Your support fuels our research to #EndALS! Donate Now
Quick Info
Ongoing, But Not Recruiting
Trial Type
Non-Randomized, Parallel Assignment, No masking
Treatment Type
Drug: BIIB067
Open label trial
Start Date
Contact Information
Belgium, Other
Research Site, Leuven, 3000, Belgium
Canada, Alberta
Research Site, Calgary, AB, T2N 4Z6, Canada
Research Site, Edmonton, AB, T6G 2B7, Canada
Canada, Ontario
Research Site, Toronto, ON, M4N 3M5, Canada
Canada, Quebec
Research Site, Montreal, QC, H3A2B4, Canada
France, Other
Research Site, Clermont-Ferrand cedex, 63003, France
Germany, Other
Research Site, Ulm, 89081, Germany
Italy, Other
Research Site, Torino, 10126, Italy
Japan, Other
Research Site, Bunkyo-ku, Japan
Research Site, Kagoshima-shi, Japan
Research Site, Shinjuku-ku, Japan
Research Site, Suita-shi, Japan
New Zealand, Other
Research Site, Christchurch, 8011, New Zealand
United Kingdom, Other
Research Site, Sheffield, S102HQ, United Kingdom
United States, Arizona
Research Site, Phoenix, AZ, 85013, United States
United States, California
Research Site, La Jolla, CA, 92093-0949, United States
Research Site, San Francisco, CA, 94118, United States
United States, Florida
Research Site, Jacksonville, FL, 32224, United States
Research Site, Miami, FL, 33136, United States
Research Site, Orlando, FL, 32806, United States
United States, Georgia
Research Site, Atlanta, GA, 30322, United States
United States, Illinois
Research Site, Chicago, IL, 60611, United States
United States, Maryland
Research Site, Baltimore, MD, 21287, United States
United States, Massachusetts
Research Site, Boston, MA, 02114, United States
United States, Minnesota
Research Site, Rochester, MN, 55905, United States
United States, Missouri
Research Site, Saint Louis, MO, 63110, United States
United States, Nebraska
Research Site, Lincoln, NE, 68510, United States
United States, Ohio
Research Site, Cleveland, OH, 44106, United States
United States, Oregon
Research Site, Portland, OR, 97213, United States
United States, Tennessee
Research Site, Knoxville, TN, 37920, United States
Enrollment Criteria
Breathing Ability
Percent lung function (FVC) or (SVC)
Months Since Onset
Number of months since first symptoms of ALS.
Non-Invasive Ventilation (NIV)
Can PALS use a BiPAP in the trial?
Diaphragm Pacer (DPS)
Can PALS use a DPS in the trial?
Edaravone Usage
Can a PALS use edaravone (Radicut/Radicava) while enrolled in the trial?
Open Label
Update Notes
Completion date, protocol updates
No longer recruiting
New sites added
New sites added
No significant update.
More research sites added.
Enrolling by invitation.
No Significant Updates
No significant updates.
Location, protocol, and enrollment criteria updated.
No significant updates
No significant updates
No significant updates
No significant updates
No significant updates
Contact info updated
Enrolling by invitation
Enrolling by invitation
Locations updated
Trial Added

Other Information

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), pharmacodynamic (PD), biomarker effects, and efficacy of BIIB067 administered to participants with ALS and a confirmed SOD1 mutation.
Key Inclusion Criteria:
- Must have diagnosis of superoxide dismutase 1-amyotrophic lateral sclerosis (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, participant must be receiving a stable dose for ≥30 days prior to Day 1.
- 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.
- 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.
- For female 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.
- 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.
Key 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.
- 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.
- Female participants who are pregnant or currently breastfeeding.
- Current enrollment in any other interventional study.
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply
Trial Protocol as Published on
NCT03070119 (First Published: 2/28/2017)