Amyotrophic Lateral Sclerosis (ALS)
Conditions
Brief summary
This is a Phase 3, international, multicenter, open-label, long-term extension study. The primary objective of this study is to evaluate the long-term safety and tolerability of oral edaravone in subjects with Amyotrophic Lateral Sclerosis (ALS) for up to 96 weeks.
Interventions
DRUGMT-1186
Treatment cycles with daily dosing for 10 days out of a 14-day period, followed by a 14 day drug free period up to 96 weeks of treatment or until the drug is commercially available in that country.
Sponsors
Shionogi
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Single Arm Open Label
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Subjects must provide signed and dated informed consent form (ICF) 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. 2. Subjects must be willing to cooperate and comply with all protocol restrictions and requirements. 3. Subjects who successfully completed Study MT-1186-A01.
Exclusion criteria
1. Subjects of childbearing potential unwilling to use a highly effective method of contraception from Visit 1 until 3 months after the last dose of study medication. 2. 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 C-SSRS at Visit 3. Subjects who are not eligible to continue in the study, as judged by the Investigator. 4. Subjects who are unable to take their medications orally or through a PEG/RIG tube.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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 | 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. - 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. |
| 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 | 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. |
Countries
Canada, France, Germany, Italy, Japan, United States
Contacts
STUDY_DIRECTORShionogi Clinical Trials Administrator Clinical Support Help Line
Shionogi
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| MT-1186 MT-1186 105mg (2 weeks On/Off) | 124 |
| Total | 124 |
Baseline characteristics
| Characteristic | MT-1186 |
|---|---|
| Age, Continuous | 59.0 years STANDARD_DEVIATION 10.1 |
| Age, Customized Adults (18-64 years) | 84 Participants |
| Age, Customized From 65 to 84 years | 40 Participants |
| Ethnicity (NIH/OMB) Hispanic or Latino | 2 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 118 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 4 Participants |
| Sex: Female, Male Female | 41 Participants |
| Sex: Female, Male Male | 83 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 19 / 124 |
| other Total, other adverse events | 70 / 124 |
| serious Total, serious adverse events | 52 / 124 |
Outcome results
Primary
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
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.
Time frame: up to 96 Weeks
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| MT-1186 | 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 |
| MT-1186 | 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 |
| MT-1186 | 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 |
| MT-1186 | 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 |
| MT-1186 | 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 |
| MT-1186 | 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
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
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.
Time frame: up to 96 Weeks
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| MT-1186 | 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 |
| MT-1186 | 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 |
| MT-1186 | 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 |
| MT-1186 | 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 |
| MT-1186 | 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 |
| MT-1186 | 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 |
Other Pre-specified
Change From Baseline of ALS Functional Rating Scale-Revised (ALSFRS-R) at 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.
Time frame: Baseline to Week 96
Other Pre-specified
Number of Events of Death, Tracheostomy, or Permanent Assisted Mechanical Ventilation
Time frame: up to 96 Weeks