Study to Assess the Safety, Tolerability, Pharmacokinetics, and Effect on Disease Progression of BIIB078 Administered to Previously Treated Adults C9ORF72-Associated Amyotrophic Lateral Sclerosis (ALS)
NCT ID: NCT04288856
Last Updated: 2023-04-18
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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TERMINATED
PHASE1
75 participants
INTERVENTIONAL
2020-04-28
2022-05-03
Brief Summary
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The secondary objective is to evaluate the pharmacokinectic (PK) of BIIB078 in participants with C9ORF72-ALS.
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Detailed Description
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Conditions
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Study Design
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NON_RANDOMIZED
PARALLEL
TREATMENT
NONE
* Investigator
* Study staff
* Participants
After the loading period has been completed, subsequent doses will be open-label.
Study Groups
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Cohort A: BIIB078 First Dosage
BIIB078 will be administered as 3 doses during the loading period, approximately 2 weeks apart, and maintenance doses, approximately 4 weeks apart, via IT infusion.
BIIB078
Administered as specified in the treatment arm.
Cohort B: BIIB078 Second Dosage
BIIB078 will be administered as 3 doses during the loading period, approximately 2 weeks apart, and maintenance doses, approximately 4 weeks apart, via IT infusion.
BIIB078
Administered as specified in the treatment arm.
Cohort C: BIIB078 Third Dosage
BIIB078 will be administered as 3 doses during the loading period, approximately 2 weeks apart, and maintenance doses, approximately 4 weeks apart, via IT infusion.
BIIB078
Administered as specified in the treatment arm.
Possible Cohort D: BIIB078 Fourth Dosage
BIIB078 will be administered as 3 doses during the loading period, approximately 2 weeks apart, and maintenance doses, approximately 4 weeks apart, via IT infusion.
BIIB078
Administered as specified in the treatment arm.
Interventions
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BIIB078
Administered as specified in the treatment arm.
Eligibility Criteria
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Inclusion Criteria
* 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.
Exclusion Criteria
* 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.
18 Years
ALL
No
Sponsors
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Biogen
INDUSTRY
Responsible Party
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Principal Investigators
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Medical Director
Role: STUDY_DIRECTOR
Biogen
Locations
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Research Site
La Jolla, California, United States
Research Site
Los Angeles, California, United States
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
Research Site
New York, New York, United States
Research Site
Knoxville, Tennessee, United States
Research Site
Calgary, Alberta, Canada
Research Site
Edmonton, Alberta, Canada
Research Site
Toronto, Ontario, Canada
Research Site
Montreal, Quebec, Canada
Research Site
Utrecht, , Netherlands
Research Site
Sankt Gallen, , Switzerland
Research Site
London, Greater London, United Kingdom
Research Site
London, Greater London, United Kingdom
Research Site
Sheffield, South Yorkshire, United Kingdom
Countries
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Other Identifiers
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2019-004798-14
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
245AS102
Identifier Type: -
Identifier Source: org_study_id
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