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Quick Info
Status
Ongoing, But Not Recruiting
Phase
3
Trial Type
Double Blind, Placebo Controlled
Treatment Type
Single and Multiple Ascending Dose
Randomization
2:1
Enrollment
178
Start Date
1/20/2016
Sponsor
Contact Information
Locations
Australia, Other
Westmead Hospital, Westmead, 2145, Australia
Belgium, Other
UZ Leuven, Leuven, 3000, Belgium
Canada, Alberta
University of Calgary - Health Sciences Centre, Calgary, AB, T2N 1N4, Canada
Research Site, Edmonton, AB, T6G 2G3, Canada
Canada, Ontario
Sunnybrook Health Sciences Centre, Toronto, ON, M4N 3M5, Canada
Canada, Quebec
Montreal Neurological Institute, Montreal, QC, H3A 2B4, Canada
Denmark, Other
Bispebjerg Hospital, Copenhagen, 2400, Denmark
France, Other
Hopital Pitie Salpetriere, Paris, 75651, France
Germany, Other
University of Ulm, Ulm, 89081, Germany
Italy, Other
ALS Center - Dept. of Neuroscience "Rita Levi Montalcini", University of Turin, Torino, 10126, Italy
Japan, Other
The University of Tokyo Hospital, Bunkyo-Ku, Japan
Research Site, Fukuoka-shi, Japan
Research Site, Kagoshima City, Japan
Research Site, Shinjuku-ku, Japan
Research Site, Suita-Shi, Japan
Other, Other
Research Site, Seoul, 04763, Other
Research Site, Yangsan-si, 50612, Other
Poland, Other
Research Site, Warszawa, 01684, Poland
United Kingdom, Other
Research Site, London, SE5 9RS, United Kingdom
Research Site, Sheffield, S10 2HQ, United Kingdom
United States, Arizona
Barrow Neurological Institute, Phoenix, AZ, 85013, United States
United States, California
University of California San Diego Medical Center, La Jolla, CA, 92093-0949, United States
California Pacific Medical Center, San Francisco, CA, 94115, United States
United States, Florida
Mayo Clinic in Florida, Jacksonville, FL, 32224, United States
University of Miami School of Medicine, Miami, FL, 33136, United States
Bioclinica Research, Orlando, FL, 32806, United States
United States, Georgia
Emory University Hospital, Atlanta, GA, 30322, United States
United States, Illinois
Northwestern University Feinberg School of Medicine, Chicago, IL, 60611, United States
United States, Maryland
Johns Hopkins University, Baltimore, MD, 21287, United States
United States, Massachusetts
Massachusetts General Hospital, Boston, MA, 02114, United States
United States, Michigan
Henry Ford Hospital, Detroit, MI, 48202, United States
United States, Minnesota
Mayo Clinic - Rochester, Rochester, MN, 55905, United States
United States, Missouri
Washington University School of Medicine, Saint Louis, MO, 63110, United States
United States, Nebraska
Neurology Associates, P.C., Lincoln, NE, 68506, United States
United States, New York
Columbia University Medical Center, New York, NY, 10032, United States
United States, Ohio
The Cleveland Clinic Foundation, Cleveland, OH, 44106, United States
United States, Oregon
Providence ALS Center, Portland, OR, 97213, United States
United States, Pennsylvania
University of Pennsylvania, Philadelphia, PA, 19104, United States
United States, Tennessee
New Orleans Center for Clinical Research/Volunteer Research Group, an AMR Company, Knoxville, TN, 37920, United States
United States, Texas
Methodist Neurological Institute, Houston, TX, 77030, United States
Enrollment Criteria
Breathing Ability
Percent lung function (FVC) or (SVC)
≥50%
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?
Maybe
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
No significant updates
3/24/2021
New sites added
1/22/2021
Study no longer recruiting
1/20/2021
New site recruiting
11/23/2020
New locations added
10/8/2020
New site added
9/28/2020
No significant updates
9/9/2020
New locations added
8/19/2020
New location added in Denark
7/22/2020
Contact information updated.
6/15/2020
No significant updates.
5/22/2020
No significant updates.
5/14/2020
No significant updates.
3/5/2020
No significant updates.
2/28/2020
New recruitment sites added
2/7/2020
No significant update
1/21/2020
No significant updates
1/13/2020
No significant updates
1/7/2020
No significant updates
1/7/2020
No significant updates
1/7/2020
No significant updates
12/19/2019
Updated recruitment locations
12/13/2019
No significant updates
12/6/2019
No significant update
12/2/2019
Added multiple new location sites
11/19/2019
Updated location site
11/7/2019
Recruitment Status updated
9/6/2019
Updated Study Purpose/ Recruitment status updated. More cites are currently recruiting.
