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ALSPhase 3NCT04569084

Efficacy and Safety Study of Oral Edaravone Administered in Subjects With ALS

A Phase 3 study testing MT-1186 in people with ALS, run by Shionogi at 95 sites in 7 countries.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

To evaluate and compare the efficacy of two dosing regimens of oral edaravone in subjects with amyotrophic lateral sclerosis (ALS) based on the change in ALS Functional Rating Scale- Revised (ALSFRS-R) score from baseline up to Week 48: Read more about this study

Who is it for?
Ages 18 to 75 years
Conditions the study is for
ALS
What is being tested?
MT-1186
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
384 participants
Where
95 sitesin 7 countries
Study dates
13 Nov 2020to 29 Sep 2023
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 75 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Subjects must provide a signed and dated informed consent form (ICF) to participate in the study. Subjects must be able (in the judgment of the Investigator) to understand the nature of the study and all risks involved with participation in the study. Subjects must be willing to cooperate and comply with all protocol restrictions and requirements.
  • 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 or Probable ALS according to the El Escorial revised criteria for the diagnosis of ALS.
  • Subjects with a baseline score ≥ 2 points on each individual item of the ALSFRS- R at screening and baseline visits.
  • Subjects have a screening and baseline %forced vital capacity (FVC) ≥ 70%.
  • Subjects with 1- to 4-point decline for 8 weeks (±7 days) in ALSFRS-R total score between screening and baseline visits.
  • Subjects whose first symptom of ALS has occurred within 2 years of providing written informed consent.

Who cannot join

  • Exclusions Related to Primary Diagnosis
  • Subjects with a history of spinal surgery after the onset of ALS, such as surgery for cervical spondylosis or a herniated disc, or plans for such surgery during the study period.
  • Exclusions Related to Other Neurological Disorders (including, but not limited to the following)
  • Subjects with the possibility that the current symptoms may be symptoms of a disease requiring differential diagnosis, such as cervical spondylosis and multifocal motor neuropathy, cannot be ruled out.
  • Exclusions Related to General Health or Concomitant Conditions
  • Subjects undergoing treatment for a malignancy.
Show all 20Show fewer
  • Subjects with a complication that could have a significant effect on efficacy evaluations, such as Parkinson's disease or syndrome, schizophrenia, bipolar disorder, and dementia.
  • Subjects who have the presence or history of any clinically significant (CS) 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 who are female, of childbearing potential, and pregnant (a positive pregnancy test) or lactating at the screening visit (Visit 1).
  • 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 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 alanine aminotransferase (ALT) or aspartate aminotransferase (AST) elevations greater than 2 times the upper limit of normal (ULN) at screening.
  • Subjects with a Glomerular Filtration Rate (GFR) < 30 mL/Min Per 1.73 m2 at screening, using the Larsson Equation.
  • Exclusions Related to Medications
  • Subjects with history of hypersensitivity to edaravone, any of the additives or inactive ingredients of edaravone, or sulfites.
  • Subjects with hereditary problems of fructose intolerance (eg, fructose, sucrose, invert sugar, and sorbitol).
  • 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 have received any previous treatment with edaravone.
  • Subjects who have received stem cell therapy.
  • Subjects who are unable to take their medications orally at baseline (Visit 2).

Trial locations and contacts

95 sites in 7 countries.

95 sites in 7 countries

United States34 sites

  • Dent Neurologic Institute

    Amherst, New York, 14226

  • Emory University - School of Medicine

    Atlanta, Georgia, 30322

  • University of Colorado Anschutz Medical Campus

    Aurora, Colorado, 80045

  • Austin Neuromuscular Center

    Austin, Texas, 78756

  • Johns Hopkins University

    Baltimore, Maryland, 21205

  • Lahey Hospital

    Burlington, Massachusetts, 01805

  • The University of Vermont (UVM) and UVM Medical Center National ALS Center of Excellence

    Burlington, Vermont, 05401-3456

  • Northwestern University Feinberg School of Medicine

    Chicago, Illinois, 60611-2605

  • Wesley Neurology Clinic, P.C.

