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

ALS Clinical Trial

Official title:

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)

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT05151471
• Other study ID #: MT-1186-A04
• Status: Terminated
• Phase: Phase 3
• Start date: January 11, 2022
• Est. completion date: September 29, 2023

Study information

• Verified date: October 2023
• Source: Mitsubishi Tanabe Pharma America Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 202
• Est. completion date: September 29, 2023
• Est. primary completion date: September 29, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• ALS

Intervention

Drug:
• MT-1186
Oral edaravone
• Placebo
Oral

Locations

Country	Name	City	State
Canada	University of Alberta - Walter C Mackenzie Health Sciences Centre (WCM)	Edmonton	Alberta
Canada	Health Science Center Mcmaster University	Hamilton	Ontario
Canada	London Health Sciences Centre - University Hospital	London	Ontario
Canada	CHU de Quebec-Hopital-Enfant-Jesus	Quebec City	Quebec
Germany	Universitaetsklinikum Wuerzburg	Würzburg
Japan	National Hospital Organization Chibahigashi National Hospital	Chiba-shi	Chiba
Japan	Tokyo Metropolitan Neurological Hospital	Fuchu-city	Tokyo
Japan	Murakami Karindoh Hospital	Fukuoka-city	Fukuoka
Japan	Fukushima Medical University Hospital	Fukushima-shi	Fukushima
Japan	Hiroshima University Hospital	Hiroshima-shi	Hiroshima
Japan	Teikyo University Hospital	Itabashi-ku	Tokyo
Japan	National Hospital Organization Iou National Hospital	Kanazawa-shi	Ishikawa
Japan	Kagawa University Hospital	Kita-gun	Kagawa
Japan	National Hospital Organization Kumamoto Saishun Medical Center	Koshi	Kumamoto
Japan	National Hospital Organization Utano National Hospital	Kyoto City	Kyoto
Japan	Nagoya University Hospital	Nagoya	Aichi
Japan	National Hospital Organization Higashinagoya National Hospital	Nagoya	Aichi
Japan	Niigata University Medical & Dental Hospital	Niigata-shi	Niigata
Japan	Kansai Electric Power Hospital	Osaka-shi	Osaka
Japan	Toho University Omori Medical Center	Ota-ku	Tokyo
Japan	Saitama Neuropsychiatric Institute	Saitama
Japan	Tohoku University Hospital	Sendai	Miyagi
Japan	National Hospital Organization Shizuoka Institute of Epilepsy and Neurological Disorders	Shizuoka-city	Shizuoka
Japan	National Hospital Organization Osaka Toneyama Medical Center	Toyonaka	Osaka
Japan	Yokohama City University Hospital	Yokohama-shi	Kanagawa
Korea, Republic of	Hanyang University Medical Center	Wangsimni-ro	Seongdong-gu
Switzerland	Chefarzt Muskelzentrum/ALS Clinic Kantonsspital St.Gallen Muskelzentrum/ALS Clinic	St.Gallen
United States	Emory University - School of Medicine	Atlanta	Georgia
United States	Johns Hopkins University	Baltimore	Maryland
United States	Northwestern University Feinberg School of Medicine	Chicago	Illinois
United States	UF Health Cancer Center/Clinical Trials Office	Gainesville	Florida
United States	Nerve And Muscle Center Of Texas	Houston	Texas
United States	Neurology Associates, P.C - Lincoln	Lincoln	Nebraska
United States	West Virginia University School of Medicine (WVUSoM) - Movement Disorder Clinic	Morgantown	West Virginia
United States	University Of Pittsburgh Medical Center	Pittsburgh	Pennsylvania
United States	Woodland Research Northwest, LLC	Rogers	Arkansas
United States	University of Washington Medical Center	Seattle	Washington

Sponsors (1)

Lead Sponsor	Collaborator
Mitsubishi Tanabe Pharma America Inc.

Countries where clinical trial is conducted

United States,  Canada,  Germany,  Japan,  Korea, Republic of,  Switzerland, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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 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.	Up to 96 weeks
Secondary	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.	Up to 96 weeks
Secondary	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).	Up to 96 weeks
Secondary	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.	Up to 96 weeks
Secondary	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
Secondary	Time from the randomization date in Study MT-1186-A02 to death or permanent assisted mechanical ventilation (>23 hours/day)		Up to 96 weeks
Secondary	Time from the randomization date in Study MT-1186-A02 to death		Up to 96 weeks