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)

Amyotrophic Lateral Sclerosis Clinical Trial

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT04288856
• Other study ID #: 245AS102
• Secondary ID: 2019-004798-14
• Status: Terminated
• Phase: Phase 1
• Start date: April 28, 2020
• Est. completion date: May 3, 2022

Study information

• Verified date: April 2023
• Source: Biogen
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Description:

This study is an extension study of NCT03626012.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 75
• Est. completion date: May 3, 2022
• Est. primary completion date: May 3, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Amyotrophic Lateral Sclerosis
• Disease Progression
• Motor Neuron Disease
• Sclerosis

Intervention

Drug:
• BIIB078
Administered as specified in the treatment arm.

Locations

Country	Name	City	State
Canada	Research Site	Calgary	Alberta
Canada	Research Site	Edmonton	Alberta
Canada	Research Site	Montreal	Quebec
Canada	Research Site	Toronto	Ontario
Netherlands	Research Site	Utrecht
Switzerland	Research Site	St. Gallen
United Kingdom	Research Site	London	Greater London
United Kingdom	Research Site	London	Greater London
United Kingdom	Research Site	Sheffield	South Yorkshire
United States	Research Site	Atlanta	Georgia
United States	Research Site	Baltimore	Maryland
United States	Research Site	Boston	Massachusetts
United States	Research Site	Jacksonville	Florida
United States	Research Site	Knoxville	Tennessee
United States	Research Site	La Jolla	California
United States	Research Site	Lincoln	Nebraska
United States	Research Site	Los Angeles	California
United States	Research Site	Miami	Florida
United States	Research Site	New York	New York
United States	Research Site	Palo Alto	California
United States	Research Site	Saint Louis	Missouri

Sponsors (1)

Lead Sponsor	Collaborator
Biogen

Countries where clinical trial is conducted

United States,  Canada,  Netherlands,  Switzerland,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	Baseline up to Day 785
Primary	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.	Screening up to Day 785
Secondary	Serum concentration of BIIB078		Baseline and at multiple time points up to Day 729
Secondary	Cerebrospinal Fluid (CSF) concentration of BIIB078		Baseline and at multiple time points up to Day 729