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

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ClinicalTrials.gov Identifier: NCT03945279
Recruitment Status : Recruiting
First Posted : May 10, 2019
Last Update Posted : September 13, 2019
Sponsor:
Information provided by (Responsible Party):
Biogen

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

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 40 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 : December 31, 2020
Estimated Study Completion Date : December 31, 2020


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.

Placebo Comparator: Cohort 1-5: Matching Placebo
Participants will receive single oral dose of matching placebo on Day 1.
Drug: Placebo
Administered as specified in the treatment arm.




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.



Information from the National Library of Medicine

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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.


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): NCT03945279


Contacts
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Contact: US Biogen Clinical Trial Center 866-633-4636 clinicaltrials@biogen.com
Contact: Global Biogen Clinical Trial Center clinicaltrials@biogen.com

Locations
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United States, Arizona
Barrow Neurological Institute Recruiting
Phoenix, Arizona, United States, 85013
Contact       fulton.research@dignityhealth.com   
United States, California
Research Site Not yet recruiting
Loma Linda, California, United States, 92354
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
Research Site Not yet recruiting
Jacksonville, Florida, United States, 32224
University of South Florida Recruiting
Tampa, Florida, United States, 33612
United States, Maryland
Research Site Not yet recruiting
Baltimore, Maryland, United States, 21224
United States, Massachusetts
Research Site Recruiting
Boston, Massachusetts, United States, 02129
United States, Missouri
Research Site Recruiting
Saint Louis, Missouri, United States, 63110
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
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Study Director: Medical Director Biogen

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Responsible Party: Biogen
ClinicalTrials.gov Identifier: NCT03945279     History of Changes
Other Study ID Numbers: 261AS101
First Posted: May 10, 2019    Key Record Dates
Last Update Posted: September 13, 2019
Last Verified: September 2019
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
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases