Efficacy and Safety Study of MCI-186 for Treatment of Amyotrophic Lateral Sclerosis (ALS)

Study Overview
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Brief Summary
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The primary objective of this study is to confirm the efficacy of 60 mg of MCI-186 via intravenous drip once a day in 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. And in addition, this study will be performed to examine the safety of MCI-186 to ALS patients.

Official Title
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A Confirmatory Study of MCI-186 for Treatment of Amyotrophic Lateral Sclerosis in Double-blind, Parallel-group, Placebo-controlled Manner.

Conditions
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Amyotrophic Lateral Sclerosis (ALS)

Intervention / Treatment
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* Drug: MCI-186
* Drug: Placebo of MCI-186


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients who are defined as definite ALS, probable ALS or probable-laboratory-supported ALS, met diagnostic criteria revised EL Escorial for 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 3 years after the onset of ALS. Patients whose progress of the condition during 12 weeks before administration meet other requirements. Exclusion Criteria: Patients judged to be inadequate to participate in this study by their physician, because those patients' general condition deteriorated to the point that they need to be hospitalized for severe hepatic disease, severe heart disease, severe renal disease and so on, or they need to be administered antibiotics to infection. Patients who complain the difficulty in breathing caused by deteriorating the respiratory function. 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: 1<br>MCI-186 | 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). Then treatment (10 days' administration during 14 days) - observation (14 days) cycle is repeated five times (2nd-6th cycles).<br>* Other names: Radicut;|
| Placebo Comparator: 2<br>Placebo of MCI-186 | Drug: Placebo of MCI-186<br>* Two ampoules of placebo injection are intravenously administered once a day, for successive 14 days, followed by 14 days observation period (first cycle). Then treatment (10 days' administration during 14 days) - observation (14 days) cycle is repeated five times (2nd-6th cycles).<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 | ALSFRS-R Score: 0=worst; 48=best | baseline and 24 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Death or a Specified State of Disease Progression | Any of death, disability of independent ambulation, loss of upper arm function, tracheotomy, use of respirator, and use of tube feeding 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. Worst=0, Best=102 | 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, ADL and independence, eating and drinking, communication, and emotional reactions. Worst=200, Best=40 | baseline and 24 weeks | 
| Percentage of Participants With Adverse Events |  | 24 weeks | 
| Percentage of Participants With Adverse Drug Reactions |  | 24 weeks | 
| Percentage of Participants With Laboratory Tests for Which the Incidence of Abnormal Changes Was 5% or Higher in Either Group |  | 24 weeks | 
| Percentage of Participants With Abnormal Changes in Sensory Examinations |  | 24 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Amyotrophic lateral sclerosis, free radical scavenger