Phase 3 Study of MCI-186 for Treatment of Amyotrophic Lateral Sclerosis

Study Overview
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Brief Summary
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The primary objective of the study is to confirm the efficacy of 60 mg of MCI-186 via intravenous drip infusion once a day in the patients with ALS based on the changes in the revised ALS functional rating scale (ALSFRS-R) scores after 24 weeks administration in double-blind, placebo-controlled manner. The study is also to examine the safety of MCI-186 to the ALS patients.

Official Title
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Efficacy and Safety Study of MCI-186 for Treatment of the Patients With Amyotrophic Lateral Sclerosis (ALS) 2

Conditions
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Amyotrophic Lateral Sclerosis (ALS)

Intervention / Treatment
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* Drug: MCI-186
* Drug: Placebo
* Drug: MCI-186 in open label phase


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients whose conditions are defined as definite ALSor probable ALSdiagnostic criteria El Escorial and revised Airlie House. Patients who can eat a meal, excrete, or move with oneself alone, and do not need assistance in everyday life. Patients of less than 2 years after the onset of ALS. Patients whose progress of the condition during 12 weeks before administration meet other requirements. Exclusion Criteria: Patients with such complications as Parkinson's disease, schizophrenia, dementia, renal failure, or other severe complication, and patients who have the anamnesis of hypersensitivity to edaravone. Pregnant, lactating, and probably pregnant patients, and patients who want to become pregnant, and patients who can not agree to contraception. Patients who have participated in other trials within 12 weeks before consent, or who are participating in other clinical trials at present. In addition to the above exclusion criteria, patients judged to be inadequate to participate in this study by their physician.

Ages Eligible for Study
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Minimum Age: 20 Years
Maximum Age: 75 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Arm 1<br> | Drug: MCI-186<br>* Two ampoules (60 mg) of MCI-186 injection are intravenously administered once a day, for successive 14 days, followed by 14 days observation period (first cycle). The following treatment (10 days' administration during 14 days) - observation (14 days) cycle is repeated five times (2nd-6th cycles).<br>Drug: MCI-186 in open label phase<br>* All patients after the double blind phase may receive MCI-186 in 7th until 12th treatment - observation cycles at the patients' will.<br>|
| Placebo Comparator: Arm 2<br> | Drug: Placebo<br>* Two ampoules of placebo injection are intravenously administered once a day, for successive 14 days, followed by 14 days observation period (first cycle). The following treatment (10 days' administration during 14 days) - observation (14 days) cycle is repeated five times (2nd-6th cycles).<br>Drug: MCI-186 in open label phase<br>* All patients after the double blind phase may receive MCI-186 in 7th until 12th treatment - observation cycles at the patients' will.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change From Baseline in Revised ALS Functional Rating Scale (ALSFRS-R) Score in Full Analysis Set (FAS) Population at 24 Weeks | 0=worst; 48=best | baseline and 24 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants With Death or a Specified State of Disease Progression | Any of death, disability of independent ambulation, loss of upper limbs function, tracheotomy, use of respirator, use of tube feeding and loss of useful speech was defined as an event. | 24 weeks | 
| Change From Baseline in % Forced Vital Capacity (%FVC) in Full Analysis Set (FAS) Population at 24 Weeks |  | baseline and 24 weeks | 
| Change From Baseline in Modified Norris Scale Score in Full Analysis Set (FAS) Population at 24 Weeks | The Modified Norris Scale is a measure of movement disorder for patients with ALS. 0=worst; 102=best | baseline and 24 weeks | 
| Change From Baseline in ALS Assessment Questionnaire (40 Items) (ALSAQ40) in Full Analysis Set (FAS) Population at 24 Weeks | The ALSAQ40 score is a measure of QoL for patients with ALS. The ALSAQ40 evaluates domains that include physical mobility, Activities of daily living (ADL) and independence, eating and drinking, communication, and emotional reactions. 200=worst; 40=best | baseline and 24 weeks | 
| Percentage of Participants With Adverse Events |  | 24 weeks | 
| Percentage of Participants With Adverse Drug Reactions |  | 24 weeks | 
| Laboratory Tests Percentage of Participants With Adverse Events by System Organ Class (SOC) of Investigations (PT, MedDRA Ver. 17.0) |  | 24 weeks | 
| Percentage of Participants With Abnormal Values in Sensory Examinations |  | baseline and 24 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Amyotrophic Lateral Sclerosis (ALS)