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)

Study Purpose:

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 Status:

Enrolling

Phase:

Phase III

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

Head of Medical Science, Mitsubishi Tanabe Pharma Development America, Inc.

Clinicaltrials.gov ID (11 digit #):

NCT04569084

Neals Affiliated?

No

Coordinating Center Contact Information

Mitsubishi Tanabe Pharma Development America, Inc.
Clinical Trials Information Desk / .(JavaScript must be enabled to view this email address) / 1-800-313-9381
.(JavaScript must be enabled to view this email address) United States

Full Study Summary:

Study Sponsor:

Mitsubishi Tanabe Pharma Development America, Inc.

Participant Duration:

Estimated Enrollment:

380

Estimated Study Start Date:

11/13/2020

Estimated Study Completion Date:

03/31/2022

Posting Last Modified Date:

12/13/2021

Date Study Added to alsconsortium.org:

12/15/2020
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    75

    Min Vital Capacity (% predicted normal):

    70

    Time since Symptom Onset:

    <24 months

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    1. 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.
    2. Subjects will be male or female, ≥ 18 to 75 years of age at the time the ICF is signed.
    3. Subjects will be diagnosed with Definite ALS or Probable ALS according to the El Escorial revised criteria for the diagnosis of ALS.
    4. Subjects with a baseline score ≥ 2 points on each individual item of the ALSFRS- R at screening and baseline visits.
    5. Subjects have a screening and baseline %forced vital capacity (FVC) ≥ 70%.
    6. Subjects with 1 to 4 points decline for 8 weeks in ALSFRS-R total score between screening and baseline visits.
    7. Subjects whose first symptom of ALS has occurred within 2 years of providing written informed consent.

    Exclusion Criteria:

    Exclusions Related to Primary Diagnosis

    1. 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)

    1. 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

    1. Subjects undergoing treatment for a malignancy.
    2. 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.
    3. 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.
    4. Subjects who are female and pregnant (a positive pregnancy test) or lactating at the screening visit (Visit 1).
    5. 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.
    6. 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.
    7. Subjects who have alanine aminotransferase (ALT) or aspartate aminotransferase (AST) elevations greater than 2 times the upper limit of normal (ULN) at screening.
    8. Subjects with a Glomerular Filtration Rate (GFR) < 30 mL/Min Per 1.73 m2 at screening, using the Larsson Equation.

    Exclusions Related to Medications

    1. Subjects with history of hypersensitivity to edaravone, any of the additives or inactive ingredients of edaravone, or sulfites.
    2. Subjects with hereditary problems of fructose intolerance (eg, fructose, sucrose, invert sugar, and sorbitol).
    3. 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.
    4. Subjects who have received any previous treatment with edaravone.
    5. Subjects who have received stem cell therapy.
    6. 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
    United States

    HonorHealth Neurology
    Scottsdale, Arizona 85251
    United States

    Woodland Research Northwest
    Rogers, Arkansas 72758
    United States

    UCSD Medical Center
    La Jolla, California 92037
    United States

    University California Los Angeles Medical Center (UCLA)
    Los Angeles, California 90095
    United States

    University of California Irvine (UCI)
    Orange, California 92868
    United States

    University of Colorado Anschutz Medical Campus
    Aurora, Colorado 80045
    United States

    UF Health Cancer Center
    Gainesville, Florida 32610
    United States

    Mayo Clinic
    Jacksonville, Florida 32224
    United States

    University of South Florida (USF)
    Tampa, Florida 33616
    United States

    Emory University
    Atlanta, Georgia 30322
    United States

    Northwestern University Feinberg School of Medicine
    Chicago, Illinois 60611
    United States

    Johns Hopkins University
    Baltimore, Maryland 21205
    United States

    Lahey Hospital
    Burlington, Massachusetts 01805
    United States

    Mayo Clinic
    Rochester, Minnesota 55905
    United States

    Neurology Associates, P.C.
    Lincoln, Nebraska 68506
    United States

    Las Vegas Clinic
    Las Vegas, Nevada 89145
    United States

    Dent Neurologic Institute
    Amherst, New York 14226
    United States

    SUNY Upstate Medical University
    Syracuse, New York 13210
    United States

    Lewis Katz School of Medicine at Temple University
    Philadelphia, Pennsylvania 19140
    United States

    University Of Pittsburgh Medical Center
    Pittsburgh, Pennsylvania 15261
    United States

    Wesley Neurology Clinic, P.C.
    Cordova, Tennessee 38018
    United States

    Austin Neuromuscular Center
    Austin, Texas 78756
    United States

    Nerve And Muscle Center Of Texas
    Houston, Texas 77019
    United States

    The University of Vermont (UVM)
    Burlington, Vermont 05401
    United States

    Sentara Neurology Specialists
    Virginia Beach, Virginia 23456
    United States

    University of Washington Medical Center
    Seattle, Washington 98195
    United States

    St. Luke's Rehabilitation Institute
    Spokane, Washington 99202
    United States

    West Virginia University School of Medicine
    Morgantown,, West Virginia 26506
    United States

    Medical College of Wisconsin
    Milwaukee, Wisconsin 53226
    United States

    University of Alberta
    Edmonton, Alberta
    Canada

    Regional Health Authority B
    Fredericton, New Brunswick
    Canada

    Health Science Center Mcmaster University
    Hamilton, Ontario
    Canada

    London Health Sciences Centre
    London, Ontario
    Canada

    Odette Cancer Center-Sunnybrook Health Sciences Centre
    Toronto, Ontario
    Canada

    Recherche Sepmus, Inc
    Greenfield Park, Quebec
    Canada

    Centre Hospitalier De L'Universite De Montreal (Chum) Notre-Dame Hospital
    Montreal, Quebec
    Canada

    Montreal Neurological Institute And Hospital
    Montreal, Quebec
    Canada

    CHU de Quebec-Hopital-Enfant-Jesus
    Quebec City, Quebec
    Canada

    Saskatoon City Hospital
    Saskatoon, Saskatchewan
    Canada

    Charite Campus Virchow
    Berlin,
    Germany

    Universit├Ątsklinik Bonn-Motoneuronambulanz
    Bonn,
    Germany

    Universitaetsklinikum Jena
    Jena,
    Germany

    University Medical Center Rostock
    Rostock,
    Germany

    Universitaets- und Rehabilitationskliniken Ulm
    Ulm,
    Germany

    Deutsche Klinik fuer Diagnostik
    Wiesbaden,
    Germany

    Universitaetsklinikum Wuerzburg
    Wuezburg,
    Germany

    Medizinische Hochschule Hannover
    Hannover, Lower Saxony
    Germany

    UKRUB
    Bochum, Nordrhein-Westfalen
    Germany

    Fondazione Serena Onlus
    Milan,
    Italy

    Istituto Nazionale Neurologico Carlo Besta
    Milano,
    Italy

    Ospedale San Raffaele (HSR)
    Milano,
    Italy

    Istituto Auxologico Italiano
    Modena,
    Italy

    Universita degli Studi di Torino
    Turin, Piemonte
    Italy

    Fukushima Medical University Hospital
    Fukushima-shi, Fukushima
    Japan

    National Hospital Organization Hokkaido Medical Center
    Sapporo-shi, Hokkaido
    Japan

    National Hospital Organization Iou National Hospital
    Kanazawa-shi, Ishikawa
    Japan

    National Hospital Organization Kumamoto Saishun Medical Center
    Koshi-shi, Kumamoto
    Japan

    Seoul National University Hospital
    Seoul,
    Republic of Korea

    Zentrumsleiter Muskelzentrum
    St.Gallen,
    Switzerland