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出境医 / 临床实验 / A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BIIB100 Administered Orally to Adults With Amyotrophic Lateral Sclerosis

A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BIIB100 Administered Orally to Adults With Amyotrophic Lateral Sclerosis

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.

Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis Drug: BIIB100 Drug: Placebo Phase 1

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 48 participants
Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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
Actual Study Start Date : May 30, 2019
Estimated Primary Completion Date : June 14, 2021
Estimated Study Completion Date : June 14, 2021
Arms and Interventions
Arm Intervention/treatment
Experimental: Cohort 1: BIIB100 Dose 1
Participants will receive single oral dose of BIIB100 on Day 1.
 Drug: BIIB100
Administered as specified in the treatment arm.
Experimental: Cohort 2: BIIB100 Dose 2
Participants will receive single oral dose of BIIB100 on Day 1.
 Drug: BIIB100
Administered as specified in the treatment arm.
Experimental: Cohort 3: BIIB100 Dose 3
Participants will receive single oral dose of BIIB100 on Day 1.
 Drug: BIIB100
Administered as specified in the treatment arm.
Experimental: Cohort 4: BIIB100 Dose 4
Participants will receive single oral dose of BIIB100 on Day 1.
 Drug: BIIB100
Administered as specified in the treatment arm.
Experimental: Cohort 5: BIIB100 Dose 5
Participants will receive single oral dose of BIIB100 on Day 1.
 Drug: BIIB100
Administered as specified in the treatment arm.
Experimental: Cohort 6: BIIB100 Dose 6
Participants will receive single oral dose of BIIB100 on Day 1.
 Drug: BIIB100
Administered as specified in the treatment arm.
Placebo Comparator: Cohort 1-6: Matching Placebo
Participants will receive single oral dose of matching placebo on Day 1.
 Drug: Placebo
Administered as specified in the treatment arm.
Outcome Measures
Primary Outcome Measures :
  1. Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: Screening (Day -28 ) up to Day 15 ]
    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 :
  1. Area Under the Concentration-Time Curve From Time 0 to Time of the Last Measurable Concentration (AUClast) of BIIB100 [ Time Frame: Day 1 (pre-dose) up to Day 3 ]
    BIIB100 will be measured in the plasma.

  2. Area Under the Concentration-Time Curve From Time 0 to Infinity (AUCinf) of BIIB100 [ Time Frame: Day 1 (pre-dose) up to Day 3 ]
    BIIB100 will be measured in the plasma.

  3. Maximum Observed Concentration (Cmax) of BIIB100 [ Time Frame: Day 1 (pre-dose) up to Day 3 ]
    BIIB100 will be measured in the plasma.

  4. Time to Reach Cmax (Tmax) of BIIB100 [ Time Frame: Day 1 (pre-dose) up to Day 3 ]
    BIIB100 will be measured in the plasma.

  5. Terminal Elimination Half-life (t1/2) of BIIB100 [ Time Frame: Day 1 (pre-dose) up to Day 3 ]
    BIIB100 will be measured in the plasma.

  6. Apparent Clearance (CL/F) of BIIB100 [ Time Frame: Day 1 (pre-dose) up to Day 3 ]
    BIIB100 will be measured in the plasma.

  7. Apparent Volume of Distribution During the Terminal Elimination (Vz/F) of BIIB100 [ Time Frame: Day 1 (pre-dose) up to Day 3 ]
    BIIB100 will be measured in the plasma.


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:

  • 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.

Contacts and Locations

Contacts
Layout table for location contacts
Contact: US Biogen Clinical Trial Center 866-633-4636 clinicaltrials@biogen.com
Contact: Global Biogen Clinical Trial Center clinicaltrials@biogen.com

