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

NCT05151471 | Terminated Phase 3 Results DMC FDA Drug

• 3 other identifiers: MT-1186-A04jRCT20712101172021-003900-42
• Study Type: interventional
• Target: 202
• Locations: 6 countries
• Sites: 37

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 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

Conditions

ALS

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (6)

• 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

  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

  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

  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

Study Arms (2)

MT-1186 - Group 1

EXPERIMENTAL

Oral edaravone administered once daily for up to 48 weeks or until the drug is commercially available in that country.

Drug: MT-1186

MT-1186 - Group 2

EXPERIMENTAL

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; Drug: Placebo

Interventions

MT-1186 DRUG

Oral edaravone

Also known as: Oral edaravone

MT-1186 - Group 1; MT-1186 - Group 2

Placebo DRUG

Oral

Also known as: Oral

MT-1186 - Group 2

Eligibility Criteria

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

You may qualify if:

• Subjects or their legally authorized representative must provide a signed and dated informed consent form 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 must have successfully completed all Study MT-1186-A02 visits and have been compliant with study drug.

You may not qualify if:

• 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.
• Subjects who are female, of childbearing potential, and pregnant (a positive pregnancy test) or lactating at the Day 1/screening visit.
• 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.
• Subjects who are not eligible to continue in the study, as judged by the Investigator in conjunction with the MTDA medical monitor.
• Subjects who are unable to take their medications orally or through a PEG/RIG tube.

Sponsors & Collaborators

• Tanabe Pharma America, Inc.: lead

Study Sites (37)

Woodland Research Northwest, LLC

Rogers, Arkansas, 72758, United States

Location

UF Health Cancer Center/Clinical Trials Office

Gainesville, Florida, 32610-3633, United States

Location

Emory University - School of Medicine

Atlanta, Georgia, 30317-2819, United States

Location

Northwestern University Feinberg School of Medicine

Chicago, Illinois, 60611-2605, United States

Location

Johns Hopkins University

Baltimore, Maryland, 21205, United States

Location

Neurology Associates, P.C - Lincoln

Lincoln, Nebraska, 68506, United States

Location

University Of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, 15213, United States

Location

Nerve And Muscle Center Of Texas

Houston, Texas, 77019, United States

Location

University of Washington Medical Center

Seattle, Washington, 98195, United States

Location

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

Morgantown, West Virginia, 26506-9180, United States

Location

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

Edmonton, Alberta, T6G 2B7, 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

CHU de Quebec-Hopital-Enfant-Jesus

Québec, Quebec, G1J 1Z4, Canada

Location

Universitaetsklinikum Wuerzburg

Würzburg, 97080, Germany

Location

National Hospital Organization Higashinagoya National Hospital

Nagoya, Aichi-ken, 465-8620, Japan

Location

Nagoya University Hospital

Nagoya, Aichi-ken, 466-8560, Japan

Location

National Hospital Organization Chibahigashi National 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

Hiroshima University Hospital

Hiroshima, Hiroshima, 734-8551, 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

Yokohama City University Hospital

Yokohama, Kanagawa, 236-0004, Japan

Location

National Hospital Organization Kumamoto Saishun Medical Center

Kōshi, Kumamoto, 861-1196, Japan

Location

National Hospital Organization Utano National Hospital

Kyoto, Kyoto, 616-8255, Japan

Location

Tohoku University Hospital

Sendai, Miyagi, 980-8574, Japan

Location

Niigata University Medical & Dental Hospital

Niigata, Niigata, 951-8520, Japan

Location

Kansai Electric Power Hospital

Osaka, Osaka, 553-0003, Japan

Location

National Hospital Organization Osaka Toneyama Medical Center

Toyonaka, Osaka, 560-8552, Japan

Location

National Hospital Organization Shizuoka Institute of Epilepsy and Neurological Disorders

Shizuoka, Shizuoka, 420-8688, 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

Saitama Neuropsychiatric Institute

Saitama, 338-8577, Japan

Location

Hanyang University Medical Center

Wangsimni-ro, Seongdong-gu, 04763, South Korea

Location

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

Sankt Gallen, 9007, Switzerland

Location

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: November 28, 2021
• First Posted: December 9, 2021
• Study Start: January 11, 2022
• Primary Completion: September 29, 2023
• Study Completion: September 29, 2023
• Last Updated: December 30, 2025
• Results First Posted: March 27, 2025

Record last verified: 2025-12

Locations

US (10); KR (1); JP (20); DE (1); CH (1); CA (4)