Efficacy and Safety Extension Study of Oral Edaravone Administered in Subjects With ALS
- 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
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 202
- 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.
- 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.
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 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.
- 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 Baseline, Week 72 and Week 96 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).
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 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 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 Baseline, Week 72 and Week 96 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.
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