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

Phase 3

Completed

• Conditions: Amyotrophic Lateral Sclerosis (ALS)
• Interventions: Drug: MCI-186; Drug: Placebo; Drug: MCI-186 in open label phase

• Registration Number: NCT01492686
• Lead Sponsor: Mitsubishi Tanabe Pharma Corporation

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2011-12
• Study First Submitted: 2011-12-11
• Study First Submitted QC: 2011-12-14
• Study First Posted: 2011-12-15
• Primary Completion: 2014-09
• Study Completion: 2014-10
• Results First Submitted: 2017-09-06
• Status Verified: 2018-06
• Results First Submitted QC: 2018-06-26
• Last Update Submitted: 2018-06-26
• Results First Posted: 2018-12-31
• Last Update Posted: 2018-12-31

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 137

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Arm 1	MCI-186	-
Arm 2	MCI-186 in open label phase	-
Arm 2	Placebo	-
Arm 1	MCI-186 in open label phase	-

Primary Outcome Measures

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

Secondary Outcome Measures

Name	Time	Method
Number of Participants With Death or a Specified State of Disease Progression	24 weeks	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.
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	baseline and 24 weeks	The Modified Norris Scale is a measure of movement disorder for patients with ALS. 0=worst; 102=best
Change From Baseline in ALS Assessment Questionnaire (40 Items) (ALSAQ40) in Full Analysis Set (FAS) Population at 24 Weeks	baseline and 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
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