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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)

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ClinicalTrials.gov Identifier: NCT04288856
Recruitment Status : Enrolling by invitation
First Posted : February 28, 2020
Last Update Posted : September 29, 2020
Sponsor:
Information provided by (Responsible Party):
Biogen

Study Description
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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.


Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis Drug: BIIB078 Phase 1

Detailed Description:
This study is an extension study of NCT03626012.
Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 90 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : April 28, 2020
Estimated Primary Completion Date : July 19, 2023
Estimated Study Completion Date : July 19, 2023

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Arms and Interventions
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Arm Intervention/treatment
Experimental: 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.
 Drug: BIIB078
Administered as specified in the treatment arm.
Experimental: 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.
 Drug: BIIB078
Administered as specified in the treatment arm.
Experimental: 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.
 Drug: BIIB078
Administered as specified in the treatment arm.


Outcome Measures
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Primary Outcome Measures :
  1. Number of Participants with Adverse Events (AEs) [ Time Frame: 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.

  2. Number of Participants with Serious Adverse Events (SAEs) [ Time Frame: 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 Outcome Measures :
  1. Serum concentration of BIIB078 [ Time Frame: Baseline and at multiple time points up to Day 729 ]
  2. Cerebrospinal Fluid (CSF) concentration of BIIB078 [ Time Frame: Baseline and at multiple time points up to Day 729 ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04288856


Locations
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United States, California
Research Site
La Jolla, California, United States, 92037-0886
Research Site
Palo Alto, California, United States, 94304
United States, Florida
Research Site
Miami, Florida, United States, 33136
United States, Georgia
Research Site
Atlanta, Georgia, United States, 30322
United States, Maryland
Research Site
Baltimore, Maryland, United States, 21287
United States, Massachusetts
Research Site
Boston, Massachusetts, United States, 02114
United States, Missouri
Research Site
Saint Louis, Missouri, United States, 63110
United States, Nebraska
Research Site
Lincoln, Nebraska, United States, 68506-2960
United States, New York
Research Site
New York, New York, United States, 10032
United States, Tennessee
Research Site
Knoxville, Tennessee, United States, 37920
Canada, Quebec
Research Site
Montreal, Quebec, Canada, H3A 2B4
Switzerland
Research Site
St. Gallen, Switzerland, 9007
United Kingdom
Research Site
London, Greater London, United Kingdom, SE5 9RS
Sponsors and Collaborators
Biogen
Investigators
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Study Director: Medical Director Biogen
More Information
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Responsible Party: Biogen
ClinicalTrials.gov Identifier: NCT04288856    
Other Study ID Numbers: 245AS102
2019-004798-14 ( EudraCT Number )
First Posted: February 28, 2020    Key Record Dates
Last Update Posted: September 29, 2020
Last Verified: September 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on http://clinicalresearch.biogen.com/
URL: http://www.biogenclinicaldatarequest.com/

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Sclerosis
Disease Progression
Pathologic Processes
Disease Attributes
Neurodegenerative Diseases
 Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases