Full results & analysis
Available in the Gosset platform where reported.
01
Overview
Phase 4, open-label, single-arm trial of tofersen in amyotrophic lateral sclerosis (ALS), sponsored by Biogen. Primary endpoint: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) at 52 weeks. Started Dec 2025; status: active, not recruiting.
02
Design
1 arm- Study design
- Non-randomizedOpen-labelSingle group
Tofersen 100 mg
Participants receive tofersen 100 mg by intrathecal injection.
03
Who it enrols
Adults with SOD1-mutated ALS, naive to tofersen and other gene/stem cell therapies, with stable doses of riluzole or edaravone if taking them.
04
Endpoints
Primary
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Secondary
Maximum Observed Plasma Concentration (Cmax) of Tofersen
Time to Reach Maximum Plasma Concentration (Tmax) of Tofersen
Area Under the Concentration-Time Curve (AUC) of Tofersen in Plasma
Tofersen Cerebrospinal Fluid (CSF) Concentration
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Competitive context
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