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

NCT04856982 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 158

Last updated 2026-03-30

No results posted yet for this study

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

DRUG

Tofersen

Administered as specified in the treatment arm

DRUG

Placebo

Administered as specified in the treatment arm

Sponsors & Collaborators

Principal Investigators

  • Medical Director · Biogen

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-05-17
Primary Completion
2027-08-07
Completion
2028-04-30
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Belgium
  • Brazil
  • Canada
  • France
  • Germany
  • Italy
  • Japan
  • Poland
  • South Korea
  • Spain
  • Sweden
  • United Kingdom

Study Locations

More Related Trials

Entities

Drugs
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04856982 on ClinicalTrials.gov