Efficacy and Safety Study of Oral Edaravone Administered in Subjects With ALS

NCT04569084 | Terminated Phase 3 Results FDA Drug

• 3 other identifiers: MT-1186-A02jRCT20312003012019-004256-11
• Study Type: interventional
• Target: 384
• Locations: 7 countries
• Sites: 95

Brief Summary

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:

Conditions

ALS

Outcome Measures

Primary Outcomes (1)

• CAFS Score at Week 48

  CAFS ranks patients' clinical outcomes based on survival time and change in the ALSFRS-R score. To calculate a patient's CAFS, each patient is compared individually to all other patients in the study. The summary score for each patient is the sum of the comparisons ( 1, 0, 1) against all other patients. After that, patients' summary scores are ranked. The CAFS rank is 1-383 and a higher CAFS rank indicates a better outcome than does a lower CAFS. Since CAFS were calculated using the imputed ALSFRS-R scores with multiple imputation method, and the maximum varies for each simulation, 383 is the maximum possible value of full range for measure of dispersion.

  up to 48 Weeks

Secondary Outcomes (5)

• Change From Baseline in % Slow Vital Capacity (SVC) at Week 48

  up to 48 Weeks

• Change From Baseline in Amyotrophic Lateral Sclerosis Assessment Questionnaire (ALSAQ)40 at Week 48

  up to 48 Weeks

• Time to Death, Tracheostomy or Permanent Assisted Mechanical Ventilation (≥ 23 Hours/Day)

  up to 48 Weeks

• Time to Death or PAMV (≥ 23 Hours/Day)

  up to 48 Weeks

• Time to Death

  up to 48 Weeks

Study Arms (2)

MT-1186

EXPERIMENTAL

Drug: MT-1186

MT-1186 and Placebo

EXPERIMENTAL

Drug: MT-1186; Drug: Placebo

Interventions

MT-1186 DRUG

Oral edaravone

Also known as: Oral edaravone

MT-1186; MT-1186 and Placebo

Placebo DRUG

Oral

MT-1186 and Placebo

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• 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.
• 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.
• 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, of childbearing potential, 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.
• 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.

Sponsors & Collaborators

• Tanabe Pharma America, Inc.: lead

Study Sites (95)

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

Phoenix, Arizona, 85013, United States

Location

HonorHealth Neurology

Scottsdale, Arizona, 85251, United States

Location

Woodland Research Northwest

Rogers, Arkansas, 72758, United States

Location

UCSD Medical Center

La Jolla, California, 92037-0897, United States

Location

Loma Linda University Health Care - Department of Neurology

Loma Linda, California, 92354, United States

Location

University California Los Angeles Medical Center (UCLA)

Los Angeles, California, 90095, United States

Location

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

Orange, California, 92868, United States

Location

University of Colorado Anschutz Medical Campus

Aurora, Colorado, 80045, United States

Location

UF Health Cancer Center

Gainesville, Florida, 32610-3633, United States

Location

Mayo Clinic

Jacksonville, Florida, 32224, United States

Location

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

Tampa, Florida, 33616, United States

Location

Emory University - School of Medicine

Atlanta, Georgia, 30322, United States

Location

Northwestern University Feinberg School of Medicine

Chicago, Illinois, 60611-2605, United States

Location

Ochsner Center for Primary Care and Wellness

Jefferson, Louisiana, 70121, United States

Location

Johns Hopkins University

Baltimore, Maryland, 21205, United States

Location

Lahey Hospital

Burlington, Massachusetts, 01805, United States

Location

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

Neurology Associates, P.C. - Lincoln

Lincoln, Nebraska, 68506-2960, United States

Location

Las Vegas Clinic

Las Vegas, Nevada, 89145, United States

Location

Dent Neurologic Institute

Amherst, New York, 14226, United States

Location

SUNY Upstate Medical University

Syracuse, New York, 13210, United States

Location

Penn State Hershey Medical Center

Hershey, Pennsylvania, 17033, United States

Location

Thomas Jefferson University, Jefferson Weinberg ALS Center

Philadelphia, Pennsylvania, 19107, United States

Location

Lewis Katz School of Medicine at Temple University

Philadelphia, Pennsylvania, 19140, United States

Location

University Of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, 15261, United States

Location

Wesley Neurology Clinic, P.C.

