A Study to Evaluate the Long-Term Effectives on ALS Disease Progression of BIIB067


About this study

The primary objective of the study is to evaluate the long-term safety and tolerability of BIIB067 in participants with amyotrophic lateral sclerosis (ALS) and confirmed superoxide dismutase 1 (SOD1) mutation. The secondary objectives are to evaluate the pharmacokinetic (PK) and pharmacodynamic (PD) profiles and effects on disease progression of BIIB067 administered to participants with ALS and confirmed SOD1 mutation.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

Inclusion Criteria:

  • Must have diagnosis of SOD1-ALS, and must have completed the End of Study Visit for either Parts A, B, or C of Study 233AS101 (NCT02623699) (i.e., were not withdrawn).
  • If taking riluzole, must be receiving a stable dose for ≥ 30 days prior to Day 1. 
  • For participants of childbearing potential must agree to practice effective contraception during the study and be willing and able to continue contraception 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. 
  • Participants from Study 233AS101 Parts A and B must have a washout ≥ 16 weeks between the last dose of study treatment received in Study 233AS101 and the first dose of BIIB067 received in the current Study 233AS102.
  • If taking edaravone, participant must have initiated edaravone ≥ 60 days (2 treatment cycles) prior to Day 1.
    • Edaravone may not be administered on dosing days during this study.

Exclusion Criteria: 

  • 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. 
  • Current hepatitis B infection (defined as positive for hepatitis B surface antigen [HBsAg] and/or hepatitis B core antibody [HBcAb]). Participants with immunity to hepatitis B from previous natural infection (defined as negative HBsAg, positive hepatitis B surface antibody immunoglobulin G, and positive HBcAb) or vaccination (defined as positive anti-HBs) are eligible to participate in the study. 
  • Presence of an implanted intravenous port/catheter.
    • NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status Contact

Rochester, Minn.

Mayo Clinic principal investigator

Eric Sorenson, M.D.

Closed for enrollment

Contact information:

Megan Means M.B.A.

(507) 293-0545


Jacksonville, Fla.

Mayo Clinic principal investigator

Bjorn Oskarsson, M.D.

Closed for enrollment

Contact information:

Amelia Robertson CCRP



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