Back to resultsA Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BIIB100 Administered Orally to Adults With Amyotrophic Lateral Sclerosis
Primary Purpose
Amyotrophic Lateral Sclerosis
Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
BIIB100
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Amyotrophic Lateral Sclerosis
Eligibility Criteria
Key Inclusion Criteria:
- Must meet the laboratory-supported probable, probable, or definite criteria for diagnosing ALS according to the World Federation of Neurology El Escorial criteria.
- Participants taking concomitant riluzole at study entry must be on a stable dose for greater than or equals to (>=) 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).
- Adequate respiratory function as indicated by slow vital capacity (SVC) >= 65% of predicted value as adjusted for sex, age, and height (from the sitting position).
Key Exclusion Criteria:
- Ongoing medical condition (e.g., wasting or cachexia, severe anemia) that would, in the opinion of the Investigator, interfere with the conduct or assessments of the study.
- Significant cognitive impairment or unstable psychiatric illness, including psychosis, suicidal ideation, suicide attempt, or untreated major depression less than or equals to (<=) 90 days of Screening, which in the opinion of the Investigator would interfere with the study procedures.
- Treatment with drugs that are transported by Breast Cancer Resistance Protein (BCRP) and P-glycoprotein (P-gp) including, but not limited to, rosuvastatin, sulfasalazine, dabigatran, digoxin and fexofenadine.
- Current enrollment or plan to enroll in any interventional clinical study in which an investigational treatment or approved therapy for investigational use is administered within 30 days or 5 half-lives of the agent, whichever is longer, prior to the Baseline Visit (pre-dose on Day 1). Participation in a noninterventional study focused on ALS natural history may be allowed at the discretion of the Investigator and after consultation with the Sponsor.
Note: Other protocol defined Inclusion/Exclusion criteria may apply.
Sites / Locations
- Barrow Neurological Institute
- University of California San Diego Medical Center
- Mayo Clinic Hospital
- University of South Florida
- Johns Hopkins University, Dept of Neurology
- Research Site
- Research Site
- Research Site
- Alliance for Multispecialty Research NOCCR/VRG
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Arm 5
Arm 6
Arm 7
Arm Type
Experimental
Experimental
Experimental
Experimental
Experimental
Experimental
Placebo Comparator
Arm Label
Cohort 1: BIIB100 Dose 1
Cohort 2: BIIB100 Dose 2
Cohort 3: BIIB100 Dose 3
Cohort 4: BIIB100 Dose 4
Cohort 5: BIIB100 Dose 5
Cohort 6: BIIB100 Dose 6
Cohort 1-6: Matching Placebo
Arm Description
Participants will receive single oral dose of BIIB100 on Day 1.
Participants will receive single oral dose of BIIB100 on Day 1.
Participants will receive single oral dose of BIIB100 on Day 1.
Participants will receive single oral dose of BIIB100 on Day 1.
Participants will receive single oral dose of BIIB100 on Day 1.
Participants will receive single oral dose of BIIB100 on Day 1.
Participants will receive single oral dose of matching placebo on Day 1.
Outcomes
Primary Outcome Measures
Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs)
An AE is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. An SAE is any untoward medical occurrence that at any dose results in death, is a life-threatening event, requires inpatient hospitalization or prolongation of existing hospitalization, results in a significant disability/incapacity or congenital anomaly, or is a medically important event.
Secondary Outcome Measures
Area Under the Concentration-Time Curve From Time 0 to Time of the Last Measurable Concentration (AUClast) of BIIB100
BIIB100 will be measured in the plasma.
Area Under the Concentration-Time Curve From Time 0 to Infinity (AUCinf) of BIIB100
BIIB100 will be measured in the plasma.
Maximum Observed Concentration (Cmax) of BIIB100
BIIB100 will be measured in the plasma.
Time to Reach Cmax (Tmax) of BIIB100
BIIB100 will be measured in the plasma.
Terminal Elimination Half-life (t1/2) of BIIB100
BIIB100 will be measured in the plasma.
Apparent Clearance (CL/F) of BIIB100
BIIB100 will be measured in the plasma.
Apparent Volume of Distribution During the Terminal Elimination (Vz/F) of BIIB100
BIIB100 will be measured in the plasma.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT03945279
Brief Title
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BIIB100 Administered Orally to Adults With Amyotrophic Lateral Sclerosis
Official Title
A Phase 1, Double-Blind, Placebo-Controlled, Single-Ascending-Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BIIB100 Administered Orally to Adult Participants With Amyotrophic Lateral Sclerosis
Study Type
Interventional
2. Study Status
Record Verification Date
April 2023
Overall Recruitment Status
Completed
Study Start Date
May 30, 2019 (Actual)
Primary Completion Date
June 21, 2021 (Actual)
Study Completion Date
June 21, 2021 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Biogen
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
The primary objective of this study is to evaluate the safety, tolerability of single-ascending doses of BIIB100 in adults with amyotrophic lateral sclerosis (ALS). The secondary objective of the study is to characterize the pharmacokinetic profile of BIIB100.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Amyotrophic Lateral Sclerosis
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Sequential Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
49 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Cohort 1: BIIB100 Dose 1
Arm Type
Experimental
Arm Description
Participants will receive single oral dose of BIIB100 on Day 1.
Arm Title
Cohort 2: BIIB100 Dose 2
Arm Type
Experimental
Arm Description
Participants will receive single oral dose of BIIB100 on Day 1.
