Safety Study of Oral Edaravone Administered in Subjects With ALS

• ClinicalTrials.gov Identifier: NCT04165824
• Recruitment Status: Recruiting
• First Posted: November 18, 2019
• Last Update Posted: May 18, 2020
• Sponsor: Mitsubishi Tanabe Pharma Development America, Inc.
• Information provided by (Responsible Party): Mitsubishi Tanabe Pharma Development America, Inc.

Study Description

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.

Condition or disease	Intervention/treatment	Phase
ALS	Drug: MT-1186	Phase 3

Study Design

• Study Type: Interventional (Clinical Trial)
• Estimated Enrollment: 150 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Safety Study of Oral Edaravone Administered in Subjects With ALS
• Actual Study Start Date: November 18, 2019
• Estimated Primary Completion Date: June 2021
• Estimated Study Completion Date: June 2021

Arms and Interventions

Arm	Intervention/treatment
Experimental: MT-1186	Drug: MT-1186; An initial treatment cycle with daily dosing for 14 days, followed by a 14-day drug free period; Subsequent treatment cycles with daily dosing for 10 days out of a 14-day period, followed by a 14 day drug free period. Treatment cycles are every 4 weeks.; Other Name: Oral edaravone

Outcome Measures

Primary Outcome Measures :

1. Frequency and incidence of Treatment Emergency Adverse Events [ Time Frame: up to 48 Weeks ]

Other Outcome Measures:

1. Change in ALS Functional Rating Scale - Revised from baseline [ Time Frame: up to 48 Weeks ]
2. Time to event(death, tracheostomy, gastrostomy tube placement and permanent assisted mechanical ventilation) [ Time Frame: up to 48 Weeks ]

Eligibility Criteria

• Ages Eligible for Study: 18 Years to 75 Years (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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.

Contacts and Locations

Contacts

Contact: Clinical Trials Information Desk, to prevent miscommunication,; please email:; information@mt-pharma-us.com

Locations

United States, Arizona

Neuromuscular Research Center; Recruiting

Phoenix, Arizona, United States, 85028

United States, Florida

Alleghany General Hospital; Recruiting

Tampa, Florida, United States, 33616

United States, Georgia

Emory University - School of Medicine; Recruiting

Atlanta, Georgia, United States, 30317-2819

United States, Minnesota

Essentia Institute of Rural Health; Recruiting

Duluth, Minnesota, United States, 55805

United States, Nebraska

Neurology Associates, P.C - Lincoln; Recruiting

Lincoln, Nebraska, United States, 68506-2960

United States, North Carolina

Wake Forest University Baptist Medical Center (WFUBMC) - The J. Paul Sticht Center on Aging and Rehabilitation; Recruiting

Winston-Salem, North Carolina, United States, 27157-0001

United States, Texas

Texas Neurology, PA; Recruiting

Dallas, Texas, United States, 75214

Houston Methodist Neurological Institute; Recruiting

Houston, Texas, United States, 77030

UT Health Science Center San Antonio; Recruiting

San Antonio, Texas, United States, 78229

United States, Wisconsin

Medical College of Wisconsin; Recruiting

Milwaukee, Wisconsin, United States, 53226

Sponsors and Collaborators

Mitsubishi Tanabe Pharma Development America, Inc.

Investigators

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

More Information

• Responsible Party: Mitsubishi Tanabe Pharma Development America, Inc.
• ClinicalTrials.gov Identifier: NCT04165824
• Other Study ID Numbers: MT-1186-A01
• First Posted: November 18, 2019
• Last Update Posted: May 18, 2020
• Last Verified: May 2020

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: Undecided

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

Additional relevant MeSH terms:

Edaravone; Free Radical Scavengers; Antioxidants; Molecular Mechanisms of Pharmacological Action; Neuroprotective Agents; Protective Agents; Physiological Effects of Drugs