Trial Outcomes & Findings for Efficacy and Safety Extension Study of Oral Edaravone Administered in Subjects With ALS (NCT NCT05151471)

Last Updated: 2025-12-30

Results Overview

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.

Recruitment status

TERMINATED

Study phase

PHASE3

Target enrollment

202 participants

Primary outcome timeframe

Up to 96 weeks

Results posted on

2025-12-30

Participant Flow

Participant milestones

Participant milestones
Measure	Edaravone 105 mg (Once Daily) Oral edaravone administered once daily for up to 48 weeks or until the drug is commercially available in that country. MT-1186: Oral edaravone	Edaravone 105 mg (on/Off) 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: Oral edaravone Placebo: Oral
Overall Study STARTED	104	98
Overall Study COMPLETED	24	24
Overall Study NOT COMPLETED	80	74

Reasons for withdrawal

Reasons for withdrawal
Measure	Edaravone 105 mg (Once Daily) Oral edaravone administered once daily for up to 48 weeks or until the drug is commercially available in that country. MT-1186: Oral edaravone	Edaravone 105 mg (on/Off) 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: Oral edaravone Placebo: Oral
Overall Study Adverse Event	21	16
Overall Study Death	1	0
Overall Study Lost to Follow-up	0	1
Overall Study Withdrawal by Subject	7	9
Overall Study Study terminated by sponsor	49	47
Overall Study Other	2	1

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Edaravone 105 mg (Once Daily) n=192 Participants Oral edaravone administered once daily for up to 48 weeks or until the drug is commercially available in that country. MT-1186: Oral edaravone	Edaravone 105 mg (on/Off) n=192 Participants 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: Oral edaravone Placebo: Oral	Total n=384 Participants Total of all reporting groups
Age, Customized Adults (18-64 years)	134 Participants n=174 Participants	122 Participants n=166 Participants	256 Participants n=167 Participants
Age, Customized From 65-84 years	58 Participants n=174 Participants	70 Participants n=166 Participants	128 Participants n=167 Participants
Sex: Female, Male Female	69 Participants n=174 Participants	70 Participants n=166 Participants	139 Participants n=167 Participants
Sex: Female, Male Male	123 Participants n=174 Participants	122 Participants n=166 Participants	245 Participants n=167 Participants
Race/Ethnicity, Customized White	115 Participants n=174 Participants	109 Participants n=166 Participants	224 Participants n=167 Participants
Race/Ethnicity, Customized Black or African American	3 Participants n=174 Participants	2 Participants n=166 Participants	5 Participants n=167 Participants
Race/Ethnicity, Customized Asian - Japanese	63 Participants n=174 Participants	64 Participants n=166 Participants	127 Participants n=167 Participants
Race/Ethnicity, Customized Asian - Not Japanese	8 Participants n=174 Participants	13 Participants n=166 Participants	21 Participants n=167 Participants
Race/Ethnicity, Customized American Indian or Alaska Native	0 Participants n=174 Participants	1 Participants n=166 Participants	1 Participants n=167 Participants
Race/Ethnicity, Customized Native Hawaiian or Pacific Islander	0 Participants n=174 Participants	1 Participants n=166 Participants	1 Participants n=167 Participants
Race/Ethnicity, Customized Not Reported	0 Participants n=174 Participants	1 Participants n=166 Participants	1 Participants n=167 Participants
Race/Ethnicity, Customized Other	3 Participants n=174 Participants	1 Participants n=166 Participants	4 Participants n=167 Participants

PRIMARY outcome

Timeframe: Up to 96 weeks

Population: The outcome measure time frame is from the randomization date in Study MT- 1186-A02 to at least a 12-point decrease in ALSFRS-R or death, whichever happened first.

Outcome measures

Outcome measures
Measure	Edaravone 105 mg (Once Daily) n=192 Participants Oral edaravone administered once daily for up to 48 weeks or until the drug is commercially available in that country. MT-1186: Oral edaravone	Edaravone 105 mg (on/Off) n=192 Participants 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: Oral edaravone Placebo: Oral
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.	13.9 Month Interval 13.4 to 16.59	15.05 Month Interval 13.8 to 16.62

SECONDARY outcome

Timeframe: Baseline, Week 72 and Week 96

Population: One subject randomized to the On/Off treatment group was excluded from this analysis because the subject did not receive any study treatment.

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.

Outcome measures

Outcome measures
Measure	Edaravone 105 mg (Once Daily) n=192 Participants Oral edaravone administered once daily for up to 48 weeks or until the drug is commercially available in that country. MT-1186: Oral edaravone	Edaravone 105 mg (on/Off) n=191 Participants 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: Oral edaravone Placebo: Oral
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 Week 72	193.6 units on a scale Interval 4.0 to 383.0	189.1 units on a scale Interval 1.0 to 383.0
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 Week 96	200.8 units on a scale Interval 4.0 to 383.0	181.6 units on a scale Interval 1.0 to 383.0

SECONDARY outcome

Timeframe: Baseline, Week 72 and Week 96

Population: Those patients reached to Week 72 and Week 96, and conducted ALSAQ-40 assessment.

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).

Outcome measures

Outcome measures
Measure	Edaravone 105 mg (Once Daily) n=192 Participants Oral edaravone administered once daily for up to 48 weeks or until the drug is commercially available in that country. MT-1186: Oral edaravone	Edaravone 105 mg (on/Off) n=191 Participants 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: Oral edaravone Placebo: Oral
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 Week 72	45.71 Point Standard Error 3.14	45.46 Point Standard Error 3.11
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 Week 96	54.83 Point Standard Error 4.05	57.91 Point Standard Error 4.13

SECONDARY outcome

Timeframe: Baseline, Week 72, Week 84 and Week 96

Population: Those patients reached to Week 72, Week 84 and Week 96, and conducted ALSFRS-R assessment.

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.

Outcome measures

Outcome measures
Measure	Edaravone 105 mg (Once Daily) n=192 Participants Oral edaravone administered once daily for up to 48 weeks or until the drug is commercially available in that country. MT-1186: Oral edaravone	Edaravone 105 mg (on/Off) n=191 Participants 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: Oral edaravone Placebo: Oral
Change in ALSFRS-R Score at All Visits From Baseline in Study MT-1186-A02 to the End of Study MT-1186-A04 Week 96	-18.21 Point Standard Error 1.31	-20.95 Point Standard Error 1.32
Change in ALSFRS-R Score at All Visits From Baseline in Study MT-1186-A02 to the End of Study MT-1186-A04 Week 72	-15.02 Point Standard Error 0.95	-15.90 Point Standard Error 0.95
Change in ALSFRS-R Score at All Visits From Baseline in Study MT-1186-A02 to the End of Study MT-1186-A04 Week 84	-16.79 Point Standard Error 1.11	-18.27 Point Standard Error 1.10

SECONDARY outcome

Timeframe: Up to 96 weeks

Population: The outcome measure time frame is from Baseline in Study MT-1186-A02 to the End of Study MT-1186-A04.

Outcome measures

Outcome measures
Measure	Edaravone 105 mg (Once Daily) n=192 Participants Oral edaravone administered once daily for up to 48 weeks or until the drug is commercially available in that country. MT-1186: Oral edaravone	Edaravone 105 mg (on/Off) n=192 Participants 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: Oral edaravone Placebo: Oral
Time From the Randomization Date in Study MT-1186-A02 to Death, Tracheostomy, or Permanent Assisted Mechanical Ventilation (≥23 Hours/Day)	NA Month Interval 19.36 to 21.02 The median survival for the time to death could not be calculated due to the low number of events (only 33 and 32 events of death, tracheostomy, or PAMV in the Once Daily and On/Off groups, respectively), resulting in 159 and 160 censored observations in respective group (i.e., NA means NE (Not estimable)).	NA Month Interval 19.43 to 21.13 The median survival for the time to death could not be calculated due to the low number of events (only 33 and 32 events of death, tracheostomy, or PAMV in the Once Daily and On/Off groups, respectively), resulting in 159 and 160 censored observations in respective group (i.e., NA means NE (Not estimable)).

SECONDARY outcome

Timeframe: Up to 96 weeks

Population: The outcome measure time frame is from Baseline in Study MT-1186-A02 to the End of Study MT-1186-A04.

