Global Enrollment Starts for Phase 3b Trial of Oral Edaravone
Note: This story was updated Oct. 19, 2021, to note that the trial is taking place at 90 clinical sites and to update the contact information, based on additional information from the company.
A Phase 3b clinical trial is recruiting adults with amyotrophic lateral sclerosis (ALS) to assess the safety and efficacy of two dosing regimens of oral edaravone.
The trial (NCT04569084), sponsored by Mitsubishi Tanabe Pharma America, the therapy’s developer, seeks to enroll 380 ALS patients (age 18 to 75), whose first symptoms developed in the two years prior to enrollment, according to a press release from the Muscular Dystrophy Association. Recruitment will take place at 90 clinical sites in the U.S., Canada, Germany, Italy, Switzerland, Japan, and South Korea.
Radicava — an intravenous formulation (given directly into the bloodstream) of edaravone, also developed by Mitsubishi Tanabe Pharma — was approved for ALS in the U.S. in 2017.
Previous clinical data showed that Radicava can safely and effectively slow functional decline in ALS patients. However, its dosing regimen requires long infusion periods (about one hour long) and frequent visits to the clinic. An oral formulation could therefore offer advantages over an intravenous formulation.
In the new trial, participants will be randomly assigned to a placebo or one of two doses of oral edaravone for a total of approximately 58 weeks (around 13 months). Participants will have to travel to the clinical site for eight visits, while the other visits will be conducted by telephone. Initially, patients will be monitored every four weeks and then every 12 weeks.
The trial’s main goal is to assess changes in patient functional status, assessed by the ALS functional rating scale-revised, at the end of the study.
Additional goals include the effects of oral edaravone on a patient’s well-being, including the impact of ALS symptoms on their daily life and any suicidal thoughts; their cardiac and respiratory function; their compliance with the therapeutic regimen; and measures of unsteadiness and sensory parameters.
Results are expected soon from another global Phase 3 trial (NCT04165824), which is fully enrolled and testing an oral suspension formulation of edaravone (MT-1186). Patients who finish this trial are eligible to enroll in the open-label long-term extension study (NCT04577404) where they will continue to receive the treatment for an extended period.
Edaravone works by reducing oxidative stress — an imbalance between the production of potentially harmful free radicals and the cells’ antioxidant defenses — considered one of the causes of nerve cell death in ALS.