ALS
Conditions
Brief summary
The objective of this study is to evaluate the long-term safety and tolerability of oral edaravone in subjects with Amyotrophic Lateral Sclerosis (ALS) over 24 and 48 weeks.
Interventions
DRUGMT-1186
* An initial treatment cycle with daily dosing for 14 days, followed by a 14-day drug free period * Subsequent treatment cycles with daily dosing for 10 days out of a 14-day period, followed by a 14 day drug free period. Treatment cycles are every 4 weeks.
Sponsors
Shionogi
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 75 Years
Healthy volunteers
No
Inclusion criteria
* Subjects will be male or female, ≥ 18 to 75 years of age at the time the ICF is signed * Subjects will be diagnosed with Definite ALS, Probable ALS, Probable laboratory-supported ALS, or Possible ALS according to the El Escorial revised criteria for the diagnosis of ALS. * Subjects will be living and functioning independently (eg, able to eat, excrete, ambulate independently without assistance of others). The use of supportive tools and adaptive utensil is allowed * Subjects will have a baseline forced vital capacity percentage (%FVC) ≥ 70%. * Subjects whose first symptom of ALS occurred within 3 years of the time of providing written informed consent.
Exclusion criteria
* Subjects who have the presence or history of any clinically significant disease (except ALS) that could interfere with the objectives of the study (the assessment of safety and efficacy) or the safety of the subject, as judged by the Investigator. * Subjects of childbearing potential unwilling to use acceptable method of contraception from the 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 and pregnant (a positive pregnancy test) or lactating at the screening visit (Visit 1). * Subjects who have a significant risk of suicidality. 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) within the 3 months before the screening visit. * Subjects who have ALT or AST elevations greater than 2 times the ULN at screening. * Subjects with a Glomerular Filtration Rate (GFR) \<30 mL/Min Per 1.73 m2. * Subjects with history of hypersensitivity to edaravone, any of the additives or inactive ingredients of edaravone, or sulfites. * Subjects with hereditary fructose intolerance. * Subjects who participated in another study and were administered an investigational product within 1 month or 5 half-lives of the investigational agent, whichever is longer before providing informed consent for the present study. * Subjects who are unable to take their medications orally.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Number of Treatment Emergency Adverse Events | up to 48 Weeks |
| Number of Participants With Treatment Emergency Adverse Events | Up to 48 Weeks |
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) | 185 |
| Total | 185 |
Baseline characteristics
| Characteristic | MT-1186 |
|---|---|
| Age, Continuous | 59.9 years STANDARD_DEVIATION 9.9 |
| Age, Customized 20 to 29 years | 1 Participants |
| Age, Customized 30 to 39 years | 6 Participants |
| Age, Customized 40 to 49 years | 19 Participants |
| Age, Customized 50 to 59 years | 60 Participants |
| Age, Customized 60 to 69 years | 63 Participants |
| Age, Customized >=70 years | 36 Participants |
| Ethnicity (NIH/OMB) Hispanic or Latino | 3 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 177 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 5 Participants |
| Sex: Female, Male Female | 66 Participants |
| Sex: Female, Male Male | 119 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 12 / 185 |
| other Total, other adverse events | 123 / 185 |
| serious Total, serious adverse events | 48 / 185 |
Outcome results
Primary
Number of Participants With Treatment Emergency Adverse Events
Time frame: Up to 48 Weeks
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| MT-1186 | Number of Participants With Treatment Emergency Adverse Events | 175 Participants |
Primary
Number of Treatment Emergency Adverse Events
Time frame: up to 48 Weeks
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| MT-1186 | Number of Treatment Emergency Adverse Events | 961 events |
Other Pre-specified
Change in ALS Functional Rating Scale - Revised From Baseline
Time frame: up to 48 Weeks
Other Pre-specified
Time to Event (Death, Tracheostomy, and Permanent Assisted Mechanical Ventilation)
Time frame: up to 48 Weeks