An Extension Study to Assess the Long-Term Safety, Tolerability, Pharmacokinetics, and Effect on Disease Progression of BIIB067 Administered to Previously Treated Adults With Amyotrophic Lateral Sclerosis Caused by Superoxide Dismutase 1 Mutation

Study Purpose:

The primary objective of the study is to evaluate the long-term safety and tolerability of BIIB067 in participants with Amyotrophic Lateral Sclerosis Caused by Superoxide Dismutase 1 Mutation (SOD1-ALS). The secondary objective is to evaluate the pharmacokinetic (PK) profile of BIIB067 in participants with SOD1-ALS.


Amyotrophic Lateral Sclerosis (ALS),  Familial ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Not enrolling


Phase I

Study Chair(s)/Principal Investigator(s): ID (11 digit #):


Neals Affiliated?


Coordinating Center Contact Information

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Full Study Summary:

Study Sponsor:


Participant Duration:

Estimated Enrollment:


Estimated Study Start Date:


Estimated Study Completion Date:


Posting Last Modified Date:


Date Study Added to

  • Eligibility Criteria


    Female, Male

    Minimum Age:


    Maximum Age:


    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?


    Key Inclusion Criteria:

    Must have diagnosis of SOD1-ALS, and must have completed Part A and/or Part B of Study 233AS101 (NCT02623699) (i.e., were not withdrawn and did not miss more than 1 dose of study drug).
    If taking riluzole, must be receiving a stable dose for ≥30 days prior to Day 1 and expected to remain at that dose until the final study visit.
    For participants of childbearing potential must agree to practice effective contraception during the study and for 5 months after their last dose of study treatment.
    Medically able to undergo the study procedures, and to adhere to the visit schedule at the time of study entry, as determined by the Investigator.

    Key Exclusion Criteria:

    History of or positive test result for human immunodeficiency virus.
    History of or positive test result for hepatitis C virus antibody or hepatitis B virus (defined as positive for both hepatitis B surface antigen and hepatitis B core antibody).
    History of allergies to a broad range of anesthetics.
    Presence of risk for increased or uncontrolled bleeding and/or risk of bleeding that is not managed optimally and could place a participant at an increased risk for bleeding during or after a Lumbar Puncture (LP) procedure. These risks could include, but are not limited to, anatomical factors at or near the LP site (e.g., vascular abnormalities, neoplasms, or other abnormalities) and underlying disorders of the coagulation cascade, platelet function, or platelet count (e.g., hemophilia, Von Willebrand's disease, liver disease).
    Presence of an implanted shunt for the drainage of CSF or an implanted central nervous system (CNS) catheter.
    Prior or current treatment with small interfering ribonucleic acid (RNA), stem cell therapy, or gene therapy.
    Treatment with another investigational drug, biological agent (excluding BIIB067), or device within 1 month or 5 half-lives of study agent, whichever is longer.
    Current or anticipated need, in the opinion of the Investigator, of a diaphragm pacing system (DPS) during the study period.
    Female participants who are pregnant or currently breastfeeding.
    Current enrollment in any other interventional study.
    Current or recent (within 1 month) use, or anticipated need, in the opinion of the Investigator, of copper (II) (diacetyl-bis(N4-methylthiosemicarbazone)) or pyrimethamine.

    NOTE: Other protocol defined Inclusion/Exclusion criteria may apply

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