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

Sponsor
Mitsubishi Tanabe Pharma Development America, Inc. (Industry)
Overall Status
Recruiting
CT.gov ID
NCT04569084
Collaborator
(none)
380
Enrollment
95
Locations
2
Arms
31.5
Anticipated Duration (Months)
4
Patients Per Site
0.1
Patients Per Site Per Month

Study Details

Study Description

Brief 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:
Condition or Disease Intervention/Treatment Phase
Phase 3

Detailed Description

Contacts:

  • North America (USA English, USA Spanish, and Canadian French) +1-800-313-9381; mailbox address is MT1186@iconplc.com

  • Japan: 0120-330-800 (Japan only)

Study Design

Study Type:
Interventional
Anticipated Enrollment :
380 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Masking:
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose:
Treatment
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)
Actual Study Start Date :
Nov 13, 2020
Anticipated Primary Completion Date :
Jul 1, 2023
Anticipated Study Completion Date :
Jul 1, 2023

Arms and Interventions

Arm Intervention/Treatment
Experimental: MT-1186

Drug: MT-1186
Oral edaravone
Other Names:
  • Oral edaravone

    		•
    Experimental: MT-1186 and Placebo

    Drug: MT-1186
    Oral edaravone
    Other Names:
  • Oral edaravone

    • Drug: Placebo
    Oral

    Outcome Measures

    Primary Outcome Measures

    1. Change in ALS Functional Rating Scale- Revised (ALSFRS-R) score from baseline to Week 48 of treatment [up to 48 Weeks]

      The ALSFRS-R is rating scale (ratings 0 = can't do, to 4 = normal ability) used to determine participants' assessment of their capability and independence in 12 functional activities.

    Secondary Outcome Measures

    1. Change from baseline in % slow vital capacity (SVC) at Week 48 [up to 48 Weeks]

    2. Change from baseline in Amyotrophic Lateral Sclerosis Assessment Questionnaire (ALSAQ)40 at Week 48 [up to 48 Weeks]

      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).

    Other Outcome Measures

    1. Time to death, tracheostomy or permanent assisted mechanical ventilation (≥ 23 hours/day) [up to 48 Weeks]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    18 Years to 75 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:

    1. 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.

    2. Subjects will be male or female, ≥ 18 to 75 years of age at the time the ICF is signed.

    3. Subjects will be diagnosed with Definite ALS or Probable ALS according to the El Escorial revised criteria for the diagnosis of ALS.

    4. Subjects with a baseline score ≥ 2 points on each individual item of the ALSFRS- R at screening and baseline visits.

    5. Subjects have a screening and baseline %forced vital capacity (FVC) ≥ 70%.

    6. Subjects with 1- to 4-point decline for 8 weeks (±7 days) in ALSFRS-R total score between screening and baseline visits.

    7. Subjects whose first symptom of ALS has occurred within 2 years of providing written informed consent.

    Exclusion Criteria:

    Exclusions Related to Primary Diagnosis

    1. 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)

    1. 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

    1. Subjects undergoing treatment for a malignancy.

    2. 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.

    3. 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.

    4. Subjects who are female, of childbearing potential, and pregnant (a positive pregnancy test) or lactating at the screening visit (Visit 1).

    5. 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.

    6. 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.

    7. Subjects who have alanine aminotransferase (ALT) or aspartate aminotransferase (AST) elevations greater than 2 times the upper limit of normal (ULN) at screening.

    8. Subjects with a Glomerular Filtration Rate (GFR) < 30 mL/Min Per 1.73 m2 at screening, using the Larsson Equation.

    Exclusions Related to Medications

    1. Subjects with history of hypersensitivity to edaravone, any of the additives or inactive ingredients of edaravone, or sulfites.

    2. Subjects with hereditary problems of fructose intolerance (eg, fructose, sucrose, invert sugar, and sorbitol).

    3. 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.

