A Phase 3b, Multicenter, Randomized, Double-Blind Study to Evaluate Efficacy and Safety of Oral Edaravone Administered for a Period of 48 Weeks in Subjects With Amyotrophic Lateral Sclerosis (ALS)
To evaluate and compare the efficacy of two dosing regimens of oral edaravone in subjects with amyotrophic lateral sclerosis (ALS) based on the change in ALS Functional Rating Scale- Revised (ALSFRS-R) score from baseline up to Week 48.
Disease:Amyotrophic Lateral Sclerosis (ALS), Familial ALS, Sporadic ALS
Study Type:Interventional Trial
Study Category:Drug Trial
Study Chair(s)/Principal Investigator(s):
Head of Medical Science, Mitsubishi Tanabe Pharma Development America, Inc.
Clinicaltrials.gov ID (11 digit #):NCT04569084
Coordinating Center Contact InformationMitsubishi Tanabe Pharma Development America, Inc.
Full Study Summary:
Study Sponsor:Mitsubishi Tanabe Pharma Development America, Inc.
Estimated Study Start Date:11/13/2020
Estimated Study Completion Date:03/31/2022
Posting Last Modified Date:02/17/2022
Date Study Added to alsconsortium.org:12/15/2020
Time since Symptom Onset:<24 months
Time since Diagnosis:
Can participants use Riluzole?Yes
- Subjects must provide a signed and dated informed consent form (ICF) to participate in the study. Subjects must be able (in the judgment of the Investigator) to understand the nature of the study and all risks involved with participation in the study. Subjects must be willing to cooperate and comply with all protocol restrictions and requirements.
- Subjects will be male or female, ≥ 18 to 75 years of age at the time the ICF is signed.
- Subjects will be diagnosed with Definite ALS or Probable ALS according to the El Escorial revised criteria for the diagnosis of ALS.
- Subjects with a baseline score ≥ 2 points on each individual item of the ALSFRS- R at screening and baseline visits.
- Subjects have a screening and baseline %forced vital capacity (FVC) ≥ 70%.
- Subjects with 1 to 4 points decline for 8 weeks in ALSFRS-R total score between screening and baseline visits.
- Subjects whose first symptom of ALS has occurred within 2 years of providing written informed consent.
Exclusions Related to Primary Diagnosis
- Subjects with a history of spinal surgery after the onset of ALS, such as surgery for cervical spondylosis or a herniated disc, or plans for such surgery during the study period.
Exclusions Related to Other Neurological Disorders (including, but not limited to the following)
- Subjects with the possibility that the current symptoms may be symptoms of a disease requiring differential diagnosis, such as cervical spondylosis and multifocal motor neuropathy, cannot be ruled out.
Exclusions Related to General Health or Concomitant Conditions
- Subjects undergoing treatment for a malignancy.
- Subjects with a complication that could have a significant effect on efficacy evaluations, such as Parkinson's disease or syndrome, schizophrenia, bipolar disorder, and dementia.
- Subjects who have the presence or history of any clinically significant (CS) disease (except ALS) that could interfere with the objectives of the study (the assessment of safety and efficacy) or the safety of the subject, as judged by the Investigator.
- Subjects who are female and pregnant (a positive pregnancy test) or lactating at the screening visit (Visit 1).
- Subjects of childbearing potential unwilling to use acceptable method of contraception from the screening visit until 3 months after the last dose of study medication. Subjects who are sexually active who do not agree to use contraception during the study period.
- Subjects who have a significant risk of suicidality. Subjects with any suicidal behavior or suicidal ideation of type 4 (active suicidal ideation with some intent to act, without a specific plan) or type 5 (active suicidal ideation with specific plan and intent) based on the Columbia-Suicide Severity Rating Scale (C-SSRS) within the 3 months before the screening visit.
- Subjects who have alanine aminotransferase (ALT) or aspartate aminotransferase (AST) elevations greater than 2 times the upper limit of normal (ULN) at screening.
