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)

NCT07259980 | Recruiting FDA Drug

• 1 other identifier: 233AS401
• Study Type: observational
• Target: 125
• Locations: 1 country
• Sites: 1

Brief 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. 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.

Conditions

Amyotrophic Lateral Sclerosis

Outcome Measures

Primary Outcomes (16)

• 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 Outcomes (4)

• 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

Study Arms (1)

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

Interventions

Tofersen DRUG

Administered as specified in the treatment arm.

Also known as: BIIB067, Qalsody

Tofersen

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• 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).

You may not qualify if:

• 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.

Sponsors & Collaborators

• Biogen: lead

Study Sites (1)

Mass General Hospital -MGH

Boston, Massachusetts, 02114, United States

RECRUITING

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Interventions

tofersen

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Medical Director

  Biogen

  STUDY DIRECTOR

Central Study Contacts

Study US Biogen Clinical Trial Center

CONTACT

866-633-4636; clinicaltrials@biogen.com

Global Biogen Clinical Trial Center

CONTACT

clinicaltrials@biogen.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 7 Years
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 31, 2025
• First Posted: December 2, 2025
• Study Start: January 2, 2026
• Primary Completion (Estimated): December 30, 2033
• Study Completion (Estimated): December 30, 2033
• Last Updated: April 9, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share

Locations

US (1)