    4. Subjects who have received any previous treatment with edaravone.

    5. Subjects who have received stem cell therapy.

    6. Subjects who are unable to take their medications orally at baseline (Visit 2).

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 St. Joseph's Hospital and Medical Center (SJHMC) Phoenix Arizona United States 85013
    2 HonorHealth Neurology Scottsdale Arizona United States 85251
    3 Woodland Research Northwest Rogers Arkansas United States 72758
    4 UCSD Medical Center La Jolla California United States 92037-0897
    5 Loma Linda University Health Care - Department of Neurology Loma Linda California United States 92354
    6 University California Los Angeles Medical Center (UCLA) Los Angeles California United States 90095
    7 University of California Irvine (UCI) Health - Women's Healthcare Center Orange California United States 92868
    8 University of Colorado Anschutz Medical Campus Aurora Colorado United States 80045
    9 UF Health Cancer Center Gainesville Florida United States 32610-3633
    10 Mayo Clinic Jacksonville Florida United States 32224
    11 University of South Florida (USF) - Carol and Frank Morsani Center for Advanced Health Care (CAHC) Tampa Florida United States 33616
    12 Emory University - School of Medicine Atlanta Georgia United States 30322
    13 Northwestern University Feinberg School of Medicine Chicago Illinois United States 60611-2605
    14 Ochsner Center for Primary Care and Wellness Jefferson Louisiana United States 70121
    15 Johns Hopkins University Baltimore Maryland United States 21205
    16 Lahey Hospital Burlington Massachusetts United States 01805
    17 Mayo Clinic Rochester Minnesota United States 55905
    18 Neurology Associates, P.C. - Lincoln Lincoln Nebraska United States 68506-2960
    19 Las Vegas Clinic Las Vegas Nevada United States 89145
    20 Dent Neurologic Institute Amherst New York United States 14226
    21 SUNY Upstate Medical University Syracuse New York United States 13210
    22 Penn State Hershey Medical Center Hershey Pennsylvania United States 17033
    23 Thomas Jefferson University, Jefferson Weinberg ALS Center Philadelphia Pennsylvania United States 19107
    24 Lewis Katz School of Medicine at Temple University Philadelphia Pennsylvania United States 19140
    25 University Of Pittsburgh Medical Center Pittsburgh Pennsylvania United States 15261
    26 Wesley Neurology Clinic, P.C. Cordova Tennessee United States 38018
    27 Austin Neuromuscular Center Austin Texas United States 78756
    28 Nerve And Muscle Center Of Texas Houston Texas United States 77019
    29 The University of Vermont (UVM) and UVM Medical Center National ALS Center of Excellence Burlington Vermont United States 05401-3456
    30 Sentara Neurology Specialists Virginia Beach Virginia United States 23456
    31 University of Washington Medical Center Seattle Washington United States 98195
    32 St. Luke's Rehabilitation Institute Spokane Washington United States 99202
    33 West Virginia University School of Medicine (WVUSoM) - Movement Disorder Clinic Morgantown West Virginia United States 26506-9180
    34 Medical College of Wisconsin Milwaukee Wisconsin United States 53226
    35 University of Alberta - Walter C Mackenzie Health Sciences Centre (WCM) Edmonton Alberta Canada T6G 2B7
    36 Regional Health Authority B Fredericton New Brunswick Canada E3B 0C7
    37 Health Science Center Mcmaster University Hamilton Ontario Canada L8P 1H1
    38 London Health Sciences Centre - University Hospital London Ontario Canada N6A 5A5
    39 Odette Cancer Center-Sunnybrook Health Sciences Centre Toronto Ontario Canada M4N 3M5
    40 Recherche Sepmus, Inc Greenfield Park Quebec Canada J4V 2J2
    41 Centre Hospitalier De L'Universite De Montreal (Chum) Notre-Dame Hospital Montreal Quebec Canada H2L 4M1
    42 Montreal Neurological Institute And Hospital Montreal Quebec Canada H3A 2B4
    43 CHU de Quebec-Hopital-Enfant-Jesus Quebec City Quebec Canada G1J 1Z4
    44 Saskatoon City Hospital Saskatoon Saskatchewan Canada S7K 0M7
    45 Medizinische Hochschule Hannover Hannover Lower Saxony Germany 30625
    46 UKRUB - Berufsgenossenschaftliches Universitatsklinikum Bergmannsheil GmbH - Medizinische Klinik III Bochum Nordrhein-Westfalen Germany 44789
    47 Charite Campus Virchow Berlin Germany 13353
    48 Universitätsklinik Bonn-Motoneuronambulanz, Klinik und Poliklinik für Neurodegenerative Erkrankungen und Gerontopsychiatrie Bonn Germany 53127
    49 Georg-August-Universitaet Goettingen - Universitaetsmedizin Goettingen (UMG) Goettingen Germany 37075
    50 Universitaetsklinikum Jena Jena Germany 86899