Locations
Layout table for location information
United States, Arizona
Barrow Neurological Institute Recruiting
Phoenix, Arizona, United States, 85013
Contact       Fulton.Research@DignityHealth.org   
United States, California
University of California San Diego Medical Center Recruiting
San Diego, California, United States, 92121
Contact: Rosemarie Previte    858-246-1319    rprevite@ucsd.edu   
United States, Florida
Mayo Clinic Hospital Recruiting
Jacksonville, Florida, United States, 32224
Contact    904-953-6912    mayofloridaALSresearch@mayo.edu   
University of South Florida Recruiting
Tampa, Florida, United States, 33612
United States, Maryland
Johns Hopkins University, Dept of Neurology Recruiting
Baltimore, Maryland, United States, 21205
Contact    410-502-0495    emosmil1@jhmi.edu   
United States, Massachusetts
Research Site Recruiting
Boston, Massachusetts, United States, 21219
United States, Missouri
Research Site Recruiting
Saint Louis, Missouri, United States, 63110
United States, Nebraska
Research Site Recruiting
Lincoln, Nebraska, United States, 68506-2960
United States, Tennessee
Alliance for Multispecialty Research NOCCR/VRG Recruiting
Knoxville, Tennessee, United States, 37920
Contact: Alliance for Multispeciality Research NOCCR/VRG    865-305-9100 ext 302 or 303      
Sponsors and Collaborators
Biogen
Investigators
Layout table for investigator information
Study Director: Medical Director Biogen
Tracking Information
First Submitted Date  ICMJE May 8, 2019
First Posted Date  ICMJE May 10, 2019
Last Update Posted Date March 22, 2021
Actual Study Start Date  ICMJE May 30, 2019
Estimated Primary Completion Date June 14, 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 8, 2019) 		Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: Screening (Day -28 ) up to Day 15 ]
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.
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: May 8, 2019)
  • Area Under the Concentration-Time Curve From Time 0 to Time of the Last Measurable Concentration (AUClast) of BIIB100 [ Time Frame: Day 1 (pre-dose) up to Day 3 ]
    BIIB100 will be measured in the plasma.
  • Area Under the Concentration-Time Curve From Time 0 to Infinity (AUCinf) of BIIB100 [ Time Frame: Day 1 (pre-dose) up to Day 3 ]
    BIIB100 will be measured in the plasma.
  • Maximum Observed Concentration (Cmax) of BIIB100 [ Time Frame: Day 1 (pre-dose) up to Day 3 ]
    BIIB100 will be measured in the plasma.
  • Time to Reach Cmax (Tmax) of BIIB100 [ Time Frame: Day 1 (pre-dose) up to Day 3 ]
    BIIB100 will be measured in the plasma.
  • Terminal Elimination Half-life (t1/2) of BIIB100 [ Time Frame: Day 1 (pre-dose) up to Day 3 ]
    BIIB100 will be measured in the plasma.
  • Apparent Clearance (CL/F) of BIIB100 [ Time Frame: Day 1 (pre-dose) up to Day 3 ]
    BIIB100 will be measured in the plasma.
  • Apparent Volume of Distribution During the Terminal Elimination (Vz/F) of BIIB100 [ Time Frame: Day 1 (pre-dose) up to Day 3 ]
    BIIB100 will be measured in the plasma.
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BIIB100 Administered Orally to Adults With Amyotrophic Lateral Sclerosis
Official Title  ICMJE 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
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Amyotrophic Lateral Sclerosis
Intervention  ICMJE
  • Drug: BIIB100
    Administered as specified in the treatment arm.
  • Drug: Placebo
    Administered as specified in the treatment arm.
Study Arms  ICMJE
  • Experimental: Cohort 1: BIIB100 Dose 1
    Participants will receive single oral dose of BIIB100 on Day 1.
    Intervention: Drug: BIIB100
  • Experimental: Cohort 2: BIIB100 Dose 2
    Participants will receive single oral dose of BIIB100 on Day 1.
    Intervention: Drug: BIIB100
  • Experimental: Cohort 3: BIIB100 Dose 3
    Participants will receive single oral dose of BIIB100 on Day 1.
    Intervention: Drug: BIIB100
  • Experimental: Cohort 4: BIIB100 Dose 4
    Participants will receive single oral dose of BIIB100 on Day 1.
    Intervention: Drug: BIIB100
  • Experimental: Cohort 5: BIIB100 Dose 5
    Participants will receive single oral dose of BIIB100 on Day 1.
    Intervention: Drug: BIIB100
  • Experimental: Cohort 6: BIIB100 Dose 6
    Participants will receive single oral dose of BIIB100 on Day 1.
    Intervention: Drug: BIIB100
  • Placebo Comparator: Cohort 1-6: Matching Placebo
    Participants will receive single oral dose of matching placebo on Day 1.
    Intervention: Drug: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: October 7, 2020) 		48
Original Estimated Enrollment  ICMJE
 (submitted: May 8, 2019) 		40
Estimated Study Completion Date  ICMJE June 14, 2021
Estimated Primary Completion Date June 14, 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: US Biogen Clinical Trial Center 866-633-4636 clinicaltrials@biogen.com
Contact: Global Biogen Clinical Trial Center clinicaltrials@biogen.com
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03945279
Other Study ID Numbers  ICMJE 261AS101
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
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: https://vivli.org/
Responsible Party Biogen
Study Sponsor  ICMJE Biogen
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Medical Director Biogen
PRS Account Biogen
Verification Date March 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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