The first participant has been enrolled in a global phase 3 study (NCT04165824) of an oral suspension formulation of edaravone (Radicava; Mitsubishi Tanabe Pharma, Jersey City, NJ) for potential treatment of amyotrophic lateral sclerosis (ALS).
In a prior clinical study, the intravenous (IV) formulation of edaravone slowed loss of physical function by 33% compared with placebo treatment. Loss of physical function was measured with the ALS Functional Rating Scale-Revised (ALSFRS-R).
This new study will evaluate 150 people, age 18 to 75 years with ALS, over a 48-week treatment period. After a screening period of up to 21 days, participants will receive oral edaravone in the same dosing regimen they used with the IV formulation. Exploratory endpoints will include change in ALSFRS-R score, change in percentage of forced vital capacity (FVC), and time (days) to tracheostomy or permanent assisted mechanical ventilation. Primary safety assessments will include adverse events, physical examination, electrocardiogram parameters, vital signs, laboratory safety assessments, unsteadiness, and sensory evaluations.
“Enrollment of the first patient in this clinical study marks an important milestone for our company as we continue to seek treatment options for the ALS community,” said Atsushi Fujimoto, president, Mitsubishi Tanabe Pharma America. “There is a great unmet need in the area of ALS, and we believe an oral formulation of edaravone could potentially serve as an alternative treatment option for patients suffering from this progressive condition.”