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.
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.