Long-Term Evaluation of BIIB067 (Tofersen)
Completed · Phase 3
Conditions studied: ALS Caused by Superoxide Dismutase 1 (SOD1) Mutation
In brief
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.
Key facts
- Study ID
- NCT03070119
- Run by
- Biogen
- People needed
- 139
- Starts
- 2017-03-08
- Expected to finish
- 2024-08-12
- Last updated by the study team
- 2025-08-29
Who can join
Age: 18 and older. Sex: any. Healthy volunteers: not accepted.
Where it is running
- Research Site — Phoenix, Arizona, United States
- Research Site — La Jolla, California, United States
- Research Site — San Francisco, California, United States
- Research Site — Jacksonville, Florida, United States
- Research Site — Miami, Florida, United States
- Research Site — Orlando, Florida, United States
- Research Site — Atlanta, Georgia, United States
- Research Site — Chicago, Illinois, United States
- Research Site — Baltimore, Maryland, United States
- Research Site — Boston, Massachusetts, United States
- Research Site — Rochester, Minnesota, United States
- Research Site — St Louis, Missouri, United States
- Research Site — Lincoln, Nebraska, United States
- Research Site — Cleveland, Ohio, United States
- Research Site — Portland, Oregon, United States
- Research Site — Knoxville, Tennessee, United States
- Research Site — Leuven, Belgium
- Research Site — Calgary, Alberta, Canada
- Research Site — Edmonton, Alberta, Canada
- Research Site — Toronto, Ontario, Canada
- Research Site — Montreal, Quebec, Canada
- Bispebjerg Hospital — Copenhagen, Denmark
- Research Site — Clermont-Ferrand, Puy De Dome, France
- Research Site — Ulm, Baden-Wurttemberg, Germany
- Research Site — Torino, Italy
Full record on ClinicalTrials.gov
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