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

• ClinicalTrials.gov Identifier: NCT05151471
• Recruitment Status: Recruiting
• First Posted: December 9, 2021
• Last Update Posted: November 28, 2022
• Sponsor: Mitsubishi Tanabe Pharma Development America, Inc.
• Information provided by (Responsible Party): Mitsubishi Tanabe Pharma Development America, Inc.

Tracking Information

• First Submitted Date: November 28, 2021
• First Posted Date: December 9, 2021
• Last Update Posted Date: November 28, 2022
• Actual Study Start Date: January 11, 2022
• Estimated Primary Completion Date: June 2024 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: January 25, 2022)

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. [ Time Frame: Up to 96 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.

• Original Primary Outcome Measures (submitted: November 28, 2021): 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. [ Time Frame: Up to 96 weeks ]

Current Secondary Outcome Measures (submitted: November 11, 2022)

• 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 [ Time Frame: Up to 96 weeks ]
  CAFS analysis ranks clinical outcomes on the basis of survival time and change in the ALS Functional Rating Scale-Revised (ALSFRS-R) score. A subject's score will be calculated by comparing each subject to every other subject within each treatment group in the study, resulting in a score of +1 if the outcome was better than the subject being compared, -1 if worse, and 0 if the same. The subject's score will then be calculated by summing up their comparison to all of the other subjects within each treatment group in the study as CAFS score. Patients' summary scores are ranked; a higher score indicates a better performance. The score range is from 1 to N, where N is the total sample size.
• 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 [ Time Frame: Up to 96 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).
• Change in ALSFRS-R score at all visits from baseline in Study MT-1186-A02 to the end of Study MT-1186-A04 [ Time Frame: Up to 96 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.
• Time from the randomization date in Study MT-1186-A02 to death, tracheostomy, or permanent assisted mechanical ventilation (≥23 hours/day) [ Time Frame: Up to 96 weeks ]
• Time from the randomization date in Study MT-1186-A02 to death or permanent assisted mechanical ventilation (>23 hours/day) [ Time Frame: Up to 96 weeks ]
• Time from the randomization date in Study MT-1186-A02 to death [ Time Frame: Up to 96 weeks ]

Original Secondary Outcome Measures (submitted: November 28, 2021)

• The Combined Assessment of Function and Survival (CAFS) score from baseline in Study MT-1186-A02 to Weeks 24 and 48 in Study MT-1186-A04 [ Time Frame: Up to 96 weeks ]
• Change in the Amyotrophic Lateral Sclerosis Assessment Questionnaire 40 score from baseline in Study MT-1186-A02 to Weeks 24 and 48 in Study MT-1186-A04 [ Time Frame: Up to 96 weeks ]

• Current Other Pre-specified Outcome Measures: Not Provided

Original Other Pre-specified Outcome Measures (submitted: November 28, 2021)

• Change in ALSFRS-R score from baseline in Study MT-1186-A02 to Weeks 12, 24, 36, and 48 in Study MT-1186-A04 [ Time Frame: Up to 96 weeks ]
• Time from the randomization date in Study MT-1186-A02 to death, tracheostomy, or permanent assisted mechanical ventilation (≥23 hours/day) [ Time Frame: Up to 96 weeks ]

Descriptive Information

• Brief Title: 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)
• 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
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 3
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor); Primary Purpose: Treatment
• Condition: ALS

Intervention

• Drug: MT-1186
  Oral edaravone
  Other Name: Oral edaravone
• Drug: Placebo
  Oral
  Other Name: Oral

Study Arms

• Experimental: MT-1186 - Group 1
  Oral edaravone administered once daily for up to 48 weeks or until the drug is commercially available in that country.
  Intervention: Drug: MT-1186
• Experimental: MT-1186 - Group 2
  Oral edaravone administered for 10 days followed by 18-day placebo (regimen denoted as on/off) for up to 48 weeks or until the drug is commercially available in that country.
  Interventions:

  • Drug: MT-1186
  • Drug: Placebo

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Recruiting
• Estimated Enrollment (submitted: November 28, 2021): 300
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: June 2024
• Estimated Primary Completion Date: June 2024 (Final data collection date for primary outcome measure)

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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Contacts

Contact: Clinical Trials Information Desk, to prevent miscommunication,; Please E-mail; information@mt-pharma-us.com

• Listed Location Countries: Canada, Germany, Japan, Korea, Republic of, Switzerland, United States
• Removed Location Countries: United Kingdom

Administrative Information

• NCT Number: NCT05151471
• Other Study ID Numbers: MT-1186-A04
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: Undecided

• Current Responsible Party: Mitsubishi Tanabe Pharma Development America, Inc.
• Original Responsible Party: Same as current
• Current Study Sponsor: Mitsubishi Tanabe Pharma Development America, Inc.
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

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

• PRS Account: Mitsubishi Tanabe Pharma Development America, Inc.
• Verification Date: November 2022