Efficacy and Safety Study of MCI-186 for Treatment of Amyotrophic Lateral Sclerosis (ALS) Who Met Severity Classification III

Study Overview
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Brief Summary
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The primary objective of this study is to evaluate the efficacy of 60mg of MCI-186 via intravenous drip once a day in patients with ALS whose severity is classified as grade III, 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 who met severity classification III.

Official Title
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An Exploratory Study of MCI-186 for Treatment of Amyotrophic Lateral Sclerosis (Severity Classification III) 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 cannot take at least one action of eating a meal, excreting, or moving with oneself alone, and need assistance in everyday life. 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, sever heart disease, sever 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 been administered other investigational products 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> | 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> | 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 | No primary endpoint was used, because various exploratory analyses were performed. 0=worst; 48=best | baseline and 24 weeks | 
| Death or a Specified State of Disease Progression | No primary endpoint was used, because various exploratory analyses were performed. 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 | No primary endpoint was used, because various exploratory analyses were performed. | baseline and 24 weeks | 
| Percentage of Participants With Adverse Events | No primary endpoint was used, because various exploratory analyses were performed. | 24 weeks | 
| Percentage of Participants With Adverse Drug Reactions | No primary endpoint was used, because various exploratory analyses were performed. | 24 weeks | 
| The Percentage of Participants With an Abnormal Change in Laboratory Tests That Occurred in More Than Two Patients | No primary endpoint was used, because various exploratory analyses were performed. | 24 weeks | 
| Percentage of Participants With Abnormal Changes in Sensory Examinations | No primary endpoint was used, because various exploratory analyses were performed. | 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