158 Participants Needed

Tofersen for ALS

(ATLAS Trial)

Recruiting at 66 trial locations
UB
GB
RR
NJ
DN
KR
Overseen ByKristen Riley
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Pivotal Trial (Near Approval)This treatment is in the last trial phase before FDA approval
Prior Safety DataThis treatment has passed at least one previous human trial

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial is testing a medication called tofersen in adults who have a genetic mutation that can lead to ALS, a serious nerve disease. These individuals show early signs of nerve damage. Tofersen works by lowering harmful proteins in the body to protect nerves and potentially delay or prevent the disease.

Do I need to stop my current medications to join the trial?

Yes, you may need to stop certain medications. If you are taking riluzole, edaravone, or sodium phenylbutyrate/taurursodiol, you must discontinue them for at least 5 half-lives before screening. You also cannot use off-label ALS treatments or other investigational drugs within a specified period before the study.

Will I have to stop taking my current medications?

You may need to stop taking certain medications like riluzole, edaravone, and sodium phenylbutyrate/taurursodiol at least 5 half-lives before the screening. The protocol does not specify about other medications, so it's best to discuss with the study team.

What safety data exists for Tofersen in ALS treatment?

Tofersen, also known as Qalsody, has been studied in various clinical trials, including a phase III study (VALOR) and a phase 1-2 trial, for its safety and efficacy in treating ALS associated with SOD1 mutations. It was approved by the US FDA on April 25, 2023, for adults with SOD1 ALS, indicating that safety data was sufficient for regulatory approval. Additionally, it has been used in an expanded access program, suggesting ongoing evaluation of its safety profile in clinical practice.12345

Is Tofersen safe for humans?

Tofersen, also known as Qalsody, has been approved for treating a specific type of ALS in adults, indicating it has undergone safety evaluations. However, the available research primarily focuses on its use for ALS, and specific safety details are not provided in the abstracts.12345

Is Tofersen a promising drug for ALS?

Tofersen is considered a promising drug for ALS because it has shown potential in treating the disease, offering hope for patients who have limited treatment options.678910

What data supports the idea that Tofersen for ALS is an effective drug?

The available research shows that Tofersen, also known as Qalsody, is effective for treating ALS in adults with a specific genetic mutation. It was approved in the USA for this purpose in April 2023. Tofersen works by targeting and reducing a harmful protein linked to ALS. Unlike other treatments like riluzole and edaravone, which only help with symptoms, Tofersen addresses a key cause of the disease. This makes it the first gene therapy for ALS, offering a new way to manage the condition.123511

What data supports the effectiveness of the drug Tofersen for ALS?

Tofersen has been approved in the USA for treating ALS in adults with a specific genetic mutation (SOD1 gene), and it works by reducing the production of a harmful protein linked to the disease. It is the first gene therapy for ALS, addressing a key aspect of the disease's progression.123511

Who Is on the Research Team?

MD

Medical Director

Principal Investigator

Biogen

Are You a Good Fit for This Trial?

This trial is for adults who carry a specific gene mutation (SOD1) linked to ALS but don't yet show symptoms. They must have low neurofilament levels and not be on certain ALS treatments or other clinical trials. People with severe mental health issues, active infections like HIV or hepatitis, or those at risk of bleeding complications can't participate.

Inclusion Criteria

I do not show any symptoms of ALS.
I do not show any symptoms of ALS.
My ALS is due to a specific SOD1 mutation confirmed by experts.
See 2 more

Exclusion Criteria

I have stopped my ALS medications for enough time before screening.
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
I haven't taken any experimental drugs or treatments recently.
See 10 more

Timeline for a Trial Participant

Natural History Run-in

Participants undergo blood draws approximately once every 28 days to assess neurofilament light chain (NfL) levels

Varies
1 visit every 28 days

Randomized, Double-Blind, Placebo-Controlled

Participants receive tofersen 100 mg or placebo via intrathecal injection on Days 1, 15, 29, and every 28 days thereafter

Up to approximately 5.6 years
1 visit every 28 days

Open-Label Treatment

Participants receive tofersen 100 mg via IT injection on Days 1, 15, 29, and every 28 days thereafter for up to 2 years

Up to 2 years
1 visit every 28 days

Open-Label Extension

Participants who develop clinically manifest ALS may receive tofersen 100 mg via IT injection on Days 1, 29, and every 28 days thereafter

