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)

Full Study Summary:

• Eligibility Criteria

  Gender:

  Female, Male

  Minimum Age:

  18

  Maximum Age:

  75

  Min Vital Capacity (% predicted normal):

  70

  Time since Symptom Onset:

  <24 months

  Time since Diagnosis:

  Can participants use Riluzole?

  Yes

  
  

  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 points decline for 8 weeks 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 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).

• Site Contact Information

  St. Joseph's Hospital and Medical Center (SJHMC)
  Phoenix, Arizona 85013
  United States

  HonorHealth Neurology
  Scottsdale, Arizona 85251
  United States

  Woodland Research Northwest
  Rogers, Arkansas 72758
  United States

  UCSD Medical Center
  La Jolla, California 92037
  United States

  University California Los Angeles Medical Center (UCLA)
  Los Angeles, California 90095
  United States

  University of California Irvine (UCI)
  Orange, California 92868
  United States

  University of Colorado Anschutz Medical Campus
  Aurora, Colorado 80045
  United States

  UF Health Cancer Center
  Gainesville, Florida 32610
  United States

  Mayo Clinic
  Jacksonville, Florida 32224
  United States

  University of South Florida (USF)
  Tampa, Florida 33616
  United States

  Emory University
  Atlanta, Georgia 30322
  United States

  Northwestern University Feinberg School of Medicine
  Chicago, Illinois 60611
  United States

  Johns Hopkins University
  Baltimore, Maryland 21205
  United States

  Lahey Hospital
  Burlington, Massachusetts 01805
  United States

  Mayo Clinic
  Rochester, Minnesota 55905
  United States

  Neurology Associates, P.C.
  Lincoln, Nebraska 68506
  United States

  Las Vegas Clinic
  Las Vegas, Nevada 89145
  United States

  Dent Neurologic Institute
  Amherst, New York 14226
  United States

  SUNY Upstate Medical University
  Syracuse, New York 13210
  United States

  Lewis Katz School of Medicine at Temple University
  Philadelphia, Pennsylvania 19140
  United States

  University Of Pittsburgh Medical Center
  Pittsburgh, Pennsylvania 15261
  United States

  Wesley Neurology Clinic, P.C.
  Cordova, Tennessee 38018
  United States

  Austin Neuromuscular Center
  Austin, Texas 78756
  United States

  Nerve And Muscle Center Of Texas
  Houston, Texas 77019
  United States

  The University of Vermont (UVM)
  Burlington, Vermont 05401
  United States

  Sentara Neurology Specialists
  Virginia Beach, Virginia 23456
  United States

  University of Washington Medical Center
  Seattle, Washington 98195
  United States

  St. Luke's Rehabilitation Institute
  Spokane, Washington 99202
  United States

  West Virginia University School of Medicine
  Morgantown,, West Virginia 26506
  United States

  Medical College of Wisconsin
  Milwaukee, Wisconsin 53226
  United States

  University of Alberta
  Edmonton, Alberta
  Canada

  Regional Health Authority B
  Fredericton, New Brunswick
  Canada

  Health Science Center Mcmaster University
  Hamilton, Ontario
  Canada

  London Health Sciences Centre
  London, Ontario
  Canada

  Odette Cancer Center-Sunnybrook Health Sciences Centre
  Toronto, Ontario
  Canada

  Recherche Sepmus, Inc
  Greenfield Park, Quebec
  Canada

  Centre Hospitalier De L'Universite De Montreal (Chum) Notre-Dame Hospital
  Montreal, Quebec
  Canada

  Montreal Neurological Institute And Hospital
  Montreal, Quebec
  Canada

  CHU de Quebec-Hopital-Enfant-Jesus
  Quebec City, Quebec
  Canada

  Saskatoon City Hospital
  Saskatoon, Saskatchewan
  Canada

  Charite Campus Virchow
  Berlin,
  Germany

  Universitätsklinik Bonn-Motoneuronambulanz
  Bonn,
  Germany

  Universitaetsklinikum Jena
  Jena,
  Germany

  University Medical Center Rostock
  Rostock,
  Germany

  Universitaets- und Rehabilitationskliniken Ulm
  Ulm,
  Germany

  Deutsche Klinik fuer Diagnostik
  Wiesbaden,
  Germany

  Universitaetsklinikum Wuerzburg
  Wuezburg,
  Germany

  Medizinische Hochschule Hannover
  Hannover, Lower Saxony
  Germany

  UKRUB
  Bochum, Nordrhein-Westfalen
  Germany

  Fondazione Serena Onlus
  Milan,
  Italy

  Istituto Nazionale Neurologico Carlo Besta
  Milano,
  Italy

  Ospedale San Raffaele (HSR)
  Milano,
  Italy

  Istituto Auxologico Italiano
  Modena,
  Italy

  Universita degli Studi di Torino
  Turin, Piemonte
  Italy

  Fukushima Medical University Hospital
  Fukushima-shi, Fukushima
  Japan

  National Hospital Organization Hokkaido Medical Center
  Sapporo-shi, Hokkaido
  Japan

  National Hospital Organization Iou National Hospital
  Kanazawa-shi, Ishikawa
  Japan

  National Hospital Organization Kumamoto Saishun Medical Center
  Koshi-shi, Kumamoto
  Japan

  Seoul National University Hospital
  Seoul,
  Republic of Korea

  Zentrumsleiter Muskelzentrum
  St.Gallen,
  Switzerland