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

A Phase 3b, Multicenter, Randomized, Double-blind Extension Study to Evaluate the Continued Efficacy and Safety of Oral Edaravone Administered for an Additional Period of up to 48 Weeks Following Study MT-1186-A02 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 time from the randomization date in Study MT-1186-A02 to at least a 12-point decrease in Revised ALS Functional Rating Score (ALSFRS-R) or death, whichever happens first, over the course of the study or until oral edaravone is commercially available in that country

Study Overview

• Status: Terminated
• Conditions: ALS
• Intervention / Treatment: Drug: MT-1186; Drug: Placebo

• Study Type: Interventional
• Enrollment (Actual): 202
• Phase: Phase 3

Contacts and Locations

Study Locations

• Canada
  • Alberta
    • Edmonton, Alberta, Canada, T6G 2B7
      • University of Alberta - Walter C Mackenzie Health Sciences Centre (WCM)
  • Ontario
    • Hamilton, Ontario, Canada, L8P 1H1
      • Health Science Center Mcmaster University
    • London, Ontario, Canada, N6A 5A5
      • London Health Sciences Centre - University Hospital
  • Quebec
    • Québec, Quebec, Canada, G1J 1Z4
      • CHU de Quebec-Hopital-Enfant-Jesus
• Germany
  • Würzburg, Germany, 97080
    • Universitaetsklinikum Wuerzburg
• Japan
  • Saitama, Japan, 338-8577
    • Saitama Neuropsychiatric Institute
  • Aichi-ken
    • Nagoya, Aichi-ken, Japan, 466-8560
      • Nagoya University Hospital
    • Nagoya, Aichi-ken, Japan, 465-8620
      • National Hospital Organization Higashinagoya National Hospital
  • Chiba
    • Chiba, Chiba, Japan, 260-8712
      • National Hospital Organization Chibahigashi National Hospital
  • Fukuoka
    • Fukuoka, Fukuoka, Japan, 819-8585
      • Murakami Karindoh Hospital
  • Fukushima
    • Fukushima, Fukushima, Japan, 960-1295
      • Fukushima Medical University Hospital
  • Hiroshima
    • Hiroshima, Hiroshima, Japan, 734-8551
      • Hiroshima University Hospital
  • Ishikawa-ken
    • Kanazawa, Ishikawa-ken, Japan, 920-0192
      • National Hospital Organization Iou National Hospital
  • Kagawa-ken
    • Kita-gun, Kagawa-ken, Japan, 761-0793
      • Kagawa University Hospital
  • Kanagawa
    • Yokohama, Kanagawa, Japan, 236-0004
      • Yokohama City University Hospital
  • Kumamoto
    • Kōshi, Kumamoto, Japan, 861-1196
      • National Hospital Organization Kumamoto Saishun Medical Center
  • Kyoto
    • Kyoto, Kyoto, Japan, 616-8255
      • National Hospital Organization Utano National Hospital
  • Miyagi
    • Sendai, Miyagi, Japan, 980-8574
      • Tohoku University Hospital
  • Niigata
    • Niigata, Niigata, Japan, 951-8520
      • Niigata University Medical & Dental Hospital
  • Osaka
    • Osaka, Osaka, Japan, 553-0003
      • Kansai Electric Power Hospital
    • Toyonaka, Osaka, Japan, 560-8552
      • National Hospital Organization Osaka Toneyama Medical Center
  • Shizuoka
    • Shizuoka, Shizuoka, Japan, 420-8688
      • National Hospital Organization Shizuoka Institute of Epilepsy and Neurological Disorders
  • Tokyo
    • Fuchū, Tokyo, Japan, 183-0042
      • Tokyo Metropolitan Neurological Hospital
    • Itabashi-ku, Tokyo, Japan, 173-8606
      • Teikyo University Hospital
    • Ōta-ku, Tokyo, Japan, 143-8541
      • Toho University Omori Medical Center
• South Korea
  • Seongdong-gu
    • Wangsimni-ro, Seongdong-gu, South Korea, 04763
      • Hanyang University Medical Center
• Switzerland
  • Sankt Gallen, Switzerland, 9007
    • Chefarzt Muskelzentrum/ALS Clinic Kantonsspital St.Gallen Muskelzentrum/ALS Clinic
• United States
  • Arkansas
    • Rogers, Arkansas, United States, 72758
      • Woodland Research Northwest, LLC
  • Florida
    • Gainesville, Florida, United States, 32610-3633
      • UF Health Cancer Center/Clinical Trials Office
  • Georgia
    • Atlanta, Georgia, United States, 30317-2819
      • Emory University - School of Medicine
  • Illinois
    • Chicago, Illinois, United States, 60611-2605
      • Northwestern University Feinberg School of Medicine
  • Maryland
    • Baltimore, Maryland, United States, 21205
      • Johns Hopkins University
  • Nebraska
    • Lincoln, Nebraska, United States, 68506
      • Neurology Associates, P.C - Lincoln
  • Pennsylvania
    • Pittsburgh, Pennsylvania, United States, 15213
      • University of Pittsburgh Medical Center
  • Texas
    • Houston, Texas, United States, 77019
      • Nerve And Muscle Center Of Texas
  • Washington
    • Seattle, Washington, United States, 98195
      • University of Washington Medical Center
  • West Virginia
    • Morgantown, West Virginia, United States, 26506-9180
      • West Virginia University School of Medicine (WVUSoM) - Movement Disorder Clinic

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Subjects or their legally authorized representative must provide a signed and dated informed consent form to participate in the study.
2. 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.
3. Subjects must be willing to cooperate and comply with all protocol restrictions and requirements.
4. Subjects must have successfully completed all Study MT-1186-A02 visits and have been compliant with study drug.

