Clinical trial NCT04165824: Safety Study of Oral Edaravone Administered in Subjects With ALS
Official title: Safety Study of Oral Edaravone Administered in Subjects With ALS
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: ALS
Lead sponsor: Mitsubishi Tanabe Pharma America Inc.
Sponsor class: INDUSTRY
First posted: 2019-11-18
Enrollment: 185
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: 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.

Primary outcomes:
measure: Number of Treatment Emergency Adverse Events
timeFrame: up to 48 Weeks
measure: Number of Participants With Treatment Emergency Adverse Events
timeFrame: Up to 48 Weeks
Secondary outcomes:
Other outcomes:
measure: Change in ALS Functional Rating Scale - Revised From Baseline
timeFrame: up to 48 Weeks
measure: Time to Event (Death, Tracheostomy, and Permanent Assisted Mechanical Ventilation)
timeFrame: up to 48 Weeks
Locations:
city: Phoenix
country: United States
facility: St. Joseph's Hospital and Medical Center (SJHMC) - Barrow Neurological Institute (BNI) - The Gregory W. Fulton ALS and Neuromuscular Disease Center
state: Arizona
city: Phoenix
country: United States
facility: Neuromuscular Research Center
state: Arizona
city: Rogers
country: United States
facility: Woodland Research Northwest
state: Arkansas
city: San Francisco
country: United States
facility: Sutter Health
state: California
city: Aurora
country: United States
facility: University of Colorado Anschutz Medical Campus
state: Colorado
city: Gainesville
country: United States
facility: UF Health Cancer Center
state: Florida
city: Atlanta
country: United States
facility: Emory University - School of Medicine
state: Georgia
city: Baltimore
country: United States
facility: Johns Hopkins University
state: Maryland
city: Ann Arbor
country: United States
facility: University of Michigan - ALS Center of Excellence
state: Michigan
city: Northville
country: United States
facility: University of Michigan Health System (UMHS) - Allergy Clinic - Northville Health Center Location
state: Michigan
city: Duluth
country: United States
facility: Essentia Institute of Rural Health
state: Minnesota
city: Lincoln
country: United States
facility: Neurology Associates, P.C - Lincoln
state: Nebraska
city: Winston-Salem
country: United States
facility: Wake Forest University Baptist Medical Center (WFUBMC) - The J. Paul Sticht Center on Aging and Rehabilitation
state: North Carolina
city: Hershey
country: United States
facility: Penn State Hershey Children's Hospital
state: Pennsylvania
city: Philadelphia
country: United States
facility: Lewis Katz School of Medicine at Temple University
state: Pennsylvania
city: Pittsburgh
country: United States
facility: Alleghany General Hospital
state: Pennsylvania
city: Cordova
country: United States
facility: Wesley Neurology Clinic, P.C.
state: Tennessee
city: Dallas
country: United States
facility: Texas Neurology, PA
state: Texas
city: Houston
country: United States
facility: Houston Methodist Neurological Institute
state: Texas
city: San Antonio
country: United States
facility: UT Health Science Center San Antonio
state: Texas
city: Seattle
country: United States
facility: University of Washington Medical Center
state: Washington
city: Milwaukee
country: United States
facility: Medical College of Wisconsin
state: Wisconsin
city: Calgary
country: Canada
facility: Heritage Medical Research Clinic - University Of Calgary
state: Alberta
city: Edmonton
country: Canada
facility: University of Alberta - Walter C Mackenzie Health Sciences Centre (WCM)
state: Alberta
city: Greenfield Park
country: Canada
facility: Recherche Sepmus, Inc
state: Quebec
city: Montreal
country: Canada
facility: Montreal Neurological Institute and Hospital
state: Quebec
city: Nice cedex 3
country: France
facility: CHU Nice-Hospital Archet I
state: Alpes Maritimes
city: Limoges
country: France
facility: Centre Hospitalier Esquirol
state: Marcland
city: Bordeaux
country: France
facility: Centre Hospitalier Universitaire (CHU) de Bordeaux
city: Bron
country: France
facility: Hopital Pierre Wertheimer - Hopital Neurologique
city: Nice cedex 1
country: France
facility: CHU de NICE Hopital Pasteur 2 -Centre de Reference des Maladies -Neuromusculaires & SLA-Pole Neurosciences Cliniques
city: Wiesbaden
country: Germany
facility: Deutsche Klinik fuer Diagnostik
state: Hessen
city: Hannover
country: Germany
facility: Medizinische Hochschule Hannover
state: Lower Saxony
city: Milano
country: Italy
facility: Ospedale San Raffaele (HSR) (Istituto Scientifico Universitario San Raffaele)
state: MI
city: Turin
country: Italy
facility: Universita degli Studi di Torino - Centro Regionale Esperto Per La Sclerosi Laterale Amiotrofica (CRESLA)
state: Piemonte
city: Milan
country: Italy
facility: Fondazione Serena Onlus - Azienda Ospedaliera Niguarda Ca Granda - Centro Clinico Nemo (Neuro Muscular Omnicentre)
city: Nagakute-shi
country: Japan
facility: Aichi Medical University Hospital
state: Aichi
city: Chiba-shi
country: Japan
facility: National Hospital Organization Chiba-East-Hospital
state: Chiba
city: Fukuoka-city
country: Japan
facility: Murakami Karindoh Hospital
state: Fukuoka
city: Fukushima-shi
country: Japan
facility: Fukushima Medical University Hospital
state: Fukushima
city: Sapporo-shi
country: Japan
facility: National Hospital Organization Hokkaido Medical Center
state: Hokkaido
city: Kanazawa-shi
country: Japan
facility: National Hospital Organization Iou National Hospital
state: Ishikawa
city: Kita-gun
country: Japan
facility: Kagawa University Hospital
state: Kagawa
city: Sagamihara-city
country: Japan
facility: Kitasato University Hospital
state: Kanagawa
city: Yokohama-shi
country: Japan
facility: Yokohama City University Hospital
state: Kanagawa
city: Koshi-shi
country: Japan
facility: National Hospital Organization Kumamoto Saishun Medical Center
state: Kumamoto
city: Kyoto-shi
country: Japan
facility: National Hospital Organization Utano Hospital
state: Kyoto
city: Sendai-city
country: Japan
facility: Tohoku University Hospital
state: Miyagi
city: Niigata-shi
country: Japan
facility: Niigata University Medical And Dental Hospital
state: Niigata
city: Toyonaka-shi
country: Japan
facility: National Hospital Organization Toneyama Medical Center
state: Osaka
city: Otsu-shi
country: Japan
facility: Shiga University of Medical Science Hospital
state: Shiga
city: Shizuoka-city
country: Japan
facility: National Hospital Organization Shizuoka Institute of Epilepsy and Neurological Disorders
state: Shizuoka
city: Bunkyo-ku
country: Japan
facility: Juntendo University Hospital
state: Tokyo
Study officials:
affiliation: Mitsubishi Tanabe Pharma America Inc.
name: Head of Medical Science,
role: STUDY_DIRECTOR
References: