Post-marketing trial of 2 Radicava ORS doses now fully enrolled
Study to test safety, efficacy of approved ALS therapy for long term
A Phase 3b study evaluating the long-term safety and effectiveness of two dosing regimens of Radicava ORS (edaravone) — an approved oral formulation of the medicine — in people with amyotrophic lateral sclerosis (ALS) is now fully enrolled.
The study was designed to fulfill a post-marketing commitment that Mitsubishi Tanabe Pharma America (MTPA) made to the U.S. Food and and Drug Administration (FDA) after the regulatory agency approved the into-the-vein (intravenous) formulation of Radicava in 2017.
The medication’s oral formulation, Radicava ORS, earned its U.S. approval in 2022.
“We’re very pleased to announce the completion of trial enrollment in our Phase 3b study of Radicava ORS, representing an important achievement in our ongoing commitment to the fight against ALS,” Atsushi Fujimoto, president of MTPA, said in a company press release.
“We are deeply grateful to the study participants and their families, as well as study investigators and clinical staff for working so diligently to help us meet this milestone,” Fujimoto added.
Phase 3b study testing 2 different doses of oral therapy
Radicava ORS is a liquid suspension taken either orally or via a feeding tube. As with the intravenous formulation, it is given in 28-day cycles.
The first cycle consists of daily treatment for 14 days followed by a two-week period (a second 14 days) without the medication. In subsequent cycles, Radicava is taken daily for 10 of the next 14 days, followed again by two weeks without treatment.
Approval of Radicava ORS was based on clinical trial data indicating that a 105 mg dose of the oral treatment delivers similar amounts of edaravone — Radicava’s active ingredient — to the body and is as good at slowing disease progression as the intravenous version (60 mg).
Now, the global MT-1186-A02 study (NCT04569084) is designed to compare the approved Radicava ORS on/off dosing regimen against once-daily dosing.
A total of 384 ALS patients, ages 18-75, whose symptoms emerged in the two years prior to the trial, were enrolled across 80 sites in the U.S., Canada, Europe, and Asia.
After an eight-week screening period, participants will either receive Radicava ORS every day, or be given the medication and a placebo. In the medication/placebo group, patients will receive Radicava ORS for 14 days and a placebo for 14 days in the first 28-day cycle, followed by 10 days of treatment and 18 days on a placebo for the next cycles.
The trial’s main goal is to evaluate changes in disease progression, as assessed by the ALS Functional Rating Scale-Revised (ALSFRS-R), over 48 weeks, or nearly one year. Secondary goals include changes in lung function and in measures of patient-reported quality of life, as well as the time patients live without requiring ventilatory support.
Top-line results from the trial, which already is underway, are expected in 2024.
“We look forward to seeing how the results of this study evaluating an alternative dosing regimen for Radicava ORS build upon our existing data to support its long-term efficacy and safety profile for people living with ALS, and how it may increase our understanding of the full potential of the medication as a treatment option,” said Michael D. Weiss, MD, of the University of Washington Medical Center, who is the study’s lead investigator.
Radicava works by combating oxidative stress, a type of cellular damage that’s been implicated in ALS. In doing so, it’s expected to protect nerve cells from damage and slow disease progression.
Intravenous Radicava also is approved in Japan, South Korea, Canada, and Switzerland, among other countries. The oral version has approvals in the U.S., Canada, and Japan.
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