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 · Biogen

Recruiting

In plain English

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Official title

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

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.

Eligibility criteria

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 design

Enrollment target: 125 participants

Age groups: adult, older_adult

Timeline

Starts: 2026-01-02

Estimated completion: 2033-12-30

Last updated: 2026-04-09

Interventions

Drug: Tofersen

Primary outcomes

• Baseline Demographic: Age (At Baseline)
• Baseline Demographic: Participant Sex (At Baseline)
• Baseline Demographic: Race/Ethnicity (At Baseline)

Sponsor

Biogen · industry

Contacts & investigators

ContactStudy US Biogen Clinical Trial Center · contact · clinicaltrials@biogen.com · 866-633-4636

ContactGlobal Biogen Clinical Trial Center · contact · clinicaltrials@biogen.com

InvestigatorMedical Director · study_director, Biogen

All locations (1)

Mass General Hospital -MGHRecruiting

Boston, Massachusetts, United States

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