BACKGROUND: An intravenous (IV) formulation of edaravone (Radicava®/Radicut) was shown to slow the rate of physical functional decline in amyotrophic lateral sclerosis (ALS). This ongoing, multicenter, phase 3b, double-blind, parallel group, randomized study is currently evaluating Radicava ORS® (edaravone) oral suspension, which was recently approved by the US Food and Drug Administration for use in patients with ALS.
OBJECTIVES: Study MT-1186-A02 hypothesized that oral edaravone would demonstrate long-term safety, efficacy, and tolerability when evaluating and comparing 2 dosing regimens for a period of 48 weeks in patients with ALS.
METHODS: Study MT-1186-A02, which began in December 2020 and is estimated to complete in December 2023, will equally randomize patients into 2 treatment groups. Group 1 will have oral edaravone (105-mg dose) administered once daily for 28 days for 12 cycles, and group 2 will have oral edaravone administered for 14 days, followed by placebo for 14 days in Cycle 1. Subsequently, in group 2, oral edaravone will be administered for 10 days followed by placebo for 18 days in Cycles 2-12.
Study MT-1186-A02 is anticipated to include approximately 380 adult patients. Patients must be diagnosed with definite or probable ALS, have a baseline forced vital capacity ≥70%, and baseline disease duration ≤2 years. The primary objective is to evaluate the efficacy of each dosing regimen based on the changes from baseline to week 48 in the revised ALS Functional Rating Scale score. Secondary objectives will evaluate the safety and tolerability of each dosing regimen. Exploratory objectives include investigation of changes in nerve conduction tests and biomarkers.
CONCLUSIONS: This study will provide important information on the safety, efficacy, and tolerability of 2 dosing regimens for oral edaravone in patients with ALS.
SPONSORSHIP: Mitsubishi Tanabe Pharma Development America, Inc., and Mitsubishi Tanabe Pharma America, Inc.
ACKNOWLEDGEMENTS: p-value communications provided editorial support.