ALS News Today recently announced the introduction of a Phase 3 clinical trial for oral edaravone, a drug that has previously been approved for slowing the decline of physical and/or cognitive functioning in amyotrophic lateral sclerosis (ALS) in an intravenous form.
First Approval by the FDA
Edaravone, under the brand name Radicava, is infused into the bloodstream (intravenous). It was sanctioned in 2017 as the first treatment approved by the FDA for ALS in twenty years.
Mitsubishi Tanabe Pharma (MPTA) developed the formula which is sold as Radicava.
About Edaravone
Edaravone effectively reduces oxidative stress which is characterized by the imbalance between the antioxidant defenses of the cells and the production of free radicals.
Scientists worldwide are in agreement that free radical damage is instrumental in the destruction of brain neurons and more specifically that it causes the death of nerve cells in ALS.
The current recommended dosing for edaravone, which was approved in 2017, is 60mg intravenously for a period of 60 minutes. The initial cycle consisted of treatments for fourteen consecutive days then two weeks free of treatment.
Cycles from that point on consisted of ten days with the following two weeks treatment free. The treatment was administered for twenty-four to forty-eight weeks.
About the Phase 3 Clinical Trial
The first patient to receive the new oral version of edaravone has been enrolled in MTPA’s trial.
The safety analysis will address adverse events, sensory and physical examinations, and laboratory assessments.
Its secondary goal is to evaluate changes to functional disability and lung function. Researchers will measure the length of time until the patient needs invasive procedures such as a gastrostomy tube, tracheostomy, or assistance with mechanical ventilation.
The dosing regimen will be the same as in the intravenous formula’s trial and have the same time frame cut-off of twenty-four to forty-eight weeks.
The trial will aim for a total enrollment of 150 adult patients between the ages of eighteen and seventy-five who have been diagnosed with ALS. The expected end date is June of 2021.
The trial is a global initiative across sixty-five sites. Half of the trial locations will be in the United States. It will be an open-label study meaning that the researchers and patients will know which medication is being administered.
