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, 125 participants, Biogen

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

Registry	NCT07259980
Status	Recruiting
Study type	Observational
Enrollment	125 (Estimated)
Sponsor	Biogen (Industry)
Conditions	Amyotrophic Lateral Sclerosis
Interventions	Tofersen (Drug)
Dates	start 2026-01-02, primary completion 2033-12-30 (estimated), completion 2033-12-30 (estimated), first posted 2025-12-02, last updated 2026-04-09
Locations	1 site in United States

-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.

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+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.

+Arms and interventions

Arm	Type	Interventions	Description
Tofersen		Drug: 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.

Tofersen (Drug), also known as BIIB067, Qalsody: Administered as specified in the treatment arm.

+Outcome measures

Primary outcomes

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

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]

+Eligibility

Sex: All, ages: 18 Years to any

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.

+Locations (1)

Facility	City	Country	Status
Mass General Hospital -MGH	Boston, Massachusetts	United States	Recruiting

+Investigators

Medical Director, Study director, Biogen