    Cordova, Tennessee, 38018

  • UF Health Cancer Center

    Gainesville, Florida, 32610-3633

  • Penn State Hershey Medical Center

    Hershey, Pennsylvania, 17033

  • Nerve And Muscle Center Of Texas

    Houston, Texas, 77019

  • Mayo Clinic

    Jacksonville, Florida, 32224

  • Ochsner Center for Primary Care and Wellness

    Jefferson, Louisiana, 70121

  • UCSD Medical Center

    La Jolla, California, 92037-0897

  • Las Vegas Clinic

    Las Vegas, Nevada, 89145

  • Neurology Associates, P.C. - Lincoln

    Lincoln, Nebraska, 68506-2960

  • Loma Linda University Health Care - Department of Neurology

    Loma Linda, California, 92354

  • University California Los Angeles Medical Center (UCLA)

    Los Angeles, California, 90095

  • Medical College of Wisconsin

    Milwaukee, Wisconsin, 53226

  • West Virginia University School of Medicine (WVUSoM) - Movement Disorder Clinic

    Morgantown, West Virginia, 26506-9180

  • University of California Irvine (UCI) Health - Women's Healthcare Center

    Orange, California, 92868

  • Thomas Jefferson University, Jefferson Weinberg ALS Center

    Philadelphia, Pennsylvania, 19107

  • Lewis Katz School of Medicine at Temple University

    Philadelphia, Pennsylvania, 19140

  • St. Joseph's Hospital and Medical Center (SJHMC)

    Phoenix, Arizona, 85013

  • University Of Pittsburgh Medical Center

    Pittsburgh, Pennsylvania, 15261

  • Mayo Clinic

    Rochester, Minnesota, 55905

  • Woodland Research Northwest

    Rogers, Arkansas, 72758

  • HonorHealth Neurology

    Scottsdale, Arizona, 85251

  • University of Washington Medical Center

    Seattle, Washington, 98195

  • St. Luke's Rehabilitation Institute

    Spokane, Washington, 99202

  • SUNY Upstate Medical University

    Syracuse, New York, 13210

  • University of South Florida (USF) - Carol and Frank Morsani Center for Advanced Health Care (CAHC)

    Tampa, Florida, 33616

  • Sentara Neurology Specialists

    Virginia Beach, Virginia, 23456

Other trials in United States

Japan26 sites

  • National Hospital Organization Shizuoka Institute of Epilepsy and Neurological Disorders

