospinal Fluid (CSF) SOD1 Concentrations
Time frame: Parts B and C: Up to 5.6 years and Part D: Up to 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 20 Oct 2026.
- Derived from this study: PubMed 35585374Benatar M, Wuu J, Andersen PM, Bucelli RC, Andrews JA, Otto M, Farahany NA, Harrington EA, Chen W, Mitchell AA, Ferguson T, Chew S, Gedney L, Oakley S, Heo J, Chary S, Fanning L, Graham D, Sun P, Liu Y, Wong J, Fradette S. Design of a Randomized, Placebo-Controlled, Phase 3 Trial of Tofersen Initiated in Clinically Presymptomatic SOD1 Variant Carriers: the ATLAS Study. Neurotherapeutics. 2022 Jul;19(4):1248-1258. doi: 10.1007/s13311-022-01237-4. Epub 2022 May 18.
- Disclaimer: Residents in the United States may click here to find out more about participation in this trial.Link provided by the sponsor.
- 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
32 sites in 14 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Tofersen in people with Amyotrophic Lateral Sclerosis Associated With a SOD1 Gene Mutation, run by Biogen at 32 sites in 14 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
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 20 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 32 sites in United States, Canada, Brazil, Germany, Japan and 9 more countries. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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?
Part B: Time to Emergence of Clinically Manifest ALS (time frame: Up to 5.6 years). 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 NCT04856982, which also lists 1 linked publication.
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 20 Oct 2026.