Exclusion Criteria:

1. Subjects of childbearing potential unwilling to use an acceptable method of contraception from the Day 1/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.
2. Subjects who are female, of childbearing potential, and pregnant (a positive pregnancy test) or lactating at the Day 1/screening visit.
3. Subjects who have a significant risk of suicide. 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) at Week 48 of Study MT-1186-A02.
4. Subjects who are not eligible to continue in the study, as judged by the Investigator in conjunction with the MTDA medical monitor.
5. Subjects who are unable to take their medications orally or through a PEG/RIG tube.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: MT-1186 - Group 1; Oral edaravone administered once daily for up to 48 weeks or until the drug is commercially available in that country.	Drug: MT-1186; Oral edaravone; Other Names: Oral edaravone
Experimental: MT-1186 - Group 2; Oral edaravone administered for 10 days followed by 18-day placebo (regimen denoted as on/off) for up to 48 weeks or until the drug is commercially available in that country.	Drug: MT-1186; Oral edaravone; Other Names: Oral edaravone; Drug: Placebo; Oral; Other Names: Oral

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Time From the Randomization Date in Study MT-1186-A02 to at Least a 12-point Decrease in ALSFRS-R or Death, Whichever Happens First.	The ASLFRS-R is Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised. The ALSFRS-R measures 12 aspects of physical function, ranging from one's ability to swallow and use utensils to climbing stairs and breathing. Each function is scored from 4 (normal) to 0 (no ability), with a maximum total score of 48 and a minimum total score of 0.	Up to 96 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The Combined Assessment of Function and Survival (CAFS) Score at All Visits From Baseline in Study MT-1186-A02 to the End of Study MT-1186-A04	CAFS analysis ranks clinical outcomes on the basis of survival time and change in the ALS Functional Rating Scale-Revised (ALSFRS-R) score. A subject's score will be calculated by comparing each subject to every other subject within each treatment group in the study, resulting in a score of +1 if the outcome was better than the subject being compared, -1 if worse, and 0 if the same. The subject's score will then be calculated by summing up their comparison to all of the other subjects within each treatment group in the study as CAFS score. Patients' summary scores are ranked; a higher score indicates a better performance. The score range is from 1 to N, where N is the total sample size. 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.	Baseline, Week 72 and Week 96
Change in the Amyotrophic Lateral Sclerosis Assessment Questionnaire 40 Score at All Visits From Baseline in Study MT-1186-A02 to the End of Study MT-1186-A04	The ALSAQ-40 is a questionnaire that consists of 40 questions/items with 5 discrete scales: physical mobility, activities of daily living and independence, eating and drinking, communication, emotional reactions, ranging from 0 (best health as assessed by the scale) to 100 (worse health as assessed by the measure).	Baseline, Week 72 and Week 96
Change in ALSFRS-R Score at All Visits From Baseline in Study MT-1186-A02 to the End of Study MT-1186-A04	The ALSFRS-R is rating scale (ratings 0 = can't do, to 4 = normal ability) used to determine participants' assessment of their capability and independence in 12 functional activities.	Baseline, Week 72, Week 84 and Week 96
Time From the Randomization Date in Study MT-1186-A02 to Death, Tracheostomy, or Permanent Assisted Mechanical Ventilation (≥23 Hours/Day)		Up to 96 weeks
Time From the Randomization Date in Study MT-1186-A02 to Death or Permanent Assisted Mechanical Ventilation (>23 Hours/Day)		Up to 96 weeks
Time From the Randomization Date in Study MT-1186-A02 to Death		Up to 96 weeks

Collaborators and Investigators

• Sponsor: Tanabe Pharma America, Inc.
• Investigators: Study Director: Head of Medical Science, Tanabe Pharma America, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): January 11, 2022
• Primary Completion (Actual): September 29, 2023
• Study Completion (Actual): September 29, 2023

Study Registration Dates

• First Submitted: November 28, 2021
• First Submitted That Met QC Criteria: November 28, 2021
• First Posted (Actual): December 9, 2021

Study Record Updates

• Last Update Posted (Actual): May 22, 2026
• Last Update Submitted That Met QC Criteria: May 21, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Azoles; Pyrazoles; Antipyrine; Pyrazolones; Edaravone
• Other Study ID Numbers: MT-1186-A04; jRCT2071210117 (Registry Identifier: Japan Registry of Clinical Trials (jRCT)); 2021-003900-42 (EudraCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No