The primary objective is to evaluate the long-term safety and tolerability of BIIB078 in participants with chromosome 9 open reading frame 72-amyotrophic lateral sclerosis (C9ORF72-ALS). The secondary objective is to evaluate the pharmacokinectic (PK) of BIIB078 in participants with C9ORF72-ALS.
This study is an extension study of NCT03626012.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
75
Administered as specified in the treatment arm.
Research Site
La Jolla, California, United States
Research Site
Los Angeles, California, United States
Number of Participants with Adverse Events (AEs)
AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of a study intervention, whether or not considered related to the study intervention.
Time frame: Baseline up to Day 785
Number of Participants with Serious Adverse Events (SAEs)
An SAE is any untoward medical occurrence that, at any dose results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent disability/incapacity, is a congenital anomaly/birth defect or any other situation according to medical or scientific judgment.
Time frame: Screening up to Day 785
Serum concentration of BIIB078
Time frame: Baseline and at multiple time points up to Day 729
Cerebrospinal Fluid (CSF) concentration of BIIB078
Time frame: Baseline and at multiple time points up to Day 729
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Research Site
Palo Alto, California, United States
Research Site
Jacksonville, Florida, United States
Research Site
Miami, Florida, United States
Research Site
Atlanta, Georgia, United States
Research Site
Baltimore, Maryland, United States
Research Site
Boston, Massachusetts, United States
Research Site
St Louis, Missouri, United States
Research Site
Lincoln, Nebraska, United States
...and 11 more locations