An Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of BIIB067 (Tofersen) in Adults With Inherited Amyotrophic Lateral Sclerosis (ALS)
Completed · Phase 3 · Has a placebo group
Conditions studied: Amyotrophic Lateral Sclerosis
In brief
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.
Key facts
- Study ID
- NCT02623699
- Run by
- Biogen
- People needed
- 176
- Starts
- 2016-01-20
- Expected to finish
- 2021-07-16
- Last updated by the study team
- 2023-07-28
Who can join
Age: 18 and older. Sex: any. Healthy volunteers: not accepted.
Where it is running
- Barrow Neurological Institute — Phoenix, Arizona, United States
- University of California San Diego Medical Center — La Jolla, California, United States
- California Pacific Medical Center — San Francisco, California, United States
- Mayo Clinic in Florida — Jacksonville, Florida, United States
- University of Miami School of Medicine — Miami, Florida, United States
- Bioclinica Research — Orlando, Florida, United States
- Emory University Hospital — Atlanta, Georgia, United States
- Northwestern University Feinberg School of Medicine — Chicago, Illinois, United States
- Johns Hopkins University — Baltimore, Maryland, United States
- Massachusetts General Hospital — Boston, Massachusetts, United States
- Henry Ford Hospital — Detroit, Michigan, United States
- Mayo Clinic - Rochester — Rochester, Minnesota, United States
- Washington University School of Medicine — St Louis, Missouri, United States
- Neurology Associates, P.C. — Lincoln, Nebraska, United States
- Columbia University Medical Center — New York, New York, United States
- The Cleveland Clinic Foundation — Cleveland, Ohio, United States
- Providence ALS Center — Portland, Oregon, United States
- University of Pennsylvania — Philadelphia, Pennsylvania, United States
- New Orleans Center for Clinical Research/Volunteer Research Group, an AMR Company — Knoxville, Tennessee, United States
- Methodist Neurological Institute — Houston, Texas, United States
- Westmead Hospital — Westmead, New South Wales, Australia
- UZ Leuven — Leuven, Belgium
- University of Calgary - Health Sciences Centre — Calgary, Alberta, Canada
- Research Site — Edmonton, Alberta, Canada
- Sunnybrook Health Sciences Centre — Toronto, Ontario, Canada
Full record on ClinicalTrials.gov
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