Safety Study of Oral Edaravone Administered in Subjects With ALS

NCT04165824 | Completed Phase 3 Results FDA Drug

• 1 other identifier: MT-1186-A01
• Study Type: interventional
• Target: 185
• Locations: 6 countries
• Sites: 53

Brief Summary

The objective of this study is to evaluate the long-term safety and tolerability of oral edaravone in subjects with Amyotrophic Lateral Sclerosis (ALS) over 24 and 48 weeks.

Conditions

ALS

Outcome Measures

Primary Outcomes (2)

• Number of Treatment Emergency Adverse Events

  up to 48 Weeks

• Number of Participants With Treatment Emergency Adverse Events

  Up to 48 Weeks

Other Outcomes (2)

• Change in ALS Functional Rating Scale - Revised From Baseline

  up to 48 Weeks

• Time to Event (Death, Tracheostomy, and Permanent Assisted Mechanical Ventilation)

  up to 48 Weeks

Study Arms (1)

MT-1186

EXPERIMENTAL

Drug: MT-1186

Interventions

MT-1186 DRUG

* An initial treatment cycle with daily dosing for 14 days, followed by a 14-day drug free period * Subsequent treatment cycles with daily dosing for 10 days out of a 14-day period, followed by a 14 day drug free period. Treatment cycles are every 4 weeks.

Also known as: Oral edaravone

MT-1186

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 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, Probable ALS, Probable laboratory-supported ALS, or Possible ALS according to the El Escorial revised criteria for the diagnosis of ALS.
• Subjects will be living and functioning independently (eg, able to eat, excrete, ambulate independently without assistance of others). The use of supportive tools and adaptive utensil is allowed
• Subjects will have a baseline forced vital capacity percentage (%FVC) ≥ 70%.
• Subjects whose first symptom of ALS occurred within 3 years of the time of providing written informed consent.

You may not qualify if:

• Subjects who have the presence or history of any clinically significant 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 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 are female and pregnant (a positive pregnancy test) or lactating at the screening visit (Visit 1).
• 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 ALT or AST elevations greater than 2 times the ULN at screening.
• Subjects with a Glomerular Filtration Rate (GFR) \<30 mL/Min Per 1.73 m2.
• Subjects with history of hypersensitivity to edaravone, any of the additives or inactive ingredients of edaravone, or sulfites.
• Subjects with hereditary fructose intolerance.
• 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 are unable to take their medications orally.

Sponsors & Collaborators

• Tanabe Pharma America, Inc.: lead

Study Sites (53)

St. Joseph's Hospital and Medical Center (SJHMC) - Barrow Neurological Institute (BNI) - The Gregory W. Fulton ALS and Neuromuscular Disease Center

Phoenix, Arizona, 85013, United States

Location

Neuromuscular Research Center

Phoenix, Arizona, 85028, United States

Location

Woodland Research Northwest

Rogers, Arkansas, 72758, United States

Location

Sutter Health

San Francisco, California, 94115, 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

Emory University - School of Medicine

Atlanta, Georgia, 30317-2819, United States

Location

Johns Hopkins University

Baltimore, Maryland, 21205, United States

Location

University of Michigan - ALS Center of Excellence

Ann Arbor, Michigan, 48109-5223, United States

Location

University of Michigan Health System (UMHS) - Allergy Clinic - Northville Health Center Location

Northville, Michigan, 48168-9493, United States

Location

Essentia Institute of Rural Health

Duluth, Minnesota, 55805, United States

Location

Neurology Associates, P.C - Lincoln

Lincoln, Nebraska, 68506-2960, United States

Location

Wake Forest University Baptist Medical Center (WFUBMC) - The J. Paul Sticht Center on Aging and Rehabilitation

Winston-Salem, North Carolina, 27157-0001, United States

Location

Penn State Hershey Children's Hospital

Hershey, Pennsylvania, 17025, United States

Location

Lewis Katz School of Medicine at Temple University

Philadelphia, Pennsylvania, 19140, United States

Location

Alleghany General Hospital

Pittsburgh, Pennsylvania, 15212, United States

Location

Wesley Neurology Clinic, P.C.

