Phase 3 Study of Oral Edaravone for ALS Resumes Patient Enrollment

Patricia Inácio, PhD avatar

by Patricia Inácio, PhD |

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ION541 trial dosing

The Phase 3 clinical trial testing an oral suspension formulation of edaravone for amyotrophic lateral sclerosis (ALS) has resumed dosing and patient recruitment, its developer, Mitsubishi Tanabe Pharma America (MTPA), announced.

The trial (NCT04165824) was launched in November, but was paused in March due to the COVID-19 pandemic.

The study, recruiting up to 150 adults with ALS (ages 18 to 75 years), is taking place at 65 clinical sites in the U.S., Canada, Europe, and Japan. Those interested in participating can find information about locations and contacts here, or by calling 800-313-9381.

“MTDA is taking a thoughtful, cautious approach to resuming this study, including working closely with the investigator sites to implement appropriate COVID-19 protocols,” Atsushi Fujimoto, president of MTPA, said in a press release. “We are extremely pleased to be able to announce that the study has resumed enrollment.”

Designed as an open-label (no placebo group) study, it intends to evaluate whether an oral formulation of edaravone is safe and effective in treating ALS.

An intravenous form of edaravone (given directly into the bloodstream), developed by MTPA and sold under the brand name Radicava, was approved as an ALS treatment by the U.S. Food and Drug Administration (FDA) in 2017.

Edaravone reduces oxidative stress — an imbalance between the production of potentially harmful free radicals and the cells’ antioxidant defenses — which is believed to be one of the causes of nerve cell death in ALS.

Previous clinical studies have shown that Radicava can safely and effectively slow functional decline in people with ALS. The recommended dosing of Radicava (60 mg over 60 minutes) is administered for 14 consecutive days, followed by a two-week treatment-free period. Subsequent treatment cycles consist of daily treatment for 10 of the next 14 days, followed by two weeks without treatment.

After a screening period of up to 21 days, all enrolled patients in the Phase 3 trial will receive oral edaravone — at the same dosing regimen of the intravenous formulation — over 48 weeks.

The study’s main (primary) goal is to assess the safety of oral edaravone, including evaluations of adverse events (side effects), laboratory assessments, and physical and sensory examinations.

Additional (secondary) goals include changes in functional disability, as measured by the ALS functional rating scale-revised (ALSFRS-R); lung function, as measured by forced vital capacity (the amount of air that can be forcibly exhaled from the lungs after taking the deepest breath possible); and the time to invasive procedures needed to prevent respiratory failure or malnutrition (tracheostomy, permanent assisted mechanical ventilation, or gastrostomy tube placement).