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An Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of BIIB067 (Tofersen) in Adults With Inherited Amyotrophic Lateral Sclerosis (ALS)
PLS AVAILABLE
Details
Development Status
Approved
Study Type
Interventional
Results Available
Yes
Study Dates
01/2016 - 07/2021
Enrollment
176
Commercial Product
QALSODY™
Study Drug
Placebo, BIIB067 (tofersen)
Summary
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.
Study Results
View and download the Results Summaries with the results of this completed study.
CSR Synopsis
PDF Document
Plain Language Summary
PDF Document
View study results on registries
