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Quick Info
Status
Ongoing, But Not Recruiting
Estimated Enrollment
90
Phase
1
Treatment Type
Drug: BIIB078
Trial Type
Randomized, Sequential assignment, quadruple masking
Sponsor
Primary Investigator
Medical Director, Biogen
Contact Information
Locations
Canada, Quebec
Montreal, QC, H3A 2B4, Canada
Netherlands, Other
Netherlands, Utrecht , Netherlands
United Kingdom, Other
United Kingdom Research Site, London, United Kingdom
Research site, Sheffield, United Kingdom
United States, California
Research Site, La Jolla, CA, 92037, United States
Los Angeles , CA, 90048, United States
Palo Alto, CA, 94304, United States
United States, Florida
Jacksonville, FL, 32224, United States
Miami, FL, 33136, United States
United States, Georgia
Atlanta, GA, 30322, United States
United States, Maryland
Baltimore, MD, 21287, United States
United States, Massachusetts
Boston, MA, 02114, United States
United States, Missouri
Saint Louis, MO, 63110, United States
United States, Nebraska
Lincoln, NE, 68506, United States
United States, New York
New York, NY, 10032, United States
United States, Tennessee
Knoxville, TN, 37920, United States
Enrollment Criteria
Breathing Ability
Percent lung function (FVC) or (SVC)
(SVC) ≥ 50%
Months Since Onset
Number of months since first symptoms of ALS.
not specified
Non-Invasive Ventilation (NIV)
Can PALS use a BiPAP in the trial?
no
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
Update Notes
No significant updates
5/5/2020
No significant updates.
3/14/2020
No significant updates
12/2/2019
Locations added
10/25/2019
Updated anticipated trial completion date
10/4/2019
No significant updates
9/24/2019
Added New Site Location
9/12/2019
No significant updates
6/5/2019
Recruitment status updated
5/20/2019
Locations updated
3/20/2019
Locations updated
3/20/2019
Locations updated
3/4/2019
Locations and recruitment status updated
11/15/2018
Locations updated
10/26/2018
Recruitment status updated
9/17/2018
Trial added
8/20/2018

Other Information

Purpose
The primary objective of this study is to evaluate the safety and tolerability of BIIB078 in adults with C9ORF72-ALS. The secondary objective of this study is to evaluate the pharmacokinetic profile of BIIB078. As the first-in-human study, the study enrolls a small number of patients in each cohort. Every patient in a cohort is treated with the same dose or placebo. The study is designed to evaluate and confirm the safety of each dose before enrolling and exposing new patients to a higher dose in the next cohort. Therefore, proceeding from cohort to cohort, the recruitment status of "Active, not recruiting" may change. Please contact clinicaltrials@biogen.com for further information
Eligibility
18 Years and older, all genders. Not accepting healthy volunteers
Details
Collaborator(s)
    -
Trial Protocol as Published on Clinicaltrials.gov
NCT03626012 (First Published: 8/10/2018)