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

Purpose

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.

Condition	Intervention	Phase
Amyotrophic Lateral Sclerosis (ALS)	Drug: MCI-186 Drug: Placebo Drug: MCI-186 in open label phase	Phase 3


Study Type:	Interventional
Study Design:	Allocation: Randomized Endpoint Classification: Efficacy Study Intervention Model: Parallel Assignment Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor) Primary Purpose: Treatment
Official Title:	Efficacy and Safety Study of MCI-186 for Treatment of the Patients With Amyotrophic Lateral Sclerosis (ALS) 2

Resource links provided by NLM:

Genetics Home Reference related topics: amyotrophic lateral sclerosis

MedlinePlus related topics: Amyotrophic Lateral Sclerosis

Genetic and Rare Diseases Information Center resources: Amyotrophic Lateral Sclerosis

U.S. FDA Resources

Further study details as provided by Mitsubishi Tanabe Pharma Corporation:

Primary Outcome Measures:

Revised ALS functional rating scale (ALSFRS-R) scores [ Time Frame: 24 weeks ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

Time to death from date of randomization [ Time Frame: 24 weeks ] [ Designated as safety issue: No ]
Time to a certain state from date of randomization [ Time Frame: 24 weeks ] [ Designated as safety issue: No ]
inability to walk alone; failure of arm function; tracheostomy; respirator installation; tubal feeding replenishment
% Forced Vital Capacity (%FVC) [ Time Frame: 24 weeks ] [ Designated as safety issue: No ]
Modified Norris scale score [ Time Frame: 24 weeks ] [ Designated as safety issue: No ]
Amyotrophic Lateral Sclerosis Assessment Questionnaire (ALSAQ-40 score) [ Time Frame: 24 weeks ] [ Designated as safety issue: No ]
Adverse events, adverse drug reactions, laboratory test and sensory examinations [ Time Frame: 24 weeks ] [ Designated as safety issue: Yes ]


Enrollment:	137
Study Start Date:	December 2011
Study Completion Date:	October 2014
Primary Completion Date:	September 2014 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Experimental: Arm 1	Drug: MCI-186 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). Drug: MCI-186 in open label phase All patients after the double blind phase may receive MCI-186 in 7th until 12th treatment - observation cycles at the patients' will.
Placebo Comparator: Arm 2	Drug: Placebo 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). Drug: MCI-186 in open label phase All patients after the double blind phase may receive MCI-186 in 7th until 12th treatment - observation cycles at the patients' will.

Arms

Assigned Interventions

Experimental: Arm 1

Drug: MCI-186

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).

Drug: MCI-186 in open label phase

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

Placebo Comparator: Arm 2

Drug: Placebo

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).

Drug: MCI-186 in open label phase

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

Eligibility


Ages Eligible for Study:	20 Years to 75 Years (Adult, Senior)
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Patients whose conditions are defined as "definite ALS"or "probable ALS"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 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.

Contacts and Locations

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01492686

Locations


Japan

Osaka, Japan

Sponsors and Collaborators

Mitsubishi Tanabe Pharma Corporation

More Information


Responsible Party:	Mitsubishi Tanabe Pharma Corporation
ClinicalTrials.gov Identifier:	NCT01492686 History of Changes
Other Study ID Numbers:	MCI186-19
Study First Received:	December 11, 2011
Last Updated:	June 18, 2015
Health Authority:	Japan: Ministry of Health, Labor and Welfare

Keywords provided by Mitsubishi Tanabe Pharma Corporation:


Amyotrophic Lateral Sclerosis (ALS)

Additional relevant MeSH terms:


Sclerosis Motor Neuron Disease Amyotrophic Lateral Sclerosis Pathologic Processes Neurodegenerative Diseases Nervous System Diseases Neuromuscular Diseases Spinal Cord Diseases Central Nervous System Diseases TDP-43 Proteinopathies Proteostasis Deficiencies Metabolic Diseases Phenylmethylpyrazolone	Antipyrine Free Radical Scavengers Antioxidants Molecular Mechanisms of Pharmacological Action Protective Agents Physiological Effects of Drugs Anti-Inflammatory Agents, Non-Steroidal Analgesics, Non-Narcotic Analgesics Sensory System Agents Peripheral Nervous System Agents Anti-Inflammatory Agents Antirheumatic Agents

ClinicalTrials.gov processed this record on September 29, 2016