Cordova, Tennessee, 38018, United States

Location

Texas Neurology, PA

Dallas, Texas, 75214, United States

Location

Houston Methodist Neurological Institute

Houston, Texas, 77030, United States

Location

UT Health Science Center San Antonio

San Antonio, Texas, 78229, United States

Location

University of Washington Medical Center

Seattle, Washington, 98195, United States

Location

Medical College of Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

Heritage Medical Research Clinic - University Of Calgary

Calgary, Alberta, T2N 4Z6, Canada

Location

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

Edmonton, Alberta, T6G 2B7, Canada

Location

Recherche Sepmus, Inc

Greenfield Park, Quebec, J4V 2J2, Canada

Location

Montreal Neurological Institute and Hospital

Montreal, Quebec, H3A 2B4, Canada

Location

CHU Nice-Hospital Archet I

Nice, Alpes Maritimes, 06202, France

Location

Centre Hospitalier Esquirol

Limoges, Marcland, 87025, France

Location

Centre Hospitalier Universitaire (CHU) de Bordeaux

Bordeaux, 33400, France

Location

Hopital Pierre Wertheimer - Hopital Neurologique

Bron, 69677, France

Location

CHU de NICE Hopital Pasteur 2 -Centre de Reference des Maladies -Neuromusculaires & SLA-Pole Neurosciences Cliniques

Nice, 6001, France

Location

Deutsche Klinik fuer Diagnostik

Wiesbaden, Hesse, 65191, Germany

Location

Medizinische Hochschule Hannover

Hanover, Lower Saxony, 30625, Germany

Location

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

Milan, MI, 20132, Italy

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, 20162, Italy

Location

Aichi Medical University Hospital

Nagakute-shi, Aichi-ken, 480-1195, Japan

Location

National Hospital Organization Chiba-East-Hospital

Chiba, Chiba, 260-8712, Japan

Location

Murakami Karindoh Hospital

Fukuoka, Fukuoka, 819-8585, Japan

Location

Fukushima Medical University Hospital

Fukushima, Fukushima, 960-1295, 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

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

Location

Kitasato University Hospital

Sagamihara, Kanagawa, 252-0375, Japan

Location

Yokohama City University Hospital

Yokohama, Kanagawa, 236-0004, Japan

Location

National Hospital Organization Kumamoto Saishun Medical Center

Koshi-shi, Kumamoto, 861-1196, Japan

Location

National Hospital Organization Utano Hospital

Kyoto, Kyoto, 616-8255, Japan

Location

Tohoku University Hospital

Sendai, Miyagi, 980-8574, Japan

Location

Niigata University Medical And Dental Hospital

Niigata, Niigata, 951-8520, Japan

Location

National Hospital Organization Toneyama Medical Center

Toyonaka-shi, Osaka, 560-8552, Japan

Location

Shiga University of Medical Science Hospital

Ōtsu, Shiga, 520-2192, Japan

Location

National Hospital Organization Shizuoka Institute of Epilepsy and Neurological Disorders

Shizuoka, Shizuoka, 420-8688, Japan

Location

Juntendo University Hospital

Bunkyo-ku, Tokyo, 113-8431, Japan

Location

Related Publications (1)

• Genge A, Pattee GL, Sobue G, Aoki M, Yoshino H, Couratier P, Lunetta C, Petri S, Selness D, Bidani S, Hirai M, Sakata T, Salah A, Apple S, Wamil A, Kalin A, Jackson CE. Oral edaravone demonstrated a favorable safety profile in patients with amyotrophic lateral sclerosis after 48 weeks of treatment. Muscle Nerve. 2023 Feb;67(2):124-129. doi: 10.1002/mus.27768. Epub 2022 Dec 28.

  PMID: 36504406 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

Please email

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: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 13, 2019
• First Posted: November 18, 2019
• Study Start: November 18, 2019
• Primary Completion: October 7, 2021
• Study Completion: October 7, 2021
• Last Updated: December 30, 2025
• Results First Posted: August 29, 2022

Record last verified: 2025-12

Locations

JP (17); US (22); CA (4); DE (2); IT (3); FR (5)