A Study of BIIB067 (Tofersen) Initiated in Clinically Presymptomatic Adults With a Confirmed Superoxide Dismutase 1 Mutation
Running, not enrolling · Phase 3 · Has a placebo group
Conditions studied: Amyotrophic Lateral Sclerosis Associated With a SOD1 Gene Mutation
In brief
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).
Key facts
- Study ID
- NCT04856982
- Run by
- Biogen
- People needed
- 158
- Starts
- 2021-05-17
- Expected to finish
- 2028-04-30
- Last updated by the study team
- 2026-03-30
Who can join
Age: 18 and older. Sex: any. Healthy volunteers: not accepted.
Where it is running
- HonorHealth Neurology — Scottsdale, Arizona, United States
- University of California San Diego Medical Center — La Jolla, California, United States
- California Pacific Medical Center Research Institute — San Francisco, California, United States
- Holy Cross Hospital — Fort Lauderdale, Florida, United States
- University of Miami School of Medicine — Miami, Florida, United States
- The Emory Clinic — Atlanta, Georgia, United States
- Northwestern Medicine — Chicago, Illinois, United States
- Johns Hopkins Hospital — Baltimore, Maryland, United States
- Massachusetts General Hospital, MA — Charlestown, Massachusetts, United States
- Washington University School of Medicine — St Louis, Missouri, United States
- Columbia University Medical center — New York, New York, United States
- Austin Neuromuscular Center — Austin, Texas, United States
- Macquarie University Hospital — Macquarie Park, New South Wales, Australia
- UZ Leuven — Leuven, Belgium
- Hospital Sao Paulo — São Paulo, São Paulo, Brazil
- PSEG Centro de Pesquisa Clinica — São Paulo, Brazil
- University of Calgary — Calgary, Alberta, Canada
- Sunnybrook Health Sciences Centre — Toronto, Ontario, Canada
- Genge Partners — Montreal, Quebec, Canada
- Groupe Hospitalier Pitie-Salpetriere — Paris, Paris, France
- Universitaetsklinikum Ulm — Ulm, Baden-Wurttemberg, Germany
- Medizinische Hochschule Hannover — Hanover, Lower Saxony, Germany
- Azienda Ospedaliero-Universitaria Città della Salute e della Scienza di Torino — Torino, Italy
- Kagoshima University Hospital — Kagoshima, Kagoshima-ken, Japan
- University of Tokyo Hospital — Bunkyō City, Tokyo-To, Japan
Full record on ClinicalTrials.gov
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