CI

At a glance

ClinicalIndex Comparison Record
Phase 3Completed· 176 enrolled
Drug / intervention
Tofersen +1 moredrug
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/NCT02623699
NCT02623699Phase 3Completed
CompareCT.gov

A Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BIIB067 Administered to Adult Subjects With Amyotrophic Lateral Sclerosis and Confirmed Superoxide Dismutase 1 Mutation

Biogen·interventional·Posted Dec 8, 2015·Updated Jul 28, 2023

In Brief

A Phase 3 clinical trial evaluating Tofersen and Placebo for Amyotrophic Lateral Sclerosis. Completed, enrolled 176 participants across 40 sites in 12 countries.

Detailed Summary

The primary objectives of Parts A and B of this study are to evaluate the safety, tolerability, and pharmacokinetics (PK) of ascending doses of tofersen in adults with ALS and a documented superoxide dismutase 1 (SOD1) mutation. The primary objective of Part C of this study is to evaluate the clinical efficacy of tofersen administered to adults with ALS and a confirmed SOD1 mutation. The secondary objective of Parts A and B of this study is to evaluate the effects of tofersen on levels of total SOD1 protein in the cerebrospinal fluid (CSF). The secondary objectives of Part C are to evaluate the safety, tolerability, pharmacodynamic (PD), and biomarker effects of tofersen.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsAmyotrophic Lateral Sclerosis
CountriesAustralia, Belgium, Canada, Denmark, France, Germany, Italy, Japan, Poland, South Korea, United Kingdom, United States
CollaboratorsIonis Pharmaceuticals, Inc.

Timeline

Phase 3CompletedFinished
20162017201820192020202120222023202420252026
First PostedDec 8, 2015
Enrollment StartJan 20, 2016
Primary CompletionJul 16, 2021
TodayJul 2, 2026
Enrollment to primary: 5.5 yearsPosted 10.6 years ago

Interventions

Tofersendrug

Administered as specified in the treatment arm.

Placebodrug

Administered as specified in the treatment arm.