Clinical trial NCT05151471: Efficacy and Safety Extension Study of Oral Edaravone Administered in Subjects With ALS
Official title: A Phase 3b, Multicenter, Randomized, Double-blind Extension Study to Evaluate the Continued Efficacy and Safety of Oral Edaravone Administered for an Additional Period of up to 48 Weeks Following Study MT-1186-A02 in Subjects With Amyotrophic Lateral Sclerosis (ALS)
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE3
Conditions: ALS
Lead sponsor: Mitsubishi Tanabe Pharma America Inc.
Sponsor class: INDUSTRY
First posted: 2021-12-09
Enrollment: 202
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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 time from the randomization date in Study MT-1186-A02 to at least a 12-point decrease in Revised ALS Functional Rating Score (ALSFRS-R) or death, whichever happens first, over the course of the study or until oral edaravone is commercially available in that country


Eligibility criteria: Inclusion Criteria:

1. Subjects or their legally authorized representative must provide a signed and dated informed consent form to participate in the study.
2. 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.
3. Subjects must be willing to cooperate and comply with all protocol restrictions and requirements.
4. Subjects must have successfully completed all Study MT-1186-A02 visits and have been compliant with study drug.

Exclusion Criteria:

1. Subjects of childbearing potential unwilling to use an acceptable method of contraception from the Day 1/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.
2. Subjects who are female, of childbearing potential, and pregnant (a positive pregnancy test) or lactating at the Day 1/screening visit.
3. 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 Columbia-Suicide Severity Rating Scale (C-SSRS) at Week 48 of Study MT-1186-A02.
4. Subjects who are not eligible to continue in the study, as judged by the Investigator in conjunction with the MTDA medical monitor.
5. Subjects who are unable to take their medications orally or through a PEG/RIG tube.

Primary outcomes:
measure: Time From the Randomization Date in Study MT-1186-A02 to at Least a 12-point Decrease in ALSFRS-R or Death, Whichever Happens First.
timeFrame: Up to 96 weeks
Secondary outcomes:
measure: The Combined Assessment of Function and Survival (CAFS) Score at All Visits From Baseline in Study MT-1186-A02 to the End of Study MT-1186-A04
timeFrame: Baseline, Week 72 and Week 96
measure: Change in the Amyotrophic Lateral Sclerosis Assessment Questionnaire 40 Score at All Visits From Baseline in Study MT-1186-A02 to the End of Study MT-1186-A04
timeFrame: Baseline, Week 72 and Week 96
measure: Change in ALSFRS-R Score at All Visits From Baseline in Study MT-1186-A02 to the End of Study MT-1186-A04
timeFrame: Baseline, Week 72, Week 84 and Week 96
measure: Time From the Randomization Date in Study MT-1186-A02 to Death, Tracheostomy, or Permanent Assisted Mechanical Ventilation (≥23 Hours/Day)
timeFrame: Up to 96 weeks
measure: Time From the Randomization Date in Study MT-1186-A02 to Death or Permanent Assisted Mechanical Ventilation (>23 Hours/Day)
timeFrame: Up to 96 weeks
measure: Time From the Randomization Date in Study MT-1186-A02 to Death
timeFrame: Up to 96 weeks
Other outcomes:
Locations:
city: Rogers
country: United States
facility: Woodland Research Northwest, LLC
state: Arkansas
city: Gainesville
country: United States
facility: UF Health Cancer Center/Clinical Trials Office
state: Florida
city: Atlanta
country: United States
facility: Emory University - School of Medicine
state: Georgia
city: Chicago
country: United States
facility: Northwestern University Feinberg School of Medicine
state: Illinois
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: Pittsburgh
country: United States
facility: University Of Pittsburgh Medical Center
state: Pennsylvania
city: Houston
country: United States
facility: Nerve And Muscle Center Of Texas
state: Texas
city: Seattle
country: United States
facility: University of Washington Medical Center
state: Washington
city: Morgantown
country: United States
facility: West Virginia University School of Medicine (WVUSoM) - Movement Disorder Clinic
state: West Virginia
city: Edmonton
country: Canada
facility: University of Alberta - Walter C Mackenzie Health Sciences Centre (WCM)
state: Alberta
city: Hamilton
country: Canada
facility: Health Science Center Mcmaster University
state: Ontario
city: London
country: Canada
facility: London Health Sciences Centre - University Hospital
state: Ontario
city: Quebec City
country: Canada
facility: CHU de Quebec-Hopital-Enfant-Jesus
state: Quebec
city: Würzburg
country: Germany
facility: Universitaetsklinikum Wuerzburg
city: Nagoya
country: Japan
facility: National Hospital Organization Higashinagoya National Hospital
state: Aichi
city: Nagoya
country: Japan
facility: Nagoya 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: Hiroshima-shi
country: Japan
facility: Hiroshima University Hospital
state: Hiroshima
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: Yokohama-shi
country: Japan
facility: Yokohama City University Hospital
state: Kanagawa
city: Koshi
country: Japan
facility: National Hospital Organization Kumamoto Saishun Medical Center
state: Kumamoto
city: Kyoto City
country: Japan
facility: National Hospital Organization Utano National Hospital
state: Kyoto
city: Sendai
country: Japan
facility: Tohoku University Hospital
state: Miyagi
city: Niigata-shi
country: Japan
facility: Niigata University Medical & Dental Hospital
state: Niigata
city: Osaka-shi
country: Japan
facility: Kansai Electric Power Hospital
state: Osaka
city: Toyonaka
country: Japan
facility: National Hospital Organization Osaka Toneyama Medical Center
state: Osaka
city: Shizuoka-city
country: Japan
facility: National Hospital Organization Shizuoka Institute of Epilepsy and Neurological Disorders
state: Shizuoka
city: Fuchu-city
country: Japan
facility: Tokyo Metropolitan Neurological Hospital
state: Tokyo
city: Itabashi-ku
country: Japan
facility: Teikyo University Hospital
state: Tokyo
city: Ota-ku
country: Japan
facility: Toho University Omori Medical Center
state: Tokyo
city: Saitama
country: Japan
facility: Saitama Neuropsychiatric Institute
city: Wangsimni-ro
country: Korea, Republic of
facility: Hanyang University Medical Center
state: Seongdong-gu
city: St.Gallen
country: Switzerland
facility: Chefarzt Muskelzentrum/ALS Clinic Kantonsspital St.Gallen Muskelzentrum/ALS Clinic
Study officials:
affiliation: Mitsubishi Tanabe Pharma America Inc.
name: Head of Medical Science
role: STUDY_DIRECTOR
References: