An Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of BIIB067 in Adults With Inherited Amyotrophic Lateral Sclerosis (ALS)
Completed
Ionis Pharmaceuticals, Inc.
Phase 3
2016-01-20
The primary objectives of Parts A and B of this study are to evaluate the safety,
tolerability, and pharmacokinetics (PK) of ascending doses of BIIB067 (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 BIIB067 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 BIIB067
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 BIIB067.
An Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of BIIB067 in Adults With Inherited Amyotrophic Lateral Sclerosis (ALS)
Completed
Biogen
Phase 3
2016-01-20
The primary objectives of Parts A and B of this study are to evaluate the safety,
tolerability, and pharmacokinetics (PK) of ascending doses of BIIB067 (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 BIIB067 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 BIIB067
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 BIIB067.
A Study of BIIB067 When Initiated in Clinically Presymptomatic Adults With a Confirmed Superoxide Dismutase 1 Mutation
Recruiting
Biogen
Phase 3
2021-05-17
The primary objective of this study is to evaluate the efficacy of BIIB067 when initiated 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 of BIIB067 and to evaluate the effect of BIIB067 on pharmacodynamics
(PD)/treatment response biomarkers when initiated prior to versus at the time of emergence of
clinically manifest amyotrophic lateral sclerosis (ALS).
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