Amyotrophic Lateral Sclerosis
Conditions
Brief summary
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.
Detailed description
This study is an extension study of NCT03626012.
Interventions
DRUGBIIB078
Administered as specified in the treatment arm.
Sponsors
Biogen
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Masking description
During the blinded loading period, the following individuals will be masked: * Investigator * Study staff * Participants After the loading period has been completed, subsequent doses will be open-label.
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: * Participants must have completed study NCT03626012 through the first follow-up clinic visit that follows the final dosing visit without missing more than 1 dose of study treatment. * Participants taking concomitant riluzole at study entry must be on a stable dose for ≥30 days prior to the first dose of study treatment (Day 1). Participants taking concomitant riluzole must be willing to continue with the same dose regimen throughout the study, unless the Investigator determines that riluzole should be discontinued for medical reasons, in which case it may not be restarted during the study. * Participants taking concomitant edaravone at study entry must be on a stable dose for ≥60 days prior to the first dose of study treatment (Day 1). Participants taking concomitant edaravone must be willing to continue with the same dose regimen throughout the study, 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. Key
Exclusion criteria
* History of drug abuse or alcoholism ≤6 months before study enrollment that would limit participation in the study, as determined by the Investigator. * Presence of an implanted shunt for the drainage of CSF or an implanted central nervous system (CNS) catheter. * History of or positive test result at Screening for human immunodeficiency virus. The requirement for testing at Screening may be omitted if it is not permitted by local regulations. * Treatment with another investigational drug (including investigational drugs for ALS through compassionate use programs) or biological agent within 1 month of Screening or 5 half-lives of study agent, whichever is longer. Note: Other protocol-specific inclusion/
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants with Adverse Events (AEs) | Baseline up to Day 785 | 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. |
| Number of Participants with Serious Adverse Events (SAEs) | Screening up to Day 785 | 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. |
Secondary
| Measure | Time frame |
|---|---|
| Serum concentration of BIIB078 | Baseline and at multiple time points up to Day 729 |
| Cerebrospinal Fluid (CSF) concentration of BIIB078 | Baseline and at multiple time points up to Day 729 |
Countries
Canada, Netherlands, Switzerland, United Kingdom, United States
Outcome results
None listed