Clinical trial NCT04288856: 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)
Official title: An Extension Study to Assess the Long-Term Safety, Tolerability, Pharmacokinetics, and Effect on Disease Progression of BIIB078 Administered to Previously Treated Adults With C9ORF72-Associated Amyotrophic Lateral Sclerosis
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE1
Conditions: Amyotrophic Lateral Sclerosis
Lead sponsor: Biogen
Sponsor class: INDUSTRY
First posted: 2020-02-28
Enrollment: 75
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility 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/exclusion criteria may apply.

Primary outcomes:
measure: Number of Participants with Adverse Events (AEs)
timeFrame: Baseline up to Day 785
measure: Number of Participants with Serious Adverse Events (SAEs)
timeFrame: Screening up to Day 785
Secondary outcomes:
measure: Serum concentration of BIIB078
timeFrame: Baseline and at multiple time points up to Day 729
measure: Cerebrospinal Fluid (CSF) concentration of BIIB078
timeFrame: Baseline and at multiple time points up to Day 729
Other outcomes:
Locations:
city: La Jolla
country: United States
facility: Research Site
state: California
city: Los Angeles
country: United States
facility: Research Site
state: California
city: Palo Alto
country: United States
facility: Research Site
state: California
city: Jacksonville
country: United States
facility: Research Site
state: Florida
city: Miami
country: United States
facility: Research Site
state: Florida
city: Atlanta
country: United States
facility: Research Site
state: Georgia
city: Baltimore
country: United States
facility: Research Site
state: Maryland
city: Boston
country: United States
facility: Research Site
state: Massachusetts
city: Saint Louis
country: United States
facility: Research Site
state: Missouri
city: Lincoln
country: United States
facility: Research Site
state: Nebraska
city: New York
country: United States
facility: Research Site
state: New York
city: Knoxville
country: United States
facility: Research Site
state: Tennessee
city: Calgary
country: Canada
facility: Research Site
state: Alberta
city: Edmonton
country: Canada
facility: Research Site
state: Alberta
city: Toronto
country: Canada
facility: Research Site
state: Ontario
city: Montreal
country: Canada
facility: Research Site
state: Quebec
city: Utrecht
country: Netherlands
facility: Research Site
city: St. Gallen
country: Switzerland
facility: Research Site
city: London
country: United Kingdom
facility: Research Site
state: Greater London
city: London
country: United Kingdom
facility: Research Site
state: Greater London
city: Sheffield
country: United Kingdom
facility: Research Site
state: South Yorkshire
Study officials:
affiliation: Biogen
name: Medical Director
role: STUDY_DIRECTOR
References: