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Quick Info
Status
Ongoing, But Not Recruiting
Phase
3
Trial Type
Non-Randomized, Parallel Assignment, No masking
Treatment Type
Drug: BIIB067
Randomization
Open label trial
Enrollment
138
Start Date
3/8/2017
Sponsor
Contact Information
Locations
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)
N/A
Months Since Onset
Number of months since first symptoms of ALS.
N/A
Non-Invasive Ventilation (NIV)
Can PALS use a BiPAP in the trial?
N/A
Diaphragm Pacer (DPS)
Can PALS use a DPS in the trial?
No
Edaravone Usage
Can a PALS use edaravone (Radicut/Radicava) while enrolled in the trial?
Yes
Open Label
Yes
Update Notes
Completion date, protocol updates
9/13/2021
No longer recruiting
8/4/2021
New sites added
1/26/2021
New sites added
11/3/2020
No significant update.
6/15/2020
More research sites added.
6/4/2020
Enrolling by invitation.
3/27/2020
No Significant Updates
12/19/2019
No significant updates.
7/24/2019
Location, protocol, and enrollment criteria updated.
3/27/2019
No significant updates
8/13/2018
No significant updates
4/9/2018
No significant updates
1/10/2018
No significant updates
10/16/2017
No significant updates
7/17/2017
Contact info updated
5/23/2017
Enrolling by invitation
4/18/2017
Enrolling by invitation
4/18/2017
Locations updated
4/18/2017
Trial Added
3/6/2017

Other Information

Purpose
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.
Eligibility
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
Details
Collaborator(s)
Trial Protocol as Published on Clinicaltrials.gov
NCT03070119 (First Published: 2/28/2017)