Safety Study of Oral Edaravone Administered in Subjects With ALS

The objective of this study is to evaluate the long-term safety and tolerability of oral edaravone in subjects with Amyotrophic Lateral Sclerosis (ALS) over 24 and 48 weeks.

Study Overview

• Status: Completed
• Conditions: ALS
• Intervention / Treatment: Drug: MT-1186

• Study Type: Interventional
• Enrollment (Actual): 185
• Phase: Phase 3

Contacts and Locations

Study Locations

• Canada
  • Alberta
    • Calgary, Alberta, Canada, T2N 4Z6
      • Heritage Medical Research Clinic - University of Calgary
    • Edmonton, Alberta, Canada, T6G 2B7
      • University of Alberta - Walter C Mackenzie Health Sciences Centre (WCM)
  • Quebec
    • Greenfield Park, Quebec, Canada, J4V 2J2
      • Recherche Sepmus, Inc
    • Montreal, Quebec, Canada, H3A 2B4
      • Montreal Neurological Institute and Hospital
• France
  • Bordeaux, France, 33400
    • Centre Hospitalier Universitaire (CHU) de Bordeaux
  • Bron, France, 69677
    • Hopital Pierre Wertheimer - Hopital Neurologique
  • Nice cedex 1, France, 6001
    • CHU de NICE Hopital Pasteur 2 -Centre de Reference des Maladies -Neuromusculaires & SLA-Pole Neurosciences Cliniques
  • Alpes Maritimes
    • Nice cedex 3, Alpes Maritimes, France, 06202
      • CHU Nice-Hospital Archet I
  • Marcland
    • Limoges, Marcland, France, 87025
      • Centre Hospitalier Esquirol
• Germany
  • Hessen
    • Wiesbaden, Hessen, Germany, 65191
      • Deutsche Klinik fuer Diagnostik
  • Lower Saxony
    • Hannover, Lower Saxony, Germany, 30625
      • Medizinische Hochschule Hannover
• Italy
  • Milan, Italy, 20162
    • Fondazione Serena Onlus - Azienda Ospedaliera Niguarda Ca Granda - Centro Clinico Nemo (Neuro Muscular Omnicentre)
  • MI
    • Milano, MI, Italy, 20132
      • Ospedale San Raffaele (HSR) (Istituto Scientifico Universitario San Raffaele)
  • Piemonte
    • Turin, Piemonte, Italy, 10126
      • Universita degli Studi di Torino - Centro Regionale Esperto Per La Sclerosi Laterale Amiotrofica (CRESLA)
• Japan
  • Aichi
    • Nagakute-shi, Aichi, Japan, 480-1195
      • Aichi Medical University Hospital
  • Chiba
    • Chiba-shi, Chiba, Japan, 260-8712
      • National Hospital Organization Chiba-East-Hospital
  • Fukuoka
    • Fukuoka-city, Fukuoka, Japan, 819-8585
      • Murakami Karindoh Hospital
  • Fukushima
    • Fukushima-shi, Fukushima, Japan, 960-1295
      • Fukushima Medical University Hospital
  • Hokkaido
    • Sapporo-shi, Hokkaido, Japan, 063-0005
      • National Hospital Organization Hokkaido Medical Center
  • Ishikawa
    • Kanazawa-shi, Ishikawa, Japan, 920-0192
      • National Hospital Organization Iou National Hospital
  • Kagawa
    • Kita-gun, Kagawa, Japan, 761-0793
      • Kagawa University Hospital
  • Kanagawa
    • Sagamihara-city, Kanagawa, Japan, 252-0375
      • Kitasato University Hospital
    • Yokohama-shi, Kanagawa, Japan, 236-0004
      • Yokohama City University Hospital
  • Kumamoto
    • Koshi-shi, Kumamoto, Japan, 861-1196
      • National Hospital Organization Kumamoto Saishun Medical Center
  • Kyoto
    • Kyoto-shi, Kyoto, Japan, 616-8255
      • National Hospital Organization Utano Hospital
  • Miyagi
    • Sendai-city, Miyagi, Japan, 980-8574
      • Tohoku University Hospital
  • Niigata
    • Niigata-shi, Niigata, Japan, 951-8520
      • Niigata University Medical and Dental Hospital
  • Osaka
    • Toyonaka-shi, Osaka, Japan, 560-8552
      • National Hospital Organization Toneyama Medical Center
  • Shiga
    • Otsu-shi, Shiga, Japan, 520-2192
      • Shiga University of Medical Science Hospital
  • Shizuoka
    • Shizuoka-city, Shizuoka, Japan, 420-8688
      • National Hospital Organization Shizuoka Institute of Epilepsy and Neurological Disorders
  • Tokyo
    • Bunkyo-ku, Tokyo, Japan, 113-8431
      • Juntendo University Hospital
• United States
  • Arizona
    • Phoenix, Arizona, United States, 85013
      • St. Joseph's Hospital and Medical Center (SJHMC) - Barrow Neurological Institute (BNI) - The Gregory W. Fulton ALS and Neuromuscular Disease Center
    • Phoenix, Arizona, United States, 85028
      • Neuromuscular Research Center
  • Arkansas
    • Rogers, Arkansas, United States, 72758
      • Woodland Research Northwest
  • California
    • San Francisco, California, United States, 94115
      • Sutter Health
  • Colorado
    • Aurora, Colorado, United States, 80045
      • University of Colorado Anschutz Medical Campus
  • Florida
    • Gainesville, Florida, United States, 32610-3633
      • UF Health Cancer Center
  • Georgia
    • Atlanta, Georgia, United States, 30317-2819
      • Emory University - School of Medicine
  • Maryland
    • Baltimore, Maryland, United States, 21205
      • Johns Hopkins University
  • Michigan
    • Ann Arbor, Michigan, United States, 48109-5223
      • University of Michigan - ALS Center of Excellence
    • Northville, Michigan, United States, 48168-9493
      • University of Michigan Health System (UMHS) - Allergy Clinic - Northville Health Center Location
  • Minnesota
    • Duluth, Minnesota, United States, 55805
      • Essentia Institute of Rural Health
  • Nebraska
    • Lincoln, Nebraska, United States, 68506-2960
      • Neurology Associates, P.C - Lincoln
  • North Carolina
    • Winston-Salem, North Carolina, United States, 27157-0001
      • Wake Forest University Baptist Medical Center (WFUBMC) - The J. Paul Sticht Center on Aging and Rehabilitation
  • Pennsylvania
    • Hershey, Pennsylvania, United States, 17025
      • Penn State Hershey Children's Hospital
    • Philadelphia, Pennsylvania, United States, 19140
      • Lewis Katz School of Medicine at Temple University
    • Pittsburgh, Pennsylvania, United States, 15212
      • Alleghany General Hospital
  • Tennessee
    • Cordova, Tennessee, United States, 38018
      • Wesley Neurology Clinic, P.C.
  • Texas
    • Dallas, Texas, United States, 75214
      • Texas Neurology, PA
    • Houston, Texas, United States, 77030
      • Houston Methodist Neurological Institute
    • San Antonio, Texas, United States, 78229
      • UT Health Science Center San Antonio
  • Washington
    • Seattle, Washington, United States, 98195
      • University of Washington Medical Center
  • Wisconsin
    • Milwaukee, Wisconsin, United States, 53226
      • Medical College of Wisconsin

