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

Completed

• Conditions: Amyotrophic lateral sclerosis; neurodegenerative disease; 10029317

• Registration Number: NL-OMON49747
• Lead Sponsor: Biogen

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 15 April 2024

Recruitment & Eligibility

• Status: Completed
• Sex: Not specified
• Target Recruitment: 1

Inclusion Criteria

- Participants must have completed study 245AS101 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 mdiscontinued 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.
Note: Other protocol-specific inclusion criteria may apply.

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 exclusion criteria may apply.

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Incidence of AEs and serious adverse events (SAEs).</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>Serum and CSF BIIB078 concentration during the teatment (predose) and follow-up<br /><br>periods.</p><br>