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

Shionogi137 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

137

Conditions

Amyotrophic Lateral Sclerosis (ALS)

Interventions

MCI-186DRUG

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).

PlaceboDRUG

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).

MCI-186 in open label phaseDRUG

All patients after the double blind phase may receive MCI-186 in 7th until 12th treatment - observation cycles at the patients' will.

Eligibility

Sex: ALLMin age: 20 YearsMax age: 75 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Patients whose conditions are defined as "definite ALS"or "probable ALS"diagnostic 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.

Locations (1)

Unnamed facility

Osaka, Japan

Outcomes

Primary Outcomes

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

Time frame: baseline and 24 weeks

Secondary Outcomes

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.

Time frame: 24 weeks

Change From Baseline in % Forced Vital Capacity (%FVC) in Full Analysis Set (FAS) Population at 24 Weeks

Time frame: 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

Time frame: 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

Time frame: baseline and 24 weeks

Percentage of Participants With Adverse Events

Time frame: 24 weeks

Percentage of Participants With Adverse Drug Reactions

Time frame: 24 weeks

Data from ClinicalTrials.gov

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