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 ALS

Study Purpose:

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.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Enrolling

Phase:

Phase I

Study Chair(s)/Principal Investigator(s):

Clinicaltrials.gov ID (11 digit #):

NCT03945279

Neals Affiliated?

No

Coordinating Center Contact Information


US Biogen Clinical Trial Center / .(JavaScript must be enabled to view this email address) / 866-633-4636
.(JavaScript must be enabled to view this email address)

Full Study Summary:

Study Sponsor:

Biogen

Participant Duration:

Estimated Enrollment:

40

Estimated Study Start Date:

06/19/2019

Estimated Study Completion Date:

12/31/2020

Posting Last Modified Date:

09/16/2019

Date Study Added to alsconsortium.org:

05/22/2019
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    N/A

    Min Vital Capacity (% predicted normal):

    65

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    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.

  • Site Contact Information

    Barrow Neurological Institute
    .(JavaScript must be enabled to view this email address) / Phoenix, Arizona 85013
    United States

    University of California San Diego
    Rosemarie Previte / .(JavaScript must be enabled to view this email address) / 858-246-1319
    San Diego, California 92121
    United States

    Loma Linda University Medical Center
    Loma Linda, California 92354
    United States

    University of South Florida
    Tampa, Florida 33620
    United States

    Mayo Clinic Hospital
    Jacksonville, Florida 32224
    United States

    Johns Hopkins Hospital
    Baltimore, Maryland 21224
    United States

    Massachusetts General Hospital
    Boston, Massachusetts 02114
    United States

    Washington University
    Saint Louis, Missouri 63110
    United States

    New Orleans Center for Clinical Research
    865-305-9100 ext 302 or 303
    Knoxville, Tennessee 37920
    United States