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:

Study Status:

Not recruiting



Study Type:


Type of Intervention:


Intervention Name:

MT-1186, Placebo




Phase 3

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

Head of Medical Science, Mitsubishi Tanabe Pharma America Inc.

Clinicaltrials.gov ID:


Neals Affiliated?


Coordinating Center Contact Information

Mitsubishi Tanabe Pharma Development America, Inc.

United States

Full Study Summary:


- North America (USA English, USA Spanish, and Canadian French) +1-800-313-9381; mailbox address is MT1186@iconplc.com

- Japan: 0120-330-800 (Japan only)

Study Sponsor:

Mitsubishi Tanabe Pharma America Inc.

Estimated Enrollment:


Estimated Study Start Date:

11 / 13 / 2020

Estimated Study Completion Date:

07 / 01 / 2023

Posting Last Modified Date:

05 / 15 / 2023

Date Study Added to neals.org:

09 / 29 / 2020

Minimum Age:

18 Years

Maximum Age:

75 Years

Can participants use Riluzole?


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-point decline for 8 weeks (±7 days) 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)

2. 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

3. Subjects undergoing treatment for a malignancy.

4. 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.

5. 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.

6. Subjects who are female, of childbearing potential, and pregnant (a positive pregnancy test) or lactating at the screening visit (Visit 1).

7. 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.

8. 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.

9. Subjects who have alanine aminotransferase (ALT) or aspartate aminotransferase (AST) elevations greater than 2 times the upper limit of normal (ULN) at screening.

10. Subjects with a Glomerular Filtration Rate (GFR) < 30 mL/Min Per 1.73 m2 at screening, using the Larsson Equation.

Exclusions Related to Medications

11. Subjects with history of hypersensitivity to edaravone, any of the additives or inactive ingredients of edaravone, or sulfites.

12. Subjects with hereditary problems of fructose intolerance (eg, fructose, sucrose, invert sugar, and sorbitol).

13. 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.

14. Subjects who have received any previous treatment with edaravone.

15. Subjects who have received stem cell therapy.

16. Subjects who are unable to take their medications orally at baseline (Visit 2).

HonorHealth Neurology

Scottsdale, Arizona 85251
United States

St. Joseph's Hospital and Medical Center (SJHMC)

Phoenix, Arizona 85013
United States

Woodland Research Northwest

Rogers, Arkansas 72758
United States

UCSD Medical Center

La Jolla, California 92037-0897
United States

Loma Linda University Health Care - Department of Neurology

Loma Linda, California 92354
United States

University California Los Angeles Medical Center (UCLA)

Los Angeles, California 90095
United States

University of California Irvine (UCI) Health - Women's Healthcare Center

Orange, California 92868
United States

University of Colorado Anschutz Medical Campus

Aurora, Colorado 80045
United States

University of South Florida (USF) - Carol and Frank Morsani Center for Advanced Health Care (CAHC)

Tampa, Florida 33616
United States

UF Health Cancer Center

Gainesville, Florida 32610-3633
United States

Mayo Clinic

Jacksonville, Florida 32224
United States

Emory University - School of Medicine

Atlanta, Georgia 30322
United States

Northwestern University Feinberg School of Medicine

Chicago, Illinois 60611-2605
United States

Ochsner Center for Primary Care and Wellness

Jefferson, Louisiana 70121
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

Lincoln, Nebraska 68506-2960
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

Penn State Hershey Medical Center

Hershey, Pennsylvania 17033
United States

Thomas Jefferson University, Jefferson Weinberg ALS Center

Philadelphia, Pennsylvania 19107
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) and UVM Medical Center National ALS Center of Excellence

Burlington, Vermont 05401-3456
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 (WVUSoM) - Movement Disorder Clinic

Morgantown, West Virginia 26506-9180
United States

Medical College of Wisconsin

Milwaukee, Wisconsin 53226
United States

University of Alberta - Walter C Mackenzie Health Sciences Centre (WCM)

Edmonton, Alberta T6G 2B7

Health Science Center Mcmaster University

Hamilton, Ontario L8P 1H1

London Health Sciences Centre - University Hospital

London, Ontario N6A 5A5

Odette Cancer Center-Sunnybrook Health Sciences Centre

Toronto, Ontario M4N 3M5

Recherche Sepmus, Inc

Greenfield Park, Quebec J4V 2J2

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

Montreal, Quebec H2L 4M1

Montreal Neurological Institute And Hospital

Montreal, Quebec H3A 2B4

CHU de Quebec-Hopital-Enfant-Jesus

Quebec City, Quebec G1J 1Z4

Saskatoon City Hospital

Saskatoon, Saskatchewan S7K 0M7

Regional Health Authority B

Fredericton, New Brunswick E3B 0C7

Klinikum Rechts der Isar der Technischen Universitaet Muenchen

Muenchen 81675

Deutsche Klinik fuer Diagnostik

Wiesbaden 65191

Universitaets- und Rehabilitationskliniken Ulm

Ulm 89081

University Medical Center Rostock

Rostock 18147

Universitaetsklinikum Wuerzburg

Wuezburg 97080

Universitaetsklinikum Jena

Jena 86899

Universitätsklinik Bonn-Motoneuronambulanz, Klinik und Poliklinik für Neurodegenerative Erkrankungen und Gerontopsychiatrie

