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Long-Term Evaluation of BIIB067 (Tofersen)

NCT03070119 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 139

Last updated 2025-08-29

Study results available
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Summary

The primary objective of the study is to evaluate the long-term safety and tolerability of BIIB067 (tofersen) in participants with amyotrophic lateral sclerosis (ALS) and confirmed superoxide dismutase 1 (SOD1) mutation. The secondary objectives are to evaluate the pharmacokinetic (PK), pharmacodynamic (PD), biomarker effects, and efficacy of BIIB067 administered to participants with ALS and a confirmed SOD1 mutation.

Conditions

  • ALS Caused by Superoxide Dismutase 1 (SOD1) Mutation

Interventions

DRUG

Tofersen

Participants will receive a loading dose regimen followed by maintenance dosing.

Sponsors & Collaborators

  • Ionis Pharmaceuticals, Inc.

    collaborator INDUSTRY

  • Biogen

    lead INDUSTRY

Principal Investigators

  • Medical Director · Biogen

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2017-03-08
Primary Completion
2024-08-12
Completion
2024-08-12
FDA Drug
Yes

Countries

  • United States
  • Belgium
  • Canada
  • Denmark
  • France
  • Germany
  • Italy
  • Japan
  • United Kingdom

Study Locations

More Related Trials

Entities

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 NCT03070119 on ClinicalTrials.gov