    Aoi-ku, Shizuoka-shi, Shizuoka, 420-8688

  • Niigata University Medical & Dental Hospital

    Asahimachidori, Chuo-ku, Niigata-shi, Niigata, 951-8520

  • Juntendo University Hospital

    Bunkyo-ku, Tokyo, 113-8431

  • Chiba University Hospital

    Chiba, 260-8677

  • National Hospital Organization Chibahigashi National Hospital

    Chuo-ku, Chiba-shi, Chiba, 260-8712

  • Saitama Neuropsychiatric Institute

    Chuo-ku, Saitama-shi, Saitama, 338-8577

  • Tokyo Metropolitan Neurological Hospital

    Fuchū, Tokyo, 183-0042

  • Fukushima Medical University Hospital

    Fukushima, Fukushima, 960-1295

  • Kansai Electric Power Hospital

    Fukushima-ku, Osaka-shi, Osaka, 553-0003

  • National Hospital Organization Iou National Hospital

    Kanazawa, Ishikawa-ken, 920-0192

  • Yokohama City University Hospital

    Kanazawa-ku, Yokohama-shi, Kanagawa, 236-0004

  • National Hospital Organization Kumamoto Saishun Medical Center

    Koshi-shi, Kumamoto, 861-1196

  • National Hospital Organization Higashinagoya National Hospital

    Meito-ku, Nagoya-shi, Aichi-ken, 465-8620

  • Kagawa University Hospital

    Miki-cho, Kita-gun, Kagawa-ken, 761-0793

  • Hiroshima University Hospital

    Minami-ku, Hiroshima-shi, Hiroshima, 734-8551

  • Kitasato University Hospital

    Minami-ku, Sagamihara-city, Kanagawa, 252-0375

  • Murakami Karindoh Hospital

    Nishi-ku, Fukuoka-shi, Fukuoka, 819-8585

  • Toho University Omori Medical Center

    Ōta-ku, Tokyo, 143-8541

  • Shiga University of Medical Science Hospital

    Ōtsu, Shiga, 520-2192

  • National Hospital Organization Hokkaido Medical Center

    Sapporo, Hokkaido, 063-0005

  • Tohoku University Hospital

    Sendai, Miyagi, 980-8574

  • Keio University Hospital

    Shinjuku-ku, Tokyo, 160-8582

  • Nagoya University Hospital

    Showa-ku, Nagoya, Aichi-ken, 466-8560

  • Teikyo University Hospital

    tabashi City, Tokyo, 173-8606

  • National Hospital Organization Osaka Toneyama Medical Center

    Toyonaka-shi, Osaka, 560-8552

  • National Hospital Organization Utano National Hospital

    Ukyo-ku, Kyoto City, Kyoto, 616-8255

Other trials in Japan

Germany11 sites

  • Charite Campus Virchow

    Berlin, 13353

  • UKRUB - Berufsgenossenschaftliches Universitatsklinikum Bergmannsheil GmbH - Medizinische Klinik III

    Bochum, North Rhine-Westphalia, 44789

  • Universitätsklinik Bonn-Motoneuronambulanz, Klinik und Poliklinik für Neurodegenerative Erkrankungen und Gerontopsychiatrie

    Bonn, 53127

  • Georg-August-Universitaet Goettingen - Universitaetsmedizin Goettingen (UMG)

    Göttingen, 37075

  • Medizinische Hochschule Hannover

    Hanover, Lower Saxony, 30625

  • Universitaetsklinikum Jena

    Jena, 86899

  • Klinikum Rechts der Isar der Technischen Universitaet Muenchen

    München, 81675

  • University Medical Center Rostock

    Rostock, 18147

  • Universitaets- und Rehabilitationskliniken Ulm

    Ulm, 89081

  • Deutsche Klinik fuer Diagnostik

    Wiesbaden, 65191

  • Universitaetsklinikum Wuerzburg

    Wuezburg, Germany, 97080

Other trials in Germany

Canada10 sites

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

    Edmonton, Alberta, T6G 2B7

  • Regional Health Authority B

    Fredericton, New Brunswick, E3B 0C7

  • Recherche Sepmus, Inc

    Greenfield Park, Quebec, J4V 2J2

  • Health Science Center Mcmaster University

    Hamilton, Ontario, L8P 1H1

  • London Health Sciences Centre - University Hospital

    London, Ontario, N6A 5A5

  • Centre Hospitalier De L'Universite De Montreal (Chum) Notre-Dame Hospital

    Montreal, Quebec, H2L 4M1

  • Montreal Neurological Institute And Hospital

    Montreal, Quebec, H3A 2B4

  • CHU de Quebec-Hopital-Enfant-Jesus

    Québec, Quebec, G1J 1Z4

  • Saskatoon City Hospital

    Saskatoon, Saskatchewan, S7K 0M7

  • Odette Cancer Center-Sunnybrook Health Sciences Centre

    Toronto, Ontario, M4N 3M5

Other trials in Canada

Italy7 sites

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

    Milan, Italy

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

    Milan, 20132

  • Istituto Nazionale Neurologico Carlo Besta

    Milan, Italy

  • Istituto Auxologico Italiano - Istituto Di Ricovero e Cura a Carattere Scientifico - Istituto Scientifico Ospedale San Luca

    Modena, 41126

  • Centro SLA di Palermo

    Palermo, 90129

  • Policlinico A. Gemelli

    Roma, CAP 00168

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

    Turin, Piedmont, 10126

Other trials in Italy

Switzerland4 sites

  • University hospital Bern (Inselspital)

    Bern, Canton of Bern, 3010

  • Hopitaux Universitaires de Geneve (HUG) (Hopital Cantonal)

    Geneva, 1205

  • Neurocenter of Southern Switzerland

    Lugano, 6903

  • Zentrumsleiter Muskelzentrum/ALS Clinic Kantonsspital St.Gallen Muskelzentrum/ALS Clinic

