Clinical trial NCT04577404: Safety Extension Study of Oral Edaravone Administered in Subjects With Amyotrophic Lateral Sclerosis (ALS)
Official title: A Phase 3, Multi-center, Open-label, Safety Extension Study of Oral Edaravone Administered Over 96 Weeks in Subjects With Amyotrophic Lateral Sclerosis (ALS)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Amyotrophic Lateral Sclerosis (ALS)
Lead sponsor: Mitsubishi Tanabe Pharma America Inc.
Sponsor class: INDUSTRY
First posted: 2020-10-06
Enrollment: 124
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Single Arm Open Label
Primary purpose: TREATMENT

Brief summary: This is a Phase 3, international, multicenter, open-label, long-term extension study. The primary objective of this study is to evaluate the long-term safety and tolerability of oral edaravone in subjects with Amyotrophic Lateral Sclerosis (ALS) for up to 96 weeks.


Eligibility criteria: Inclusion Criteria:

1. Subjects must provide 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.
2. Subjects must be willing to cooperate and comply with all protocol restrictions and requirements.
3. Subjects who successfully completed Study MT-1186-A01.

Exclusion Criteria:

1. Subjects of childbearing potential unwilling to use a highly effective method of contraception from Visit 1 until 3 months after the last dose of study medication.
2. Subjects who have a significant risk of suicide. 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 C-SSRS at Visit
3. Subjects who are not eligible to continue in the study, as judged by the Investigator.
4. Subjects who are unable to take their medications orally or through a PEG/RIG tube.

Primary outcomes:
measure: Number of Events of Treatment Emergency Adverse Events (TEAEs), Adverse Drug Reactions (ADRs), Severe TEAEs, TESEAs, TEAEs Leading to Discontinuation, Any TEAEs Leading to Death
timeFrame: up to 96 Weeks
measure: Number of Subjects of Treatment Emergency Adverse Events (TEAEs), Adverse Drug Reactions (ADRs), Severe TEAEs, TESAEs, TEAEs Leading to Discontinuation and TEAEs Leading to Death
timeFrame: up to 96 Weeks
Secondary outcomes:
Other outcomes:
measure: Change From Baseline of ALS Functional Rating Scale-Revised (ALSFRS-R) at Week 96
timeFrame: Baseline to Week 96
measure: Number of Events of Death, Tracheostomy, or Permanent Assisted Mechanical Ventilation
timeFrame: up to 96 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: 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: 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: Pittsburgh
country: United States
facility: Alleghany General Hospital
state: Pennsylvania
city: Dallas
country: United States
facility: Texas Neurology, PA
state: Texas
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: Montreal
country: Canada
facility: Montreal Neurological Institute and Hospital
state: Quebec
city: Nice
country: France
facility: CHU-Nice - Hopital Pasteur 2
state: Cedex 1
city: Limoges
country: France
facility: Centre Hospitalier Esquirol
state: Marcland
city: Bordeaux
country: France
facility: Centre Hospitalier Universitaire (CHU) de Bordeaux
city: Paris
country: France
facility: Hopital Pierre Wertheimer - Hopital Neurologique
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: Lombardia
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 Chibahigashi National 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: 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: Fukushima-ku, Osaka-shi
country: Japan
facility: Kansai Electric Power Hospital Recruiting
state: Osaka
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
city: Fuchu-city
country: Japan
facility: Tokyo Metropolitan Neurological Hospital
state: Tokyo
city: Ota-ku
country: Japan
facility: Toho University Omori Medical Center
state: Tokyo
Study officials:
affiliation: Mitsubishi Tanabe Pharma America Inc.
name: Head of Medical Science
role: STUDY_DIRECTOR
References: