Trial Outcomes & Findings for Safety Extension Study of Oral Edaravone Administered in Subjects With Amyotrophic Lateral Sclerosis (ALS) (NCT NCT04577404)

Last Updated: 2025-12-30

Results Overview

AEs that occurred on or after the first dose in the MT-1186-A03 will be summarized, which implies AE that occurred during MT-1186-A01 will not be summarized. * Severe TEAEs: TEAEs which was classified as severe for the severity were analyzed. The severity of TEAEs was classified according to the following criteria: Mild (The event is transient and easily tolerated by the subject.), Moderate: The event causes discomfort and interferes with the subject's general condition.), and Severe (The event causes considerable interference with the subject's general condition and may be incapacitating.) * TESAEs, which is Serious TEAEs, is the TEAEs when the patients outcome is death, life-threatening, hospitalization, or disability or permanent damage.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

124 participants

Primary outcome timeframe

up to 96 Weeks

Results posted on

2025-12-30

Participant Flow

Participant milestones

Participant milestones
Measure	MT-1186 MT-1186 105mg (2 weeks On/Off)
Overall Study STARTED	124
Overall Study COMPLETED	49
Overall Study NOT COMPLETED	75

Reasons for withdrawal

Reasons for withdrawal
Measure	MT-1186 MT-1186 105mg (2 weeks On/Off)
Overall Study Adverse Event	31
Overall Study Death	1
Overall Study Lack of Efficacy	3
Overall Study Physician Decision	7
Overall Study Withdrawal by Subject	21
Overall Study Switched to commercial oral Edaravon etc.	12

Baseline Characteristics

Safety Extension Study of Oral Edaravone Administered in Subjects With Amyotrophic Lateral Sclerosis (ALS)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	MT-1186 n=124 Participants MT-1186 105mg (2 weeks On/Off)
Age, Continuous	59.0 years STANDARD_DEVIATION 10.1 • n=174 Participants
Age, Customized Adults (18-64 years)	84 Participants n=174 Participants
Age, Customized From 65 to 84 years	40 Participants n=174 Participants
Sex: Female, Male Female	41 Participants n=174 Participants
Sex: Female, Male Male	83 Participants n=174 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	2 Participants n=174 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	118 Participants n=174 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	4 Participants n=174 Participants

PRIMARY outcome

Timeframe: up to 96 Weeks

Outcome measures

Outcome measures
Measure	MT-1186 n=124 Participants MT-1186 105mg (2 weeks On/Off)
Number of Events of Treatment Emergency Adverse Events (TEAEs), Adverse Drug Reactions (ADRs), Severe TEAEs, TESEAs, TEAEs Leading to Discontinuation, Any TEAEs Leading to Death Number of TEAEs	616 events
Number of Events of Treatment Emergency Adverse Events (TEAEs), Adverse Drug Reactions (ADRs), Severe TEAEs, TESEAs, TEAEs Leading to Discontinuation, Any TEAEs Leading to Death Number of ADRs	17 events
Number of Events of Treatment Emergency Adverse Events (TEAEs), Adverse Drug Reactions (ADRs), Severe TEAEs, TESEAs, TEAEs Leading to Discontinuation, Any TEAEs Leading to Death Number of severe TEAEs	60 events
Number of Events of Treatment Emergency Adverse Events (TEAEs), Adverse Drug Reactions (ADRs), Severe TEAEs, TESEAs, TEAEs Leading to Discontinuation, Any TEAEs Leading to Death Number of TESAEs	75 events
Number of Events of Treatment Emergency Adverse Events (TEAEs), Adverse Drug Reactions (ADRs), Severe TEAEs, TESEAs, TEAEs Leading to Discontinuation, Any TEAEs Leading to Death Number of TEAEs leading to discontinuation	35 events
Number of Events of Treatment Emergency Adverse Events (TEAEs), Adverse Drug Reactions (ADRs), Severe TEAEs, TESEAs, TEAEs Leading to Discontinuation, Any TEAEs Leading to Death Number of TEAEs leading to death	19 events

PRIMARY outcome

Timeframe: up to 96 Weeks

AEs that occurred on or after the first dose in the MT-1186-A03 will be summarized, which implies AE that occurred during MT-1186-A01 will not be summarized.

Outcome measures

Outcome measures
Measure	MT-1186 n=124 Participants MT-1186 105mg (2 weeks On/Off)
Number of Subjects of Treatment Emergency Adverse Events (TEAEs), Adverse Drug Reactions (ADRs), Severe TEAEs, TESAEs, TEAEs Leading to Discontinuation and TEAEs Leading to Death Number of TEAEs leading to discontinuation	28 Participants
Number of Subjects of Treatment Emergency Adverse Events (TEAEs), Adverse Drug Reactions (ADRs), Severe TEAEs, TESAEs, TEAEs Leading to Discontinuation and TEAEs Leading to Death Number of TEAEs leading to death	19 Participants
Number of Subjects of Treatment Emergency Adverse Events (TEAEs), Adverse Drug Reactions (ADRs), Severe TEAEs, TESAEs, TEAEs Leading to Discontinuation and TEAEs Leading to Death Number of TESEAs	52 Participants
Number of Subjects of Treatment Emergency Adverse Events (TEAEs), Adverse Drug Reactions (ADRs), Severe TEAEs, TESAEs, TEAEs Leading to Discontinuation and TEAEs Leading to Death Number of TEAEs	113 Participants
Number of Subjects of Treatment Emergency Adverse Events (TEAEs), Adverse Drug Reactions (ADRs), Severe TEAEs, TESAEs, TEAEs Leading to Discontinuation and TEAEs Leading to Death Number of ADRs	12 Participants
Number of Subjects of Treatment Emergency Adverse Events (TEAEs), Adverse Drug Reactions (ADRs), Severe TEAEs, TESAEs, TEAEs Leading to Discontinuation and TEAEs Leading to Death Number of severe TEAEs	44 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline to Week 96

ALS Functional Rating Scale- Revised (ALSFRS-R). 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 data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: up to 96 Weeks

Outcome measures

Outcome data not reported

Adverse Events

MT-1186

Serious events: 52 serious events

Other events: 70 other events

Deaths: 19 deaths

Serious adverse events

Serious adverse events
Measure	MT-1186 n=124 participants at risk MT-1186 105mg (2 weeks On/Off)
Cardiac disorders Myocarditis	0.81% 1/124 • up to 96 weeks
Vascular disorders Phlebitis	0.81% 1/124 • up to 96 weeks
Respiratory, thoracic and mediastinal disorders Acute respiratory failure	0.81% 1/124 • up to 96 weeks
Respiratory, thoracic and mediastinal disorders Asphyxia	0.81% 1/124 • up to 96 weeks
Respiratory, thoracic and mediastinal disorders Dyspnoea	3.2% 4/124 • up to 96 weeks
Respiratory, thoracic and mediastinal disorders Pneumonia aspiration	7.3% 9/124 • up to 96 weeks
Respiratory, thoracic and mediastinal disorders Pulmonary embolism	0.81% 1/124 • up to 96 weeks
Respiratory, thoracic and mediastinal disorders Respiratory disorder	0.81% 1/124 • up to 96 weeks
Respiratory, thoracic and mediastinal disorders Respiratory failure	10.5% 13/124 • up to 96 weeks
Respiratory, thoracic and mediastinal disorders Sputum retention	0.81% 1/124 • up to 96 weeks
Gastrointestinal disorders Abdominal pain	0.81% 1/124 • up to 96 weeks
Gastrointestinal disorders Colitis	0.81% 1/124 • up to 96 weeks
Gastrointestinal disorders Dysphagia	5.6% 7/124 • up to 96 weeks
Gastrointestinal disorders Ileus paralytic	0.81% 1/124 • up to 96 weeks
Gastrointestinal disorders Aerophagia	0.81% 1/124 • up to 96 weeks
Hepatobiliary disorders Cholecystitis acute	0.81% 1/124 • up to 96 weeks
General disorders Accidental death	0.81% 1/124 • up to 96 weeks
Injury, poisoning and procedural complications Fall	0.81% 1/124 • up to 96 weeks
Injury, poisoning and procedural complications Subdural haematoma	0.81% 1/124 • up to 96 weeks
Surgical and medical procedures Gastrostomy	1.6% 2/124 • up to 96 weeks
Product Issues Device malfunction	0.81% 1/124 • up to 96 weeks
Infections and infestations Pneumonia	2.4% 3/124 • up to 96 weeks
Infections and infestations Pneumonia staphylococcal	0.81% 1/124 • up to 96 weeks
Infections and infestations Urinary tract infection	0.81% 1/124 • up to 96 weeks
Infections and infestations Clostridium difficile infection	0.81% 1/124 • up to 96 weeks
Infections and infestations COVID-19	2.4% 3/124 • up to 96 weeks
Metabolism and nutrition disorders Diabetic ketoacidosis	0.81% 1/124 • up to 96 weeks
Metabolism and nutrition disorders Decreased appetite	0.81% 1/124 • up to 96 weeks
Psychiatric disorders Assisted suicide	0.81% 1/124 • up to 96 weeks
Nervous system disorders Amyotrophic lateral sclerosis	4.8% 6/124 • up to 96 weeks
Nervous system disorders Subarachnoid haemorrhage	0.81% 1/124 • up to 96 weeks
Eye disorders Cataract	0.81% 1/124 • up to 96 weeks

Other adverse events

Other adverse events
Measure	MT-1186 n=124 participants at risk MT-1186 105mg (2 weeks On/Off)
Gastrointestinal disorders Constipation	12.9% 16/124 • up to 96 weeks
Gastrointestinal disorders Dysphagia	6.5% 8/124 • up to 96 weeks
Gastrointestinal disorders Nausea	5.6% 7/124 • up to 96 weeks
Infections and infestations COVID-19	8.9% 11/124 • up to 96 weeks
Infections and infestations Urinary tract infection	6.5% 8/124 • up to 96 weeks
Injury, poisoning and procedural complications Fall	16.1% 20/124 • up to 96 weeks
Musculoskeletal and connective tissue disorders Muscular weakness	14.5% 18/124 • up to 96 weeks
Nervous system disorders Dysarthria	5.6% 7/124 • up to 96 weeks
Psychiatric disorders Anxiety	6.5% 8/124 • up to 96 weeks
Psychiatric disorders Insomnia	7.3% 9/124 • up to 96 weeks
Respiratory, thoracic and mediastinal disorders Dyspnoea	10.5% 13/124 • up to 96 weeks

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