ALS
Conditions
Brief summary
The purpose of this study is to evaluate the safety of oral edaravone at a dose of 105 mg administered once daily for 10 days out of a 14-day period, followed by a 14-day drug-free period. This study will be continued until the earlier date when oral edaravone is commercially available at each site in Japan or August 2023.
Interventions
DRUGMT-1186
Suspension
Sponsors
Shionogi
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Subjects must provide a 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 * Subjects must be willing to cooperate and comply with all protocol restrictions and requirements. * Subjects who successfully complete Week 96 of Study MT-1186-A03 or Week 48 of Study MT-1186-A04 and have been compliant with study drug (80-120%).
Exclusion criteria
* Subjects of childbearing potential unwilling to use a highly effective method of contraception from the Visit #1 until 3 months after the last dose of study medication. * 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 Week 96 of the A03 study or at Week 48 of the A04 study. * Subjects who are not eligible to continue in the study, as judged by the Investigator.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Patients With AEs and Adverse Drug Reactions | up to 8 months | Adverse Event (AE) is classified as treatment emergent if it newly occurred after the first dose of investigational product or if a pre-dose event increases in severity following the first dose of investigational product. Adverse Drug Reaction (ADR) is a noxious and unintended response to a medicinal product that occurs at doses normally used for prophylaxis, diagnosis, or therapy of disease. |
Countries
Japan
Contacts
STUDY_DIRECTORShionogi Clinical Trials Administrator Clinical Support Help Line
Shionogi
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| MT-1186 MT-1186 105mg (2weeks On/Off) | 15 |
| Total | 15 |
Baseline characteristics
| Characteristic | MT-1186 |
|---|---|
| Age, Continuous | 59.3 years STANDARD_DEVIATION 13.8 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 15 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race/Ethnicity, Customized American Indian or Alaska Native | 0 Participants |
| Race/Ethnicity, Customized Asian - Japanese | 15 Participants |
| Race/Ethnicity, Customized Asian - Not Japanese | 0 Participants |
| Race/Ethnicity, Customized Black or African American | 0 Participants |
| Race/Ethnicity, Customized Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race/Ethnicity, Customized White | 0 Participants |
| Sex: Female, Male Female | 6 Participants |
| Sex: Female, Male Male | 9 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 1 / 15 |
| other Total, other adverse events | 5 / 15 |
| serious Total, serious adverse events | 2 / 15 |
Outcome results
Primary
Number of Patients With AEs and Adverse Drug Reactions
Adverse Event (AE) is classified as treatment emergent if it newly occurred after the first dose of investigational product or if a pre-dose event increases in severity following the first dose of investigational product. Adverse Drug Reaction (ADR) is a noxious and unintended response to a medicinal product that occurs at doses normally used for prophylaxis, diagnosis, or therapy of disease.
Time frame: up to 8 months
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| MT-1186 | Number of Patients With AEs and Adverse Drug Reactions | Number of patients with AEs | 6 Participants |
| MT-1186 | Number of Patients With AEs and Adverse Drug Reactions | Number of patients with adverse drug reactions | 0 Participants |
Other Pre-specified
ALSFRS-R Total Score
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: up to 10 months or 31/Aug/2023
Other Pre-specified
Number of Death, Tracheostomy, or Permanent Assisted Mechanical Ventilation (≥23 Hours/Day)
Time frame: up to 10 months or 31/Aug/2023