Cordova, Tennessee, 38018, United States

Location

Austin Neuromuscular Center

Austin, Texas, 78756, United States

Location

Nerve And Muscle Center Of Texas

Houston, Texas, 77019, United States

Location

The University of Vermont (UVM) and UVM Medical Center National ALS Center of Excellence

Burlington, Vermont, 05401-3456, United States

Location

Sentara Neurology Specialists

Virginia Beach, Virginia, 23456, United States

Location

University of Washington Medical Center

Seattle, Washington, 98195, United States

Location

St. Luke's Rehabilitation Institute

Spokane, Washington, 99202, United States

Location

West Virginia University School of Medicine (WVUSoM) - Movement Disorder Clinic

Morgantown, West Virginia, 26506-9180, United States

Location

Medical College of Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

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

Edmonton, Alberta, T6G 2B7, Canada

Location

Regional Health Authority B

Fredericton, New Brunswick, E3B 0C7, Canada

Location

Health Science Center Mcmaster University

Hamilton, Ontario, L8P 1H1, Canada

Location

London Health Sciences Centre - University Hospital

London, Ontario, N6A 5A5, Canada

Location

Odette Cancer Center-Sunnybrook Health Sciences Centre

Toronto, Ontario, M4N 3M5, Canada

Location

Recherche Sepmus, Inc

Greenfield Park, Quebec, J4V 2J2, Canada

Location

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

Montreal, Quebec, H2L 4M1, Canada

Location

Montreal Neurological Institute And Hospital

Montreal, Quebec, H3A 2B4, Canada

Location

CHU de Quebec-Hopital-Enfant-Jesus

Québec, Quebec, G1J 1Z4, Canada

Location

Saskatoon City Hospital

Saskatoon, Saskatchewan, S7K 0M7, Canada

Location

Universitaetsklinikum Wuerzburg

Wuezburg, Germany, 97080, Germany

Location

Medizinische Hochschule Hannover

Hanover, Lower Saxony, 30625, Germany

Location

UKRUB - Berufsgenossenschaftliches Universitatsklinikum Bergmannsheil GmbH - Medizinische Klinik III

