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NCT Number: NCT07259980

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)

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.

Recruiting

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Key information

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Mass General Hospital -MGH

Boston, Massachusetts, 02114, United States

Location status: Recruiting

Location contact

Alex Sherman

PRINCIPAL_INVESTIGATOR

CONTACT

617-643-2935

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Tofersen

Drug

Administered as specified in the treatment arm.

Other names: BIIB067, Qalsody

Primary outcomes

  1. Baseline Demographic: Age

    Time frame: At Baseline

  2. Baseline Demographic: Participant Sex

    Time frame: At Baseline

  3. Baseline Demographic: Race/Ethnicity

    Time frame: At Baseline

  4. Baseline Demographic: Weight

    Time frame: At Baseline

  5. Baseline Demographic: Height

    Time frame: At Baseline

  6. Baseline Demographic: Body Mass Index (BMI)

    Time frame: At Baseline

  7. Baseline Demographic: Family History of Amyotrophic Lateral Sclerosis (ALS)

    Time frame: At Baseline

  8. Clinical Characteristics: Age at Diagnosis and Symptom Onset

    Time frame: At Baseline

  9. Clinical Characteristics: Revised El Escorial Classification

    Time frame: At Baseline

  10. Clinical Characteristics: Classification of SOD1-ALS Clinical Phenotypes

    Time frame: At Baseline

  11. Clinical Characteristics: SOD1 Mutation Type

    Time frame: At Baseline

  12. Clinical Characteristics: Medical History

    Time frame: At Baseline

  13. Clinical Characteristics: Concomitant Medications

    Time frame: At Baseline

  14. Clinical Characteristics: Disease History

    Time frame: At Baseline

  15. Clinical Characteristics: Pregnancy Status

    Time frame: At Baseline

  16. Number of Participants With Serious Adverse Events

    Time frame: Up to 7 years

Secondary outcomes

  1. Number of Participants With New Comorbid Conditions

    Time frame: Up to 7 years

  2. Number of Participants With Pregnancy and Pregnancy Outcomes

    Time frame: Up to 7 years

  3. Number of Participants With Reported Treatment Discontinuation

    Time frame: Up to 7 years

  4. Number of Participants With Reported Reasons for Treatment Discontinuation

    Time frame: Up to 7 years

Study contacts

Contact information is provided by the study sponsor or research team.

Global Biogen Clinical Trial Center

CONTACT

[email protected]

Study US Biogen Clinical Trial Center

CONTACT

[email protected]

866-633-4636

Sponsors and collaborators

Lead sponsor

Biogen

Industry

Registry information

Official study title

An Observational Registry-Based Study to Evaluate the Long-Term Safety of Tofersen in People With SOD1-ALS

Important dates

Study start
2026
Primary completion
2033
Study completion
2033
First posted
Dec 2, 2025
Registry last updated
Apr 9, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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