CI

At a glance

ClinicalIndex Comparison Record
Phase 3Completed· 139 enrolled
Drug / intervention
Tofersendrug
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT03070119
NCT03070119Phase 3Completed
CompareCT.gov

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

Biogen·interventional·Posted Mar 3, 2017·Updated Aug 29, 2025

In Brief

A Phase 3 clinical trial evaluating Tofersen for ALS Caused by Superoxide Dismutase 1 (SOD1) Mutation. Completed, enrolled 139 participants across 30 sites in 9 countries.

Detailed 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.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsALS Caused by Superoxide Dismutase 1 (SOD1) Mutation
CountriesBelgium, Canada, Denmark, France, Germany, Italy, Japan, United Kingdom, United States
CollaboratorsIonis Pharmaceuticals, Inc.

Timeline

Phase 3CompletedFinished
2017201820192020202120222023202420252026
First PostedMar 3, 2017
Enrollment StartMar 8, 2017
Primary CompletionAug 12, 2024
TodayJul 2, 2026
Enrollment to primary: 7.4 yearsPosted 9.3 years ago

Interventions

Tofersendrug

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