Last updated on January 2020

An Efficacy Safety Tolerability Pharmacokinetics and Pharmacodynamics Study of BIIB067 in Adults With Inherited Amyotrophic Lateral Sclerosis (ALS)


Brief description of study

The primary objective of Parts A and B of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of BIIB067 (Tofersen) in adult with ALS. The primary objective of Part C of this study is to evaluate the clinical efficacy of BIIB067 administered to adult participants with ALS and confirmed superoxide dismutase 1 (SOD1) mutation.

The secondary objective of Parts A and B of this study is to evaluate the effects of BIIB067 on levels of SOD1 protein in the cerebrospinal fluid (CSF). The secondary objectives of Part C are to evaluate the safety, tolerability, and pharmacodynamic (PD) effects of BIIB067 administered to adult participants with ALS and confirmed SOD1 mutation.

Detailed Study Description

This is a 3-part study to examine the efficacy, safety, tolerability, PK and PD of BIIB067. Part A is the single ascending dose (SAD) component of the study, Part B is the multiple ascending dose (MAD) component of the study and Part C is the fixed dose component of the study. Hence, the overall phase of development of the study is 1/2/3.

Parts A and B were completed on 15-Jan-2019. In total, the study is estimated to enroll 183 participants, with 99 in Part C.

Clinical Study Identifier: NCT02623699

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Bioclinica Research

Orlando, FL United States
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Emory University Hospital

Atlanta, GA United States
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Johns Hopkins University

Baltimore, MD United States
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UZ Leuven

Leuven, Belgium
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Shinjuku-ku, Japan
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Kagoshima City, Japan
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Sheffield, United Kingdom
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California Pacific Medical Center

San Francisco, CA United States
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Mayo Clinic in Florida

Jacksonville, FL United States
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Mayo Clinic - Rochester

Rochester, MN United States
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Neurology Associates, P.C.

Lincoln, NE United States
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Westmead, Australia
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Suita-Shi, Japan
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Bunkyo-Ku, Japan
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Westmead Hospital

Westmead, Australia
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UZ Leuven

Leuven, Belgium
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Providence ALS Center

Portland, OR United States
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