NCT04856982

A Study of BIIB067 (Tofersen) Initiated in Clinically Presymptomatic Adults With a Confirmed Superoxide Dismutase 1 Mutation

A Phase 3 Randomized, Placebo-Controlled Trial With a Longitudinal Natural History Run-In and Open-Label Extension to Evaluate BIIB067 Initiated in Clinically Presymptomatic Adults With a Confirmed Superoxide Dismutase 1 Mutation

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 158 (actual)
• Sponsor: Biogen · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The primary objective of this study is to evaluate the efficacy of tofersen in presymptomatic adult carriers of a superoxide dismutase 1 (SOD1) mutation with elevated neurofilament (NF). The secondary objectives of this study are to evaluate the safety and tolerability tofersen and to evaluate the effect of tofersen on pharmacodynamics (PD)/treatment response biomarkers when initiated prior to versus at the time of emergence of clinically manifest amyotrophic lateral sclerosis (ALS).

Conditions

• Amyotrophic Lateral Sclerosis Associated With a SOD1 Gene Mutation

Interventions

Type	Name	Description
DRUG	Tofersen	Administered as specified in the treatment arm
DRUG	Placebo	Administered as specified in the treatment arm

Timeline

• Start date: 2021-05-17
• Primary completion: 2027-08-07
• Completion: 2028-04-30
• First posted: 2021-04-23
• Last updated: 2026-03-30

Locations

32 sites across 14 countries: United States, Australia, Belgium, Brazil, Canada, France, Germany, Italy, Japan, Poland, South Korea, Spain, Sweden, United Kingdom

Regulatory

• FDA-regulated drug study