    Sankt Gallen, 9107

Other trials in Switzerland

South Korea3 sites

  • Seoul National University Hospital

    Seoul, 03080

  • Hanyang University Medical Center

    Seoul, 04763

  • Samsung Medical Center

    Seoul, 06351

Other trials in South Korea

About this study

Official title: A Phase 3b, Multicenter, Randomized, Double-Blind Study to Evaluate Efficacy and Safety of Oral Edaravone Administered for a Period of 48 Weeks in Subjects With Amyotrophic Lateral Sclerosis (ALS)

To evaluate and compare the efficacy of two dosing regimens of oral edaravone in subjects with amyotrophic lateral sclerosis (ALS) based on the change in ALS Functional Rating Scale- Revised (ALSFRS-R) score from baseline up to Week 48:

Conditions
ALS
Being tested
MT-1186, Placebo

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • MT-1186Drug

    Also called Oral edaravone

    Oral edaravone

  • PlaceboDrug

    Oral

Study groups in NCT04569084
Study groupRoleWhat they receive
MT-1186ExperimentalNot described
Receives: Drug: MT-1186
MT-1186 and PlaceboExperimentalNot described
Receives: Drug: MT-1186; Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. CAFS Score at Week 48

    CAFS ranks patients' clinical outcomes based on survival time and change in the ALSFRS-R score. To calculate a patient's CAFS, each patient is compared individually to all other patients in the study. The summary score for each patient is the sum of the comparisons ( 1, 0, 1) against all other patients. After that, patients' summary scores are ranked. The CAFS rank is 1-383 and a higher CAFS rank indicates a better outcome than does a lower CAFS. Since CAFS were calculated using the imputed ALSFRS-R scores with multiple imputation method, and the maximum varies for each simulation, 383 is the maximum possible value of full range for measure of dispersion.

    Measured: up to 48 Weeks

Secondary outcomes (5)

  1. Change From Baseline in % Slow Vital Capacity (SVC) at Week 48

    SVC measurements will be conducted in clinic at around the same time of day where possible with the subject in sitting upright position. Subjects should make at least 3 attempts to generate acceptable and reproducible SVC data. The highest value was selected and recorded.

    Measured: up to 48 Weeks

  2. Change From Baseline in Amyotrophic Lateral Sclerosis Assessment Questionnaire (ALSAQ)40 at Week 48

    The ALSAQ-40 is a questionnaire that consists of 40 questions/items with 5 discrete scales: physical mobility, activities of daily living and independence, eating and drinking, communication, emotional reactions, ranging from 0 (best health as assessed by the scale) to 100 (worse health as assessed by the measure).

    Measured: up to 48 Weeks

  3. Time to Death, Tracheostomy or Permanent Assisted Mechanical Ventilation (≥ 23 Hours/Day)

    Measured: up to 48 Weeks

  4. Time to Death or PAMV (≥ 23 Hours/Day)

    Measured: up to 48 Weeks

  5. Time to Death

    Measured: up to 48 Weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted29 Sep 2020
  2. Study start13 Nov 2020
  3. Primary completion29 Sep 2023
  4. Study completion29 Sep 2023

Why it stopped: Futility was met.

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
384 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04569084
Sponsor's protocol ID
MT-1186-A02
Other IDs
jRCT2031200301, 2019-004256-11, 2019
Status last verified
Jun 2026
First submitted
23 Sep 2020
Last updated
20 Jul 2026
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Undecided

Publications

  • Rothstein J, Genge A, De Silva S, Zinman L, Chum M, Chio A, Sobue G, Aoki M, Yoshino H, Doyu M, Selness D, Todorovic V, Sasson N, Hirai M, Takahashi F, Salah A, Wamil A, Apple S. Efficacy and Safety of Once Daily Dosing vs. Approved On/Off Dosing of Edaravone Oral Suspension Up to 48 Weeks in Patients With Amyotrophic Lateral Sclerosis (Study MT-1186-A02). Muscle Nerve. 2025 Sep;72(3):433-442. doi: 10.1002/mus.28448. Epub 2025 Jun 6.

    PubMed 40474686

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing MT-1186 in people with ALS, run by Shionogi at 95 sites in 7 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 20 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 20 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 95 sites in United States, Japan, Germany, Canada, Italy and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 27 Mar 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

CAFS Score at Week 48 (time frame: up to 48 Weeks). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04569084, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Jul 2026.

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