- Subjects with a Glomerular Filtration Rate (GFR) < 30 mL/Min Per 1.73 m2 at screening, using the Larsson Equation.
Exclusions Related to Medications
- Subjects with history of hypersensitivity to edaravone, any of the additives or inactive ingredients of edaravone, or sulfites.
- Subjects with hereditary problems of fructose intolerance (eg, fructose, sucrose, invert sugar, and sorbitol).
- Subjects who participated in another study and were administered an investigational product within 1 month or 5 half-lives of the investigational agent, whichever is longer, before providing informed consent for the present study.
- Subjects who have received any previous treatment with edaravone.
- Subjects who have received stem cell therapy.
- Subjects who are unable to take their medications orally at baseline (Visit 2).
Site Contact Information
St. Joseph's Hospital and Medical Center (SJHMC)
Phoenix, Arizona 85013
Scottsdale, Arizona 85251
Woodland Research Northwest
Rogers, Arkansas 72758
UCSD Medical Center
La Jolla, California 92037
University California Los Angeles Medical Center (UCLA)
Los Angeles, California 90095
University of California Irvine (UCI)
Orange, California 92868
University of Colorado Anschutz Medical Campus
Aurora, Colorado 80045
UF Health Cancer Center
Gainesville, Florida 32610
Jacksonville, Florida 32224
University of South Florida (USF)
Tampa, Florida 33616
Atlanta, Georgia 30322
Northwestern University Feinberg School of Medicine
Chicago, Illinois 60611
Johns Hopkins University
Baltimore, Maryland 21205
Burlington, Massachusetts 01805
Rochester, Minnesota 55905
Neurology Associates, P.C.
Lincoln, Nebraska 68506
Las Vegas Clinic
Las Vegas, Nevada 89145
Dent Neurologic Institute
Amherst, New York 14226
SUNY Upstate Medical University
Syracuse, New York 13210
Lewis Katz School of Medicine at Temple University
Philadelphia, Pennsylvania 19140
Thomas Jefferson University, Jefferson Weinberg ALS Center
Philadelphia, Pennsylvania 19107
University Of Pittsburgh Medical Center
Pittsburgh, Pennsylvania 15261
Wesley Neurology Clinic, P.C.
Cordova, Tennessee 38018
Austin Neuromuscular Center
Austin, Texas 78756
Nerve And Muscle Center Of Texas
Houston, Texas 77019
The University of Vermont (UVM)
Burlington, Vermont 05401
Sentara Neurology Specialists
Virginia Beach, Virginia 23456
University of Washington Medical Center
Seattle, Washington 98195
St. Luke's Rehabilitation Institute
Spokane, Washington 99202
West Virginia University School of Medicine
Morgantown,, West Virginia 26506
Medical College of Wisconsin
Milwaukee, Wisconsin 53226
University of Alberta
Regional Health Authority B
Fredericton, New Brunswick
Health Science Center Mcmaster University
London Health Sciences Centre
Odette Cancer Center-Sunnybrook Health Sciences Centre
Recherche Sepmus, Inc
Greenfield Park, Quebec
Centre Hospitalier De L'Universite De Montreal (Chum) Notre-Dame Hospital
Montreal Neurological Institute And Hospital
CHU de Quebec-Hopital-Enfant-Jesus
Quebec City, Quebec
Saskatoon City Hospital
Charite Campus Virchow
University Medical Center Rostock
Universitaets- und Rehabilitationskliniken Ulm
Deutsche Klinik fuer Diagnostik
Medizinische Hochschule Hannover
Hannover, Lower Saxony
Fondazione Serena Onlus
Istituto Nazionale Neurologico Carlo Besta
Ospedale San Raffaele (HSR)
Istituto Auxologico Italiano
Universita degli Studi di Torino
Fukushima Medical University Hospital
National Hospital Organization Hokkaido Medical Center
National Hospital Organization Iou National Hospital
National Hospital Organization Kumamoto Saishun Medical Center
Seoul National University Hospital
Republic of Korea