Arm Title
Cohort 3: BIIB100 Dose 3
Arm Type
Experimental
Arm Description
Participants will receive single oral dose of BIIB100 on Day 1.
Arm Title
Cohort 4: BIIB100 Dose 4
Arm Type
Experimental
Arm Description
Participants will receive single oral dose of BIIB100 on Day 1.
Arm Title
Cohort 5: BIIB100 Dose 5
Arm Type
Experimental
Arm Description
Participants will receive single oral dose of BIIB100 on Day 1.
Arm Title
Cohort 6: BIIB100 Dose 6
Arm Type
Experimental
Arm Description
Participants will receive single oral dose of BIIB100 on Day 1.
Arm Title
Cohort 1-6: Matching Placebo
Arm Type
Placebo Comparator
Arm Description
Participants will receive single oral dose of matching placebo on Day 1.
Intervention Type
Drug
Intervention Name(s)
BIIB100
Intervention Description
Administered as specified in the treatment arm.
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Administered as specified in the treatment arm.
Primary Outcome Measure Information:
Title
Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs)
Description
An AE is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. An SAE is any untoward medical occurrence that at any dose results in death, is a life-threatening event, requires inpatient hospitalization or prolongation of existing hospitalization, results in a significant disability/incapacity or congenital anomaly, or is a medically important event.
Time Frame
Screening (Day -28 ) up to Day 15
Secondary Outcome Measure Information:
Title
Area Under the Concentration-Time Curve From Time 0 to Time of the Last Measurable Concentration (AUClast) of BIIB100
Description
BIIB100 will be measured in the plasma.
Time Frame
Day 1 (pre-dose) up to Day 3
Title
Area Under the Concentration-Time Curve From Time 0 to Infinity (AUCinf) of BIIB100
Description
BIIB100 will be measured in the plasma.
Time Frame
Day 1 (pre-dose) up to Day 3
Title
Maximum Observed Concentration (Cmax) of BIIB100
Description
BIIB100 will be measured in the plasma.
Time Frame
Day 1 (pre-dose) up to Day 3
Title
Time to Reach Cmax (Tmax) of BIIB100
Description
BIIB100 will be measured in the plasma.
Time Frame
Day 1 (pre-dose) up to Day 3
Title
Terminal Elimination Half-life (t1/2) of BIIB100
Description
BIIB100 will be measured in the plasma.
Time Frame
Day 1 (pre-dose) up to Day 3
Title
Apparent Clearance (CL/F) of BIIB100
Description
BIIB100 will be measured in the plasma.
Time Frame
Day 1 (pre-dose) up to Day 3
Title
Apparent Volume of Distribution During the Terminal Elimination (Vz/F) of BIIB100
Description
BIIB100 will be measured in the plasma.
Time Frame
Day 1 (pre-dose) up to Day 3
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Key Inclusion Criteria:
Must meet the laboratory-supported probable, probable, or definite criteria for diagnosing ALS according to the World Federation of Neurology El Escorial criteria.
Participants taking concomitant riluzole at study entry must be on a stable dose for greater than or equals to (>=) 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).
Adequate respiratory function as indicated by slow vital capacity (SVC) >= 65% of predicted value as adjusted for sex, age, and height (from the sitting position).
Key Exclusion Criteria:
Ongoing medical condition (e.g., wasting or cachexia, severe anemia) that would, in the opinion of the Investigator, interfere with the conduct or assessments of the study.
Significant cognitive impairment or unstable psychiatric illness, including psychosis, suicidal ideation, suicide attempt, or untreated major depression less than or equals to (<=) 90 days of Screening, which in the opinion of the Investigator would interfere with the study procedures.
Treatment with drugs that are transported by Breast Cancer Resistance Protein (BCRP) and P-glycoprotein (P-gp) including, but not limited to, rosuvastatin, sulfasalazine, dabigatran, digoxin and fexofenadine.
Current enrollment or plan to enroll in any interventional clinical study in which an investigational treatment or approved therapy for investigational use is administered within 30 days or 5 half-lives of the agent, whichever is longer, prior to the Baseline Visit (pre-dose on Day 1). Participation in a noninterventional study focused on ALS natural history may be allowed at the discretion of the Investigator and after consultation with the Sponsor.
Note: Other protocol defined Inclusion/Exclusion criteria may apply.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Director
Organizational Affiliation
Biogen
Official's Role
Study Director
Facility Information:
Facility Name
Barrow Neurological Institute
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85013
Country
United States
Facility Name
University of California San Diego Medical Center
City
San Diego
State/Province
California
ZIP/Postal Code
92121
Country
United States
Facility Name
Mayo Clinic Hospital
City
Jacksonville
State/Province
Florida
ZIP/Postal Code
32224
Country
United States
Facility Name
University of South Florida
City
Tampa
State/Province
Florida
ZIP/Postal Code
33612
Country
United States
Facility Name
Johns Hopkins University, Dept of Neurology
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21205
Country
United States
Facility Name
Research Site
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
21219
Country
United States
Facility Name
Research Site
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
Research Site
City
Lincoln
State/Province
Nebraska
ZIP/Postal Code
68506
Country
United States
Facility Name
Alliance for Multispecialty Research NOCCR/VRG
City
Knoxville
State/Province
Tennessee
ZIP/Postal Code
37920
Country
United States
12. IPD Sharing Statement
Plan to Share IPD
Yes
IPD Sharing Plan Description
In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/
IPD Sharing URL
https://vivli.org/
Learn more about this trial
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BIIB100 Administered Orally to Adults With Amyotrophic Lateral Sclerosis
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