Amyotrophic Lateral Sclerosis (ALS) MedDRA version: 21.1 Level: PT Classification code 10002026 Term: Amyotrophic lateral sclerosis System Organ Class: 10029205 - Nervous system disorders
Conditions
Interventions
Product Name: Edaravone
Product Code: MT-1186
Pharmaceutical Form: Oral suspension
INN or Proposed INN: EDARAVONE
CAS Number: 89-25-8
Current Sponsor code: MT-1186
Concentration unit: mg/ml milligram(
Sponsors
Mitsubishi Tanabe Pharma Development America, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Subjects must provide signed and dated informed consent form (ICF) to participate in the study. Subjects must be able to (in the judgement of the Investigator) 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. 2. Subjects will be male or female, = 18 to 75 years of age at the time the ICF is signed. 3. 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. 4. 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. 5. Subjects will have a baseline forced vital capacity percentage (%FVC) = 70%. 6. Subjects whose first symptom of ALS has occurred within 3 years at the time of providing written informed consent. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 75 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 75
Exclusion criteria
Exclusion criteria: Exclusions Related to General Health or Concomitant Conditions: 1. Subjects undergoing treatment for a malignancy or those with a pending biopsy result. 2. 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. 3. Subjects of childbearing potential unwilling to use an acceptable method of contraception from the screening visit until 3 months after the last dose of study medication. Subjects who are sexually active and who do not agree to use contraception during the study period. 4. Subjects who are female and pregnant (a positive pregnancy test) or lactating at the screening visit (Visit 1). 5. 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 Columbia–Suicide Severity Rating Scale (C-SSRS) within the 3 months before the screening visit. 6. Subjects who have alanine aminotransferase (ALT) or aspartate aminotransferase (AST) elevations greater than 2 times the upper limit of normal (ULN) at screening. 7. Subjects with a glomerular filtration rate (GFR) <30 mL/min per 1.73 m2 at screening. Exclusions Related to Medications: 8. Subjects with history of hypersensitivity to edaravone, any of the additives or inactive ingredients of edaravone, or sulfites. 9. Subjects with hereditary fructose intolerance. 10. 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. 11. Subjects who are unable to take their medications orally.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Primary end point(s): The primary safety endpoints are to evaluate the safety and tolerability of oral edaravone and include the following safety assessments: - Adverse events (AEs), adverse drug reactions (ADRs), and treatment-emergent adverse events ([TEAEs] eg, grade, incidence and severity); - Physical examination; - Body weight; - 12-lead electrocardiogram (ECG) parameters; - Vital signs (heart rate, sitting systolic and diastolic blood pressure, and axillary, oral, or tympanic body temperature); - Laboratory safety assessments (eg, hematology, chemistry, and urinalysis); - Unsteadiness and sensory evaluation (eg, assessment of unsteadiness and peripheral sensation will be evaluated by assessment of vibratory sensation with a tuning fork applied to the lateral side of the right and left ankles); - Columbia–Suicide Severity Rating Scale (C-SSRS); - Forced vital capacity percentage (%FVC). ;Timepoint(s) of evaluation of this end point: - Visit 1- 15 - Visit 1, 2, 4, 6, 9, 12 and 15 - Visit 1, 2, 4, 6, 9, 12 and 15 - Visit 1, 2, 9 and 15 - Visit 1, 2, 4, 6, 9, 12 and 15 - Visit 1, 2, 4, 6, 9, 12 and 15 - Visit 1, 2, 4, 6, 9, 12 and 15 - Visit 1, 6, 9 and 15 - Visit 1, 2, 4, 6, 9, 12 and 15;Main Objective: To evaluate the long-term safety and tolerability of oral edaravone in subjects with Amyotrophic Lateral Sclerosis (ALS) over 24 and 48 weeks. ;Secondary Objective: Not applicable. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Not applicable.;Timepoint(s) of evaluation of this end point: Not applicable. | — |
Countries
Canada, France, Germany, Italy, Japan, United States
Contacts
Public ContactDaniel Selness
Mitsubishi Tanabe Pharma Development America, Inc.
Outcome results
None listed