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 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:

• 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.

Study Overview

• Status: Recruiting
• Conditions: Amyotrophic Lateral Sclerosis
• Intervention / Treatment: Drug: Tofersen

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 Type: Observational
• Enrollment (Estimated): 125

Contacts and Locations

• Study Contact: Name: Global Biogen Clinical Trial Center; Email: clinicaltrials@biogen.com
• Study Contact Backup: Name: Study US Biogen Clinical Trial Center; Phone Number: 866-633-4636; Email: clinicaltrials@biogen.com

Study Locations

• United States
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Recruiting
      • Mass General Hospital -MGH
      • Contact: Phone Number: 617-643-2935
      • Principal Investigator: Alex Sherman

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

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).

Description

Key Inclusion Criteria:

• 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:

• 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.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
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.	Drug: Tofersen; Administered as specified in the treatment arm.; Other Names: BIIB067, Qalsody

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Baseline Demographic: Age	At Baseline
Baseline Demographic: Participant Sex	At Baseline
Baseline Demographic: Race/Ethnicity	At Baseline
Baseline Demographic: Weight	At Baseline
Baseline Demographic: Height	At Baseline
Baseline Demographic: Body Mass Index (BMI)	At Baseline
Baseline Demographic: Family History of Amyotrophic Lateral Sclerosis (ALS)	At Baseline
Clinical Characteristics: Age at Diagnosis and Symptom Onset	At Baseline
Clinical Characteristics: Revised El Escorial Classification	At Baseline
Clinical Characteristics: Classification of SOD1-ALS Clinical Phenotypes	At Baseline
Clinical Characteristics: SOD1 Mutation Type	At Baseline
Clinical Characteristics: Medical History	At Baseline
Clinical Characteristics: Concomitant Medications	At Baseline
Clinical Characteristics: Disease History	At Baseline
Clinical Characteristics: Pregnancy Status	At Baseline
Number of Participants With Serious Adverse Events	Up to 7 years

Secondary Outcome Measures

Outcome Measure	Time Frame
Number of Participants With New Comorbid Conditions	Up to 7 years
Number of Participants With Pregnancy and Pregnancy Outcomes	Up to 7 years
Number of Participants With Reported Treatment Discontinuation	Up to 7 years
Number of Participants With Reported Reasons for Treatment Discontinuation	Up to 7 years

Collaborators and Investigators

• Sponsor: Biogen
• Investigators: Study Director: Medical Director, Biogen

Study record dates

Study Major Dates

• Study Start (Actual): January 2, 2026
• Primary Completion (Estimated): December 30, 2033
• Study Completion (Estimated): December 30, 2033

Study Registration Dates

• First Submitted: October 31, 2025
• First Submitted That Met QC Criteria: November 28, 2025
• First Posted (Actual): December 2, 2025

Study Record Updates

• Last Update Posted (Actual): April 9, 2026
• Last Update Submitted That Met QC Criteria: April 6, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Central Nervous System Diseases; Nervous System Diseases; Neuromuscular Diseases; Metabolic Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Motor Neuron Disease; Nutritional and Metabolic Diseases; Amyotrophic Lateral Sclerosis; tofersen
• Other Study ID Numbers: 233AS401

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No