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
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 Chair(s)/Principal Investigator(s):
Clinicaltrials.gov ID (11 digit #):NCT03945279
Coordinating Center Contact Information
Full Study Summary:
Estimated Study Start Date:05/30/2019
Estimated Study Completion Date:03/31/2021
Posting Last Modified Date:01/07/2021
Date Study Added to alsconsortium.org:05/22/2019
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
University of California San Diego
San Diego, California 92121
Mayo Clinic Hospital
Jacksonville, Florida 32224
University of South Florida
Tampa, Florida 33620
Johns Hopkins Hospital
Baltimore, Maryland 21224
Massachusetts General Hospital
Boston, Massachusetts 02114
Saint Louis, Missouri 63110
Lincoln, Nebraska 68506
Alliance for Multispecialty Research NOCCR/VRG
865-305-9100 ext 302 or 303
Knoxville, Tennessee 37920