Safety Study of Oral Edaravone Administered in Subjects With ALS

NCT ID: NCT04165824

Last Updated: 2025-12-30

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

185 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-11-18

Study Completion Date

2021-10-07

Brief Summary

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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.

Detailed Description

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Conditions

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ALS

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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MT-1186

Group Type EXPERIMENTAL

MT-1186

Intervention Type DRUG

* 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.

Interventions

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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.

Intervention Type DRUG

Other Intervention Names

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Oral edaravone

Eligibility Criteria

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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.
Minimum Eligible Age

18 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Tanabe Pharma America, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Head of Medical Science,

Role: STUDY_DIRECTOR

Tanabe Pharma America, Inc.

Locations

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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

Site Status

Neuromuscular Research Center

Phoenix, Arizona, United States

Site Status

Woodland Research Northwest

Rogers, Arkansas, United States

Site Status

Sutter Health

San Francisco, California, United States

Site Status

University of Colorado Anschutz Medical Campus

Aurora, Colorado, United States

Site Status

UF Health Cancer Center

Gainesville, Florida, United States

Site Status

Emory University - School of Medicine

Atlanta, Georgia, United States

Site Status

Johns Hopkins University

Baltimore, Maryland, United States

Site Status

University of Michigan - ALS Center of Excellence

Ann Arbor, Michigan, United States

Site Status

University of Michigan Health System (UMHS) - Allergy Clinic - Northville Health Center Location

Northville, Michigan, United States

Site Status

Essentia Institute of Rural Health

Duluth, Minnesota, United States

Site Status

Neurology Associates, P.C - Lincoln

Lincoln, Nebraska, United States

Site Status

Wake Forest University Baptist Medical Center (WFUBMC) - The J. Paul Sticht Center on Aging and Rehabilitation

Winston-Salem, North Carolina, United States

Site Status

Penn State Hershey Children's Hospital

Hershey, Pennsylvania, United States

Site Status

Lewis Katz School of Medicine at Temple University

Philadelphia, Pennsylvania, United States

Site Status

Alleghany General Hospital

Pittsburgh, Pennsylvania, United States

Site Status

Wesley Neurology Clinic, P.C.

Cordova, Tennessee, United States

Site Status

Texas Neurology, PA

Dallas, Texas, United States

Site Status

Houston Methodist Neurological Institute

Houston, Texas, United States

Site Status

UT Health Science Center San Antonio

San Antonio, Texas, United States

Site Status

University of Washington Medical Center

Seattle, Washington, United States

Site Status

Medical College of Wisconsin

Milwaukee, Wisconsin, United States

Site Status

Heritage Medical Research Clinic - University Of Calgary

Calgary, Alberta, Canada

Site Status

University of Alberta - Walter C Mackenzie Health Sciences Centre (WCM)

Edmonton, Alberta, Canada

Site Status

Recherche Sepmus, Inc

Greenfield Park, Quebec, Canada

Site Status

Montreal Neurological Institute and Hospital

Montreal, Quebec, Canada

Site Status

CHU Nice-Hospital Archet I

Nice, Alpes Maritimes, France

Site Status

Centre Hospitalier Esquirol

Limoges, Marcland, France

Site Status

Centre Hospitalier Universitaire (CHU) de Bordeaux

Bordeaux, , France

Site Status

Hopital Pierre Wertheimer - Hopital Neurologique

Bron, , France

Site Status

CHU de NICE Hopital Pasteur 2 -Centre de Reference des Maladies -Neuromusculaires & SLA-Pole Neurosciences Cliniques

Nice, , France

Site Status

Deutsche Klinik fuer Diagnostik

Wiesbaden, Hesse, Germany

Site Status

Medizinische Hochschule Hannover

Hanover, Lower Saxony, Germany

Site Status

Ospedale San Raffaele (HSR) (Istituto Scientifico Universitario San Raffaele)

Milan, MI, Italy

Site Status

Universita degli Studi di Torino - Centro Regionale Esperto Per La Sclerosi Laterale Amiotrofica (CRESLA)

Turin, Piedmont, Italy

Site Status

Fondazione Serena Onlus - Azienda Ospedaliera Niguarda Ca Granda - Centro Clinico Nemo (Neuro Muscular Omnicentre)

Milan, , Italy

Site Status

Aichi Medical University Hospital

Nagakute-shi, Aichi-ken, Japan

Site Status

National Hospital Organization Chiba-East-Hospital

Chiba, Chiba, Japan

Site Status

Murakami Karindoh Hospital

Fukuoka, Fukuoka, Japan

Site Status

Fukushima Medical University Hospital

Fukushima, Fukushima, Japan

Site Status

National Hospital Organization Hokkaido Medical Center

Sapporo, Hokkaido, Japan

Site Status

National Hospital Organization Iou National Hospital

Kanazawa, Ishikawa-ken, Japan

Site Status

Kagawa University Hospital

Kita-gun, Kagawa-ken, Japan

Site Status

Kitasato University Hospital

Sagamihara, Kanagawa, Japan

Site Status

Yokohama City University Hospital

Yokohama, Kanagawa, Japan

Site Status

National Hospital Organization Kumamoto Saishun Medical Center

Koshi-shi, Kumamoto, Japan

Site Status

National Hospital Organization Utano Hospital

Kyoto, Kyoto, Japan

Site Status

Tohoku University Hospital

Sendai, Miyagi, Japan

Site Status

Niigata University Medical And Dental Hospital

Niigata, Niigata, Japan

Site Status

National Hospital Organization Toneyama Medical Center

Toyonaka-shi, Osaka, Japan

Site Status

Shiga University of Medical Science Hospital

Ōtsu, Shiga, Japan

Site Status

National Hospital Organization Shizuoka Institute of Epilepsy and Neurological Disorders

Shizuoka, Shizuoka, Japan

Site Status

Juntendo University Hospital

Bunkyo-ku, Tokyo, Japan

Site Status

Countries

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United States Canada France Germany Italy Japan

References

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Genge A, Pattee GL, Sobue G, Aoki M, Yoshino H, Couratier P, Lunetta C, Petri S, Selness D, Bidani S, Hirai M, Sakata T, Salah A, Apple S, Wamil A, Kalin A, Jackson CE. Oral edaravone demonstrated a favorable safety profile in patients with amyotrophic lateral sclerosis after 48 weeks of treatment. Muscle Nerve. 2023 Feb;67(2):124-129. doi: 10.1002/mus.27768. Epub 2022 Dec 28.

Reference Type RESULT
PMID: 36504406 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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MT-1186-A01

Identifier Type: -

Identifier Source: org_study_id