Bochum, North Rhine-Westphalia, 44789, Germany

Location

Charite Campus Virchow

Berlin, 13353, Germany

Location

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

Bonn, 53127, Germany

Location

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

Göttingen, 37075, Germany

Location

Universitaetsklinikum Jena

Jena, 86899, Germany

Location

Klinikum Rechts der Isar der Technischen Universitaet Muenchen

München, 81675, Germany

Location

University Medical Center Rostock

Rostock, 18147, Germany

Location

Universitaets- und Rehabilitationskliniken Ulm

Ulm, 89081, Germany

Location

Deutsche Klinik fuer Diagnostik

Wiesbaden, 65191, Germany

Location

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

Turin, Piedmont, 10126, Italy

Location

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

Milan, Italy, Italy

Location

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

Milan, 20132, Italy

Location

Istituto Nazionale Neurologico Carlo Besta

Milan, Italy, Italy

Location

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

Modena, 41126, Italy

Location

Centro SLA di Palermo

Palermo, 90129, Italy

Location

Policlinico A. Gemelli

Roma, CAP 00168, Italy

Location

National Hospital Organization Higashinagoya National Hospital

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

Location

Nagoya University Hospital

Showa-ku, Nagoya, Aichi-ken, 466-8560, Japan

Location

National Hospital Organization Chibahigashi National Hospital

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

Location

Murakami Karindoh Hospital

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

Location

Fukushima Medical University Hospital

Fukushima, Fukushima, 960-1295, Japan

Location

Hiroshima University Hospital

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

Location

National Hospital Organization Hokkaido Medical Center

Sapporo, Hokkaido, 063-0005, Japan

Location

National Hospital Organization Iou National Hospital

Kanazawa, Ishikawa-ken, 920-0192, Japan

Location

Kagawa University Hospital

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

Location

Yokohama City University Hospital

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

Location

Kitasato University Hospital

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

Location

National Hospital Organization Kumamoto Saishun Medical Center

Koshi-shi, Kumamoto, 861-1196, Japan

Location

National Hospital Organization Utano National Hospital

Ukyo-ku, Kyoto City, Kyoto, 616-8255, Japan

Location

Tohoku University Hospital

Sendai, Miyagi, 980-8574, Japan

Location

Niigata University Medical & Dental Hospital

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

Location

Kansai Electric Power Hospital

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

Location

National Hospital Organization Osaka Toneyama Medical Center

Toyonaka-shi, Osaka, 560-8552, Japan

Location

Saitama Neuropsychiatric Institute

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

Location

Shiga University of Medical Science Hospital

Ōtsu, Shiga, 520-2192, Japan

Location

National Hospital Organization Shizuoka Institute of Epilepsy and Neurological Disorders

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

Location

Juntendo University Hospital

Bunkyo-ku, Tokyo, 113-8431, Japan

Location

Tokyo Metropolitan Neurological Hospital

Fuchū, Tokyo, 183-0042, Japan

Location

Teikyo University Hospital

Itabashi-ku, Tokyo, 173-8606, Japan

Location

Toho University Omori Medical Center

Ōta-ku, Tokyo, 143-8541, Japan

Location

Keio University Hospital

Shinjuku-ku, Tokyo, 160-8582, Japan

Location

Chiba University Hospital

Chiba, 260-8677, Japan

Location

Seoul National University Hospital

Seoul, 03080, South Korea

Location

Hanyang University Medical Center

Seoul, 04763, South Korea

Location

Samsung Medical Center

Seoul, 06351, South Korea

Location

University hospital Bern (Inselspital)

Bern, Canton of Bern, 3010, Switzerland

Location

Hopitaux Universitaires de Geneve (HUG) (Hopital Cantonal)

Geneva, 1205, Switzerland

Location

Neurocenter of Southern Switzerland

Lugano, 6903, Switzerland

Location

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

Sankt Gallen, 9107, Switzerland

Location

Related Publications (1)

• Rothstein J, Genge A, De Silva S, Zinman L, Chum M, Chio A, Sobue G, Aoki M, Yoshino H, Doyu M, Selness D, Todorovic V, Sasson N, Hirai M, Takahashi F, Salah A, Wamil A, Apple S. Efficacy and Safety of Once Daily Dosing vs. Approved On/Off Dosing of Edaravone Oral Suspension Up to 48 Weeks in Patients With Amyotrophic Lateral Sclerosis (Study MT-1186-A02). Muscle Nerve. 2025 Sep;72(3):433-442. doi: 10.1002/mus.28448. Epub 2025 Jun 6.

  PMID: 40474686 RESULT

MeSH Terms

Interventions

Edaravone

Intervention Hierarchy (Ancestors)

Antipyrine; Pyrazolones; Pyrazoles; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Results Point of Contact

• Title: Clinical Trials, Information Desk
• Organization: Tanabe Pharma America, Inc.

information.US@mb.tanabe-pharma.com

908-607-1980

Study Officials

• Head of Medical Science

  Tanabe Pharma America, Inc.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 23, 2020
• First Posted: September 29, 2020
• Study Start: November 13, 2020
• Primary Completion: September 29, 2023
• Study Completion: September 29, 2023
• Last Updated: May 22, 2026
• Results First Posted: March 27, 2025

Record last verified: 2026-05

Locations

IT (7); JP (26); CA (10); KR (3); CH (4); DE (11); US (34)