Summary

Eligibility
for people ages 18 years and up (full criteria)
Location
at UCSD
Dates
study started
study ends around

Description

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).

Official Title

A Phase 3 Randomized, Placebo-Controlled Trial With a Longitudinal Natural History Run-In and Open-Label Extension to Evaluate BIIB067 Initiated in Clinically Presymptomatic Adults With a Confirmed Superoxide Dismutase 1 Mutation

Keywords

Amyotrophic Lateral Sclerosis Associated With a SOD1 Gene Mutation, Motor Neuron Disease, Amyotrophic Lateral Sclerosis, Tofersen, Natural History Run-in, Open-Label Extension

Eligibility

Locations

  • University of California San Diego Medical Center
    La Jolla California 92093-0949 United States
  • California Pacific Medical Center Research Institute
    San Francisco California 94107 United States

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Biogen
Links
Disclaimer: Residents in the United States may click here to find out more about participation in this trial.
ID
NCT04856982
Phase
Phase 3 Amyotropic Lateral Sclerosis (ALS) Research Study
Study Type
Interventional
Participants
About 158 people participating
Last Updated