Outcome measures

Outcome measures
Measure	Edaravone 105 mg (Once Daily) n=192 Participants Oral edaravone administered once daily for up to 48 weeks or until the drug is commercially available in that country. MT-1186: Oral edaravone	Edaravone 105 mg (on/Off) n=192 Participants 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: Oral edaravone Placebo: Oral
Time From the Randomization Date in Study MT-1186-A02 to Death or Permanent Assisted Mechanical Ventilation (>23 Hours/Day)	NA Month The median survival for the time to death could not be calculated due to the low number of events (only 30 and 31 events of death or PAMV in the Once Daily and On/Off groups, respectively), resulting in 162 and 161 censored observations in respective group (i.e., NA of Median means NE (Not estimable)).	NA Month The median survival for the time to death could not be calculated due to the low number of events (only 30 and 31 events of death or PAMV in the Once Daily and On/Off groups, respectively), resulting in 162 and 161 censored observations in respective group (i.e., NA of Median means NE (Not estimable)).

SECONDARY outcome

Timeframe: Up to 96 weeks

Population: The outcome measure time frame is from Baseline in Study MT-1186-A02 to the End of Study MT-1186-A04.

Outcome measures

Outcome measures
Measure	Edaravone 105 mg (Once Daily) n=192 Participants Oral edaravone administered once daily for up to 48 weeks or until the drug is commercially available in that country. MT-1186: Oral edaravone	Edaravone 105 mg (on/Off) n=192 Participants 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: Oral edaravone Placebo: Oral
Time From the Randomization Date in Study MT-1186-A02 to Death	NA Month The median survival for the time to death could not be calculated due to the low number of events (only 24 and 28 events of death in the Once Daily and On/Off groups, respectively), resulting in 168 and 164 censored observations in respective group (i.e., NA of Median means NE (Not estimable)).	NA Month The median survival for the time to death could not be calculated due to the low number of events (only 24 and 28 events of death in the Once Daily and On/Off groups, respectively), resulting in 168 and 164 censored observations in respective group (i.e., NA of Median means NE (Not estimable)).

Adverse Events

Edaravone 105 mg (Once Daily)

Serious events: 33 serious events

Other events: 24 other events

Deaths: 15 deaths

Edaravone 105 mg (on/Off)

