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

Phase 3, Randomized, Placebo-Controlled Trial

Topic / Pathology

Phase 3
Individuals with ALS risk mutations

Objectives

Primary Objective :

To evaluate the efficacy of tofersen in presymptomatic adult carriers of a superoxide dismutase 1 (SOD1) mutation with elevated neurofilament (NF).

Secondary objectives :

1. To evaluate the safety and tolerability of tofersen
2. 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).

Find out more : https://clinicaltrials.gov/ct2/show/NCT04856982