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tofersen

Generic: TOFERSEN·Brand: QALSODY, Qalsody

tofersen: 5 clinical trials tracked.

tofersen evidence translation

What we can confirm

5 tracked trials

No verified listed company relationship

What it means

AI will separate confirmed drug evidence from unknown company context.

What to watch

Watch trial status, endpoints, and the next source-backed update.
BioSniper AIPage evidence connected

Prepared research question

What is verified about tofersen, and what remains unknown?

Run cited research

Traceable citations · Unknowns marked

See linked entities and source evidence1 indications · 1 study sponsors
Clinical Trial Registry Records (5)

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

NCT07259980Registry updated 2026-03-02·BIIB
Not yet recruiting

Primary endpoint: Baseline Demographic: Age

Observational125 targetStarted 2026-03Completion 2033-121 site · United StatesBiogen

A Study to Evaluate the Biological Effect of Tofersen in Adults With Amyotrophic Lateral Sclerosis Without Mutations in SOD1

NCT07294144Registry updated 2026-06-25·Phase 2
Recruiting

Primary endpoint: Proportion of participants with ≥30% reduction in plasma NfL

Interventionalopen30 targetStarted 2025-12Completion 2028-053 sites · United StatesWashington University School of Medicine

A Multicenter, Open-Label, Postmarketing Surveillance Study of Tofersen (BIIB067) in Adults With Amyotrophic Lateral Sclerosis (ALS) Associated With a Mutation in the Superoxide Dismutase 1 (SOD1) Gene in China

NCT07223723Registry updated 2026-08-13·Phase 4·BIIB
Active, not recruiting

Primary endpoint: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

Interventionalopen12 targetStarted 2025-12Completion 2027-051 site · ChinaBiogen

An Extension Study to Assess the Long-Term Safety, Tolerability, Pharmacokinetics, and Effect on Disease Progression of BIIB067 Administered to Previously Treated Adults With Amyotrophic Lateral Sclerosis Caused by Superoxide Dismutase 1 Mutation

NCT03070119Registry updated 2025-08-29·Phase 3·BIIB
Completed

Primary endpoint: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious AEs (TESAEs)

Interventionalopen139 enrolledStarted 2017-03Completion 2024-0830 sites · Belgium, Canada, Denmark, France…Biogen

A Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BIIB067 Administered to Adult Subjects With Amyotrophic Lateral Sclerosis and Confirmed Superoxide Dismutase 1 Mutation

NCT02623699Registry updated 2023-07-28·Phase 3·BIIB
Completed

Primary endpoint: Parts A and B: Number of Participants Experiencing Adverse Events (AEs) and Serious Adverse Events (SAEs)

InterventionalRandomizedquadruple176 enrolledStarted 2016-01Completion 2021-0740 sites · Australia, Belgium, Canada, Denmark…Biogen