Serious events: 27 serious events

Other events: 25 other events

Deaths: 12 deaths

Serious adverse events

Other adverse events

Other adverse events
Measure	Edaravone 105 mg (Once Daily) n=104 participants at risk Oral edaravone administered once daily for up to 48 weeks or until the drug is commercially available in that country. MT-1186: Oral edaravone	Edaravone 105 mg (on/Off) n=98 participants at risk 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: Oral edaravone Placebo: Oral
General disorders Constipation	6.7% 7/104 • Up to 50 weeks All AEs and SAEs that occur from the time written ICF was obtained until the end of the Safety Follow-up Period or the withdrawal of the subject from the study. The All-Causes Mortality time frame is from Day1 to the End of study MT-1186-A04. On the other hand, time frame of Outcome Measure 7 is from Day1 in MT-1186-A02 to the End of study MT-1186-A04. Therefore, the number of participants of death (i.e., "15" and "12") is inconsistent with information in the NA Explanation in Outcome Measure 7.	6.1% 6/98 • Up to 50 weeks All AEs and SAEs that occur from the time written ICF was obtained until the end of the Safety Follow-up Period or the withdrawal of the subject from the study. The All-Causes Mortality time frame is from Day1 to the End of study MT-1186-A04. On the other hand, time frame of Outcome Measure 7 is from Day1 in MT-1186-A02 to the End of study MT-1186-A04. Therefore, the number of participants of death (i.e., "15" and "12") is inconsistent with information in the NA Explanation in Outcome Measure 7.
General disorders Diarrhoea	3.8% 4/104 • Up to 50 weeks All AEs and SAEs that occur from the time written ICF was obtained until the end of the Safety Follow-up Period or the withdrawal of the subject from the study. The All-Causes Mortality time frame is from Day1 to the End of study MT-1186-A04. On the other hand, time frame of Outcome Measure 7 is from Day1 in MT-1186-A02 to the End of study MT-1186-A04. Therefore, the number of participants of death (i.e., "15" and "12") is inconsistent with information in the NA Explanation in Outcome Measure 7.	5.1% 5/98 • Up to 50 weeks All AEs and SAEs that occur from the time written ICF was obtained until the end of the Safety Follow-up Period or the withdrawal of the subject from the study. The All-Causes Mortality time frame is from Day1 to the End of study MT-1186-A04. On the other hand, time frame of Outcome Measure 7 is from Day1 in MT-1186-A02 to the End of study MT-1186-A04. Therefore, the number of participants of death (i.e., "15" and "12") is inconsistent with information in the NA Explanation in Outcome Measure 7.
Infections and infestations COVID-19	5.8% 6/104 • Up to 50 weeks All AEs and SAEs that occur from the time written ICF was obtained until the end of the Safety Follow-up Period or the withdrawal of the subject from the study. The All-Causes Mortality time frame is from Day1 to the End of study MT-1186-A04. On the other hand, time frame of Outcome Measure 7 is from Day1 in MT-1186-A02 to the End of study MT-1186-A04. Therefore, the number of participants of death (i.e., "15" and "12") is inconsistent with information in the NA Explanation in Outcome Measure 7.	1.0% 1/98 • Up to 50 weeks All AEs and SAEs that occur from the time written ICF was obtained until the end of the Safety Follow-up Period or the withdrawal of the subject from the study. The All-Causes Mortality time frame is from Day1 to the End of study MT-1186-A04. On the other hand, time frame of Outcome Measure 7 is from Day1 in MT-1186-A02 to the End of study MT-1186-A04. Therefore, the number of participants of death (i.e., "15" and "12") is inconsistent with information in the NA Explanation in Outcome Measure 7.
Injury, poisoning and procedural complications Fall	7.7% 8/104 • Up to 50 weeks All AEs and SAEs that occur from the time written ICF was obtained until the end of the Safety Follow-up Period or the withdrawal of the subject from the study. The All-Causes Mortality time frame is from Day1 to the End of study MT-1186-A04. On the other hand, time frame of Outcome Measure 7 is from Day1 in MT-1186-A02 to the End of study MT-1186-A04. Therefore, the number of participants of death (i.e., "15" and "12") is inconsistent with information in the NA Explanation in Outcome Measure 7.	13.3% 13/98 • Up to 50 weeks All AEs and SAEs that occur from the time written ICF was obtained until the end of the Safety Follow-up Period or the withdrawal of the subject from the study. The All-Causes Mortality time frame is from Day1 to the End of study MT-1186-A04. On the other hand, time frame of Outcome Measure 7 is from Day1 in MT-1186-A02 to the End of study MT-1186-A04. Therefore, the number of participants of death (i.e., "15" and "12") is inconsistent with information in the NA Explanation in Outcome Measure 7.
Psychiatric disorders Insomnia	4.8% 5/104 • Up to 50 weeks All AEs and SAEs that occur from the time written ICF was obtained until the end of the Safety Follow-up Period or the withdrawal of the subject from the study. The All-Causes Mortality time frame is from Day1 to the End of study MT-1186-A04. On the other hand, time frame of Outcome Measure 7 is from Day1 in MT-1186-A02 to the End of study MT-1186-A04. Therefore, the number of participants of death (i.e., "15" and "12") is inconsistent with information in the NA Explanation in Outcome Measure 7.	5.1% 5/98 • Up to 50 weeks All AEs and SAEs that occur from the time written ICF was obtained until the end of the Safety Follow-up Period or the withdrawal of the subject from the study. The All-Causes Mortality time frame is from Day1 to the End of study MT-1186-A04. On the other hand, time frame of Outcome Measure 7 is from Day1 in MT-1186-A02 to the End of study MT-1186-A04. Therefore, the number of participants of death (i.e., "15" and "12") is inconsistent with information in the NA Explanation in Outcome Measure 7.

Additional Information

Clinical Trials, Information Desk

Tanabe Pharma America, Inc.

Phone: 908-607-1980

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: OTHER