Bonn 53127

Charite Campus Virchow

Berlin 13353

UKRUB - Berufsgenossenschaftliches Universitatsklinikum Bergmannsheil GmbH - Medizinische Klinik III

Bochum, Nordrhein-Westfalen 44789

Medizinische Hochschule Hannover

Hannover, Lower Saxony 30625

Georg-August-Universitaet Goettingen - Universitaetsmedizin Goettingen (UMG)

Goettingen 37075

Istituto Auxologico Italiano - Istituto Di Ricovero e Cura a Carattere Scientifico - Istituto Scientifico Ospedale San Luca

Modena 41126

Centro SLA di Palermo

Palermo 90129

Policlinico A. Gemelli

Roma CAP 00168

Fondazione Serena Onlus - Azienda Ospedaliera Niguarda Ca Granda - Centro Clinico Nemo (Neuro Muscular Omnicentre)

Milan Italy

Istituto Nazionale Neurologico Carlo Besta

Milano Italy

Ospedale San Raffaele (HSR) (Istituto Scientifico Universitario San Raffaele)

Milano 20132

Universita degli Studi di Torino - Centro Regionale Esperto Per La Sclerosi Laterale Amiotrofica (CRESLA)

Turin, Piemonte 10126

Chiba University Hospital

Chiba 260-8677

Keio University Hospital

Shinjuku-ku, Tokyo 160-8582

Niigata University Medical & Dental Hospital

Asahimachidori, Chuo-ku, Niigata-shi, Niigata 951-8520

Toho University Omori Medical Center

Ota-ku, Tokyo 143-8541

Teikyo University Hospital

Itabashi-ku, Tokyo 173-8606

Tokyo Metropolitan Neurological Hospital

Fuchu-city, Tokyo 183-0042

Juntendo University Hospital

Bunkyo-ku, Tokyo 113-8431

National Hospital Organization Shizuoka Institute of Epilepsy and Neurological Disorders

Aoi-ku, Shizuoka-shi, Shizuoka 420-8688

Shiga University of Medical Science Hospital

Otsu City, Shiga 520-2192

Saitama Neuropsychiatric Institute

Chuo-ku, Saitama-shi, Saitama 338-8577

National Hospital Organization Osaka Toneyama Medical Center

Toyonaka-shi, Osaka 560-8552

Kansai Electric Power Hospital

Fukushima-ku, Osaka-shi, Osaka 553-0003

National Hospital Organization Chibahigashi National Hospital

Chuo-ku, Chiba-shi, Chiba 260-8712

Tohoku University Hospital

Sendai-city, Miyagi 980-8574

Hiroshima University Hospital

Minami-ku, Hiroshima-shi, Hiroshima 734-8551

National Hospital Organization Higashinagoya National Hospital

Meito-ku, Nagoya-shi, Aichi 465-8620

Nagoya University Hospital

Showa-ku, Nagoya, Aichi 466-8560

National Hospital Organization Utano National Hospital

Ukyo-ku, Kyoto City, Kyoto 616-8255

Fukushima Medical University Hospital

Fukushima-shi, Fukushima 960-1295

Murakami Karindoh Hospital

Nishi-ku, Fukuoka-shi, Fukuoka 819-8585

National Hospital Organization Hokkaido Medical Center

Sapporo-shi, Hokkaido 063-0005

National Hospital Organization Iou National Hospital

Kanazawa-shi, Ishikawa 920-0192

Kagawa University Hospital

Miki-cho, Kita-gun, Kagawa 761-0793

Yokohama City University Hospital

Kanazawa-ku, Yokohama-shi, Kanagawa 236-0004

Kitasato University Hospital

Minami-ku, Sagamihara-city, Kanagawa 252-0375

National Hospital Organization Kumamoto Saishun Medical Center

Koshi-shi, Kumamoto 861-1196

Seoul National University Hospital

Seoul 03080
Korea, Republic of

Hanyang University Medical Center

Seoul 04763
Korea, Republic of

Samsung Medical Center

Seoul 06351
Korea, Republic of

University hospital Bern (Inselspital)

Bern, BE 3010

Hopitaux Universitaires de Geneve (HUG) (Hopital Cantonal)

Geneve 1205

Neurocenter of Southern Switzerland

Lugano 6903

Zentrumsleiter Muskelzentrum/ALS Clinic Kantonsspital St.Gallen Muskelzentrum/ALS Clinic

St.Gallen 9107