8/30/2019
Location added.
8/20/2019
Locations updated.
8/2/2019
No significant updates.
7/8/2019
No significant updates
7/1/2019
Recruitment status at locations updated
6/10/2019
No significant updates
5/31/2019
Location recruitment status updated
5/24/2019
Location recruitment status updated
5/6/2019
Location recruitment status updated
4/29/2019
Location recruitment status updated
4/22/2019
Location recruitment status updated
4/16/2019
Location recruitment status updated
4/8/2019
Location recruitment status updated
3/20/2019
Recruitment status updated
3/11/2019
Recruitment status updated
9/3/2018
No significant updates
5/31/2018
No significant updates
4/20/2018
No significant updates
3/16/2018
Recruitment status updated
2/15/2018
No significant updates
1/19/2018
No significant updates
12/5/2017
No significant updates
11/13/2017
No significant updates
10/5/2017
Recruitment status at location updated
9/15/2017
No significant updates
8/18/2017
No significant updates
7/12/2017
No significant updates
6/16/2017
No significant updates
5/1/2017
No significant updates
3/29/2017
Recruitment status updated
3/6/2017
No significant updates
3/6/2017
No significant changes
10/12/2016
Recruitment status updated
9/9/2016
Recruitment status updated
8/18/2016
Recruitment status updated
7/12/2016
No Significant Updates
6/8/2016
Sponsor/Collaborator updated.
4/11/2016
Recruitment status updated.
2/3/2016
Clinical trial added.
12/11/2015

Other Information

Purpose
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.
Eligibility
Key Inclusion Criteria: Part A and B - Weakness attributable to ALS and documented SOD1 mutation at Screening Visit 2.
- A forced vital capacity (FVC) ≥50% of predicted value as adjusted for sex, age, and height (from the sitting position). Participants with stable FVC <50% but ≥45%, whose FVC has not declined by more than 5% in the last 6 months may be considered for inclusion, at the discretion of the Investigator.
- If taking riluzole, participant must be on a stable dose for ≥30 days prior to Day 1 and expected to remain at that dose until the final study visit.
- 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: Part A and B - History of or positive test result for human immunodeficiency virus.
- History of, or positive test result at Screening, for hepatitis C virus antibody.
- 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.
- Treatment with another investigational drug, biological agent, or device within 1 month or 5 half-lives of study agent, whichever is longer. Specifically, no prior treatment with small interfering ribonucleic acid, stem cell therapy, or gene therapy is allowed.
- 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 or anticipated need, in the opinion of the Investigator, of a diaphragm pacing system (DPS) during the study period.
Key Inclusion Criteria: Part C - Weakness attributable to ALS and confirmed SOD1 mutation at Screening Visit.
- If taking riluzole, participant must be on a stable dose for ≥30 days prior to Day 1 and expected to remain at that dose until the final study visit.
- If taking edaravone, participant must have initiated edaravone ≥60 days (2 treatment cycles) prior to Day 1 and expected to remain at that dose until the final study visit, unless the Investigator determines that edaravone should be discontinued for medical reasons, in which case it may not be restarted during the study. Edaravone may not be administered on dosing days of 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.
Key Exclusion Criteria: Part C - History of or positive test result for human immunodeficiency virus.
- Current hepatitis C infection (defined as positive hepatitis C virus [HCV] antibody and detectable HCV ribonucleic acid [RNA]). Participants with positive HCV antibody and undetectable HCV RNA are eligible to participate in the study (United States Centers for Disease Control and Prevention).
- Current hepatitis B infection (defined as positive for HBsAg and/or anti-HBc).
participants with immunity to hepatitis B from previous natural infection (defined as negative HBsAg, positive anti-HBc, and positive anti-HBs) or vaccination (defined as negative HBsAg, negative anti-HBc, and positive anti-HBs) are eligible to participate in the study.
- Treatment with another investigational drug (including investigational drugs for ALS through compassionate use programs), biological agent, or device within 1 month or 5 half-lives of study agent, whichever is longer. Specifically, no prior treatment with small interfering RNA, stem cell therapy, or gene therapy is allowed.
- 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 or anticipated need, in the opinion of the Investigator, of a DPS during the study period.
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
Details
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.
Collaborator(s)
Trial Protocol as Published on Clinicaltrials.gov
NCT02623699 (First Published: 11/24/2015)