    51 Klinikum Rechts der Isar der Technischen Universitaet Muenchen Muenchen Germany 81675
    52 University Medical Center Rostock Rostock Germany 18147
    53 Universitaets- und Rehabilitationskliniken Ulm Ulm Germany 89081
    54 Deutsche Klinik fuer Diagnostik Wiesbaden Germany 65191
    55 Universitaetsklinikum Wuerzburg Wuezburg Germany 97080
    56 Universita degli Studi di Torino - Centro Regionale Esperto Per La Sclerosi Laterale Amiotrofica (CRESLA) Turin Piemonte Italy 10126
    57 Ospedale San Raffaele (HSR) (Istituto Scientifico Universitario San Raffaele) Milano Italy 20132
    58 Istituto Nazionale Neurologico Carlo Besta Milano Italy Italy
    59 Fondazione Serena Onlus - Azienda Ospedaliera Niguarda Ca Granda - Centro Clinico Nemo (Neuro Muscular Omnicentre) Milan Italy Italy
    60 Istituto Auxologico Italiano - Istituto Di Ricovero e Cura a Carattere Scientifico - Istituto Scientifico Ospedale San Luca Modena Italy 41126
    61 Centro SLA di Palermo Palermo Italy 90129
    62 Policlinico A. Gemelli Roma Italy CAP 00168
    63 National Hospital Organization Higashinagoya National Hospital Meito-ku, Nagoya-shi Aichi Japan 465-8620
    64 Nagoya University Hospital Showa-ku, Nagoya Aichi Japan 466-8560
    65 National Hospital Organization Chibahigashi National Hospital Chuo-ku, Chiba-shi Chiba Japan 260-8712
    66 Murakami Karindoh Hospital Nishi-ku, Fukuoka-shi Fukuoka Japan 819-8585
    67 Fukushima Medical University Hospital Fukushima-shi Fukushima Japan 960-1295
    68 Hiroshima University Hospital Minami-ku, Hiroshima-shi Hiroshima Japan 734-8551
    69 National Hospital Organization Hokkaido Medical Center Sapporo-shi Hokkaido Japan 063-0005
    70 National Hospital Organization Iou National Hospital Kanazawa-shi Ishikawa Japan 920-0192
    71 Kagawa University Hospital Miki-cho, Kita-gun Kagawa Japan 761-0793
    72 Yokohama City University Hospital Kanazawa-ku, Yokohama-shi Kanagawa Japan 236-0004
    73 Kitasato University Hospital Minami-ku, Sagamihara-city Kanagawa Japan 252-0375
    74 National Hospital Organization Kumamoto Saishun Medical Center Koshi-shi Kumamoto Japan 861-1196
    75 National Hospital Organization Utano National Hospital Ukyo-ku, Kyoto City Kyoto Japan 616-8255
    76 Tohoku University Hospital Sendai-city Miyagi Japan 980-8574
    77 Niigata University Medical & Dental Hospital Asahimachidori, Chuo-ku, Niigata-shi Niigata Japan 951-8520
    78 Kansai Electric Power Hospital Fukushima-ku, Osaka-shi Osaka Japan 553-0003
    79 National Hospital Organization Osaka Toneyama Medical Center Toyonaka-shi Osaka Japan 560-8552
    80 Saitama Neuropsychiatric Institute Chuo-ku, Saitama-shi Saitama Japan 338-8577
    81 Shiga University of Medical Science Hospital Otsu City Shiga Japan 520-2192
    82 National Hospital Organization Shizuoka Institute of Epilepsy and Neurological Disorders Aoi-ku, Shizuoka-shi Shizuoka Japan 420-8688
    83 Juntendo University Hospital Bunkyo-ku Tokyo Japan 113-8431
    84 Tokyo Metropolitan Neurological Hospital Fuchu-city Tokyo Japan 183-0042
    85 Teikyo University Hospital Itabashi-ku Tokyo Japan 173-8606
    86 Toho University Omori Medical Center Ota-ku Tokyo Japan 143-8541
    87 Keio University Hospital Shinjuku-ku Tokyo Japan 160-8582
    88 Chiba University Hospital Chiba Japan 260-8677
    89 Seoul National University Hospital Seoul Korea, Republic of 03080
    90 Hanyang University Medical Center Seoul Korea, Republic of 04763
    91 Samsung Medical Center Seoul Korea, Republic of 06351
    92 University hospital Bern (Inselspital) Bern BE Switzerland 3010
    93 Hopitaux Universitaires de Geneve (HUG) (Hopital Cantonal) Geneve Switzerland 1205
    94 Neurocenter of Southern Switzerland Lugano Switzerland 6903
    95 Zentrumsleiter Muskelzentrum/ALS Clinic Kantonsspital St.Gallen Muskelzentrum/ALS Clinic St.Gallen Switzerland 9107

    Sponsors and Collaborators

    • Mitsubishi Tanabe Pharma Development America, Inc.

    Investigators

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

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Mitsubishi Tanabe Pharma Development America, Inc.
    ClinicalTrials.gov Identifier:
    NCT04569084
    Other Study ID Numbers:
    • MT-1186-A02
    First Posted:
    Sep 29, 2020
    Last Update Posted:
    May 24, 2022
    Last Verified:
    May 1, 2022
    Individual Participant Data (IPD) Sharing Statement:
    Undecided
    Plan to Share IPD:
    Undecided
    Studies a U.S. FDA-regulated Drug Product:
    Yes
    Studies a U.S. FDA-regulated Device Product:
    No
    Additional relevant MeSH terms:
    Edaravone

    Study Results

    No Results Posted as of May 24, 2022