Phase 3, Open-Label, Safety Extension Study of Investigational Oral Edaravone Administered Over 96 Weeks in Patients with ALS (MT-1186-A03)

Topic:

Clinical Trials

Poster Number: Virtual

Author(s):

Daniel Selness, RN, BA, MBA, Mitsubishi Tanabe Pharma Development America, Inc., Manabu Hirai, MS, Mitsubishi Tanabe Pharma Corporation, Takeshi Sakata, MS, Mitsubishi Tanabe Pharma Corporation, Arthur Wamil, MD, PhD, Mitsubishi Tanabe Pharma Development America, Inc., Alejandro Salah, MD, PhD, MBA, BCMAS, Mitsubishi Tanabe Pharma America, Inc., Stephen Apple, MD, MD, Mitsubishi Tanabe Pharma America, Inc.

Background: Radicava® (edaravone injection) is a US FDA-approved treatment for amyotrophic lateral sclerosis (ALS), shown to slow the rate of physical functional decline. The ongoing, multicenter, phase 3 studies are currently assessing the safety and tolerability of an investigational oral formulation of edaravone (MT-1186) due to interest in a non-intravenous (IV) formulation.
Objectives: To assess the continued long-term safety and tolerability of investigational oral edaravone in patients with ALS.
Methods: Study MT-1186-A03 is an extension long-term safety and tolerability study for patients who complete MT-1186-A01. Patients who continue to meet the enrollment criteria will be eligible for enrollment into the MT-1186-A03 study and will continue to receive oral edaravone for an additional treatment period of 96 weeks.
Patients will continue to receive a 105-mg dose of MT-1186 administered in treatment cycles that replicate the dosing of IV edaravone. In addition to the primary safety analysis, Study MT-1186-A03 also includes exploratory end points, such as change from baseline in the revised ALS Functional Rating Scale (ALSFRS-R) score and time to death, tracheostomy, or permanent assisted mechanical ventilation.
It is anticipated that Study MT-1186-A03 will include approximately 130 patients. The primary objective of this extension study is to evaluate the continued long-term safety of MT-1186, and the study includes exploratory efficacy end points in common with Study MT-1186-A01.
Results: Ongoing.
Conclusions: This extension study will provide important information on the continued long-term safety and tolerability of this investigational oral formulation of edaravone in patients with ALS.