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years to 73 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Subjects will be male or female, ≥ 18 to 75 years of age at the time the ICF is signed
• Subjects will be diagnosed with Definite ALS, Probable ALS, Probable laboratory-supported ALS, or Possible ALS according to the El Escorial revised criteria for the diagnosis of ALS.
• Subjects will be living and functioning independently (eg, able to eat, excrete, ambulate independently without assistance of others). The use of supportive tools and adaptive utensil is allowed
• Subjects will have a baseline forced vital capacity percentage (%FVC) ≥ 70%.
• Subjects whose first symptom of ALS occurred within 3 years of the time of providing written informed consent.

Exclusion Criteria:

• Subjects who have the presence or history of any clinically significant disease (except ALS) that could interfere with the objectives of the study (the assessment of safety and efficacy) or the safety of the subject, as judged by the Investigator.
• Subjects of childbearing potential unwilling to use acceptable method of contraception from the screening visit until 3 months after the last dose of study medication. Subjects who are sexually active who do not agree to use contraception during the study period.
• Subjects who are female and pregnant (a positive pregnancy test) or lactating at the screening visit (Visit 1).
• Subjects who have a significant risk of suicidality. Subjects with any suicidal behavior or suicidal ideation of type 4 (active suicidal ideation with some intent to act, without a specific plan) or type 5 (active suicidal ideation with specific plan and intent) based on the Columbia-Suicide Severity Rating Scale (C-SSRS) within the 3 months before the screening visit.
• Subjects who have ALT or AST elevations greater than 2 times the ULN at screening.
• Subjects with a Glomerular Filtration Rate (GFR) <30 mL/Min Per 1.73 m2.
• Subjects with history of hypersensitivity to edaravone, any of the additives or inactive ingredients of edaravone, or sulfites.
• Subjects with hereditary fructose intolerance.
• Subjects who participated in another study and were administered an investigational product within 1 month or 5 half-lives of the investigational agent, whichever is longer before providing informed consent for the present study.
• Subjects who are unable to take their medications orally.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: MT-1186	Drug: MT-1186; An initial treatment cycle with daily dosing for 14 days, followed by a 14-day drug free period; Subsequent treatment cycles with daily dosing for 10 days out of a 14-day period, followed by a 14 day drug free period. Treatment cycles are every 4 weeks.; Other Names: Oral edaravone

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Number of Treatment Emergency Adverse Events	up to 48 Weeks
Number of Participants With Treatment Emergency Adverse Events	Up to 48 Weeks

Other Outcome Measures

Outcome Measure	Time Frame
Change in ALS Functional Rating Scale - Revised From Baseline	up to 48 Weeks
Time to Event (Death, Tracheostomy, and Permanent Assisted Mechanical Ventilation)	up to 48 Weeks

Collaborators and Investigators

• Sponsor: Mitsubishi Tanabe Pharma America Inc.
• Investigators: Study Director: Head of Medical Science,, Mitsubishi Tanabe Pharma America Inc.

Study record dates

Study Major Dates

• Study Start (Actual): November 18, 2019
• Primary Completion (Actual): October 7, 2021
• Study Completion (Actual): October 7, 2021

Study Registration Dates

• First Submitted: November 13, 2019
• First Submitted That Met QC Criteria: November 13, 2019
• First Posted (Actual): November 18, 2019

Study Record Updates

• Last Update Posted (Actual): May 15, 2023
• Last Update Submitted That Met QC Criteria: May 12, 2023
• Last Verified: May 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; Neuroprotective Agents; Protective Agents; Antioxidants; Free Radical Scavengers; Edaravone
• Other Study ID Numbers: MT-1186-A01

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No