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

Phase 3

Terminated

• Conditions: ALS
• Interventions: Drug: MT-1186; Drug: Placebo

• Registration Number: NCT05151471
• Lead Sponsor: Mitsubishi Tanabe Pharma America Inc.

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

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2021-11-28
• Study First Submitted QC: 2021-11-28
• Study First Posted: 2021-12-09
• Study Start: 2022-01-11
• Primary Completion: 2023-09-29
• Study Completion: 2023-09-29
• Status Verified: 2023-10
• Last Update Submitted: 2023-10-26
• Last Update Posted: 2023-10-30

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 202

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.

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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.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
MT-1186 - Group 2	Placebo	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.
MT-1186 - Group 1	MT-1186	Oral edaravone administered once daily for up to 48 weeks or until the drug is commercially available in that country.
MT-1186 - Group 2	MT-1186	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.

Primary Outcome Measures

Name	Time	Method
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.	Up to 96 weeks	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.

Secondary Outcome Measures

Name	Time	Method
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	Up to 96 weeks	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).
Time from the randomization date in Study MT-1186-A02 to death or permanent assisted mechanical ventilation (>23 hours/day)	Up to 96 weeks
Change in ALSFRS-R score at all visits from baseline in Study MT-1186-A02 to the end of Study MT-1186-A04	Up to 96 weeks	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.
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	Up to 96 weeks
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	Up to 96 weeks	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.

Trial Locations

Locations (37)

Nerve And Muscle Center Of Texas; 🇺🇸 Houston, Texas, United States

University of Washington Medical Center; 🇺🇸 Seattle, Washington, United States

University Of Pittsburgh Medical Center; 🇺🇸 Pittsburgh, Pennsylvania, United States

Fukushima Medical University Hospital; 🇯🇵 Fukushima-shi, Fukushima, Japan

Hiroshima University Hospital; 🇯🇵 Hiroshima-shi, Hiroshima, Japan

UF Health Cancer Center/Clinical Trials Office; 🇺🇸 Gainesville, Florida, United States

Emory University - School of Medicine; 🇺🇸 Atlanta, Georgia, United States

Northwestern University Feinberg School of Medicine; 🇺🇸 Chicago, Illinois, United States

Woodland Research Northwest, LLC; 🇺🇸 Rogers, Arkansas, United States

Neurology Associates, P.C - Lincoln; 🇺🇸 Lincoln, Nebraska, United States

University of Alberta - Walter C Mackenzie Health Sciences Centre (WCM); 🇨🇦 Edmonton, Alberta, Canada

Health Science Center Mcmaster University; 🇨🇦 Hamilton, Ontario, Canada

Universitaetsklinikum Wuerzburg; 🇩🇪 Würzburg, Germany

National Hospital Organization Iou National Hospital; 🇯🇵 Kanazawa-shi, Ishikawa, Japan

National Hospital Organization Kumamoto Saishun Medical Center; 🇯🇵 Koshi, Kumamoto, Japan

National Hospital Organization Higashinagoya National Hospital; 🇯🇵 Nagoya, Aichi, Japan

National Hospital Organization Chibahigashi National Hospital; 🇯🇵 Chiba-shi, Chiba, Japan

Kagawa University Hospital; 🇯🇵 Kita-gun, Kagawa, Japan

Kansai Electric Power Hospital; 🇯🇵 Osaka-shi, Osaka, Japan

Teikyo University Hospital; 🇯🇵 Itabashi-ku, Tokyo, Japan

Hanyang University Medical Center; 🇰🇷 Wangsimni-ro, Seongdong-gu, Korea, Republic of

Johns Hopkins University; 🇺🇸 Baltimore, Maryland, United States

CHU de Quebec-Hopital-Enfant-Jesus; 🇨🇦 Quebec City, Quebec, Canada

Nagoya University Hospital; 🇯🇵 Nagoya, Aichi, Japan

Murakami Karindoh Hospital; 🇯🇵 Fukuoka-city, Fukuoka, Japan

National Hospital Organization Utano National Hospital; 🇯🇵 Kyoto City, Kyoto, Japan

Niigata University Medical & Dental Hospital; 🇯🇵 Niigata-shi, Niigata, Japan

Tokyo Metropolitan Neurological Hospital; 🇯🇵 Fuchu-city, Tokyo, Japan

National Hospital Organization Osaka Toneyama Medical Center; 🇯🇵 Toyonaka, Osaka, Japan

National Hospital Organization Shizuoka Institute of Epilepsy and Neurological Disorders; 🇯🇵 Shizuoka-city, Shizuoka, Japan

Toho University Omori Medical Center; 🇯🇵 Ota-ku, Tokyo, Japan

Saitama Neuropsychiatric Institute; 🇯🇵 Saitama, Japan

Chefarzt Muskelzentrum/ALS Clinic Kantonsspital St.Gallen Muskelzentrum/ALS Clinic; 🇨🇭 St.Gallen, Switzerland

West Virginia University School of Medicine (WVUSoM) - Movement Disorder Clinic; 🇺🇸 Morgantown, West Virginia, United States

London Health Sciences Centre - University Hospital; 🇨🇦 London, Ontario, Canada

Yokohama City University Hospital; 🇯🇵 Yokohama-shi, Kanagawa, Japan

Tohoku University Hospital; 🇯🇵 Sendai, Miyagi, Japan