Up to approximately 5.6 years
1 visit every 28 days

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

  • BIIB067 (Tofersen)
  • Placebo
  • Tofersen
Trial Overview The study tests Tofersen's effectiveness in delaying the onset of ALS symptoms in people with an SOD1 mutation. Participants will either receive Tofersen or a placebo without knowing which one they're getting to compare outcomes fairly.
How Is the Trial Designed?
4Treatment groups
Experimental Treatment
Active Control
Group I: Part D: Open-Label TreatmentExperimental Treatment1 Intervention
Group II: Part C: Open-Label ExtensionExperimental Treatment2 Interventions
Group III: Part B: Randomized, Double-Blind, Placebo-ControlledExperimental Treatment2 Interventions
Group IV: Part A: Natural History Run-inActive Control1 Intervention

BIIB067 (Tofersen) is already approved in United States, European Union for the following indications:

🇺🇸
Approved in United States as Qalsody for:
🇪🇺
Approved in European Union as Qalsody for:

Find a Clinic Near You

Who Is Running the Clinical Trial?

Biogen

Lead Sponsor

Trials
655
Recruited
468,000+
Daniel Quirk profile image

Daniel Quirk

Biogen

Chief Medical Officer

MD

Christopher A. Viehbacher profile image

Christopher A. Viehbacher

Biogen

Chief Executive Officer since 2022

Graduated from Queen's University, Kingston, Ontario, Canada

Published Research Related to This Trial

Tofersen (Qalsody™) is an antisense oligonucleotide that has been approved in the USA for treating amyotrophic lateral sclerosis (ALS) specifically in adults with a mutation in the SOD1 gene.
The approval of tofersen marks a significant milestone in ALS treatment, highlighting the potential of targeted genetic therapies in managing this challenging neurodegenerative disease.
Tofersen: First Approval.Blair, HA.[2023]
In a phase 3 trial involving 108 adults with SOD1 ALS, tofersen significantly reduced levels of the SOD1 protein and neurofilament light chains in cerebrospinal fluid and plasma, indicating its mechanism of action in targeting the disease.
Despite these biochemical reductions, tofersen did not show a significant improvement in clinical outcomes, such as the ALS Functional Rating Scale score, and was associated with common adverse events related to lumbar punctures.
Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS.Miller, TM., Cudkowicz, ME., Genge, A., et al.[2022]
Tofersen, the first gene therapy approved for ALS, targets mutant SOD1 mRNA, addressing a key pathological factor in the disease and offering a potential way to slow its progression.
This review highlights that while current treatments like riluzole and edaravone only provide symptomatic relief, tofersen represents a significant advancement in ALS therapy, allowing for better monitoring and treatment options.
Breaking barriers with tofersen: Enhancing therapeutic opportunities in amyotrophic lateral sclerosis.Saini, A., Chawla, PA.[2023]

Citations

Tofersen: First Approval. [2023]
Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS. [2022]
Breaking barriers with tofersen: Enhancing therapeutic opportunities in amyotrophic lateral sclerosis. [2023]
Neurofilament light-chain response during therapy with antisense oligonucleotide tofersen in SOD1-related ALS: Treatment experience in clinical practice. [2023]
Design of a Randomized, Placebo-Controlled, Phase 3 Trial of Tofersen Initiated in Clinically Presymptomatic SOD1 Variant Carriers: the ATLAS Study. [2022]
Phase 1-2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS. [2022]
Characterization of the novel HLA-B*07:385 allele by next-generation sequencing. [2021]
A novel DRB3 allele (DRB3*0208), a new allelic variant of DRB1*1502 (DRB1*15023) and two new DQB1 (DQB1*03012 and DQB1*0614) alleles. [2019]
Characterization of the novel HLA-C*06:283 allele by next-generation sequencing. [2021]
A Novel c.796 A>C Mutation in the ABO*B.01 Allele Responsible for CisAB Phenotype. [2022]
[Study of the molecular basis for an individual with Bel variant due to deletion of B glycosyltransferase gene]. [2017]
Unbiased ResultsWe believe in providing patients with all the options.
Your Data Stays Your DataWe only share your information with the clinical trials you're trying to access.
Verified Trials OnlyAll of our trials are run by licensed doctors, researchers, and healthcare companies.
Back to top
Terms of Service·Privacy Policy·Cookies·Security