Alexion Pharmaceuticals has revealed plans for initiating the CHAMPION-ALS clinical trial, a phase 3 study to evaluate ULTOMIRIS (ravulizumab) for the treatment of amyotrophic lateral sclerosis (ALS).
The late-stage clinical trial, which will be held globally by the US pharma company for 50 weeks, will feature nearly 350 adults across a broad patient population.
The primary endpoint of the CHAMPION-ALS trial will be a change in ALS functional rating scale-revised (ALSFRS-R) score.
Alexion Pharmaceuticals filed an investigational new drug application (IND) for ULTOMIRIS in ALS to the US Food and Drug Administration (FDA) in Q4 2019 and plans to launch the phase 3 clinical trial this quarter.
ULTOMIRIS is a C5 inhibitor which has been approved in the US, Japan and EU for the treatment of adult patients having paroxsymal nocturnal hemoglobinuria (PNH), and in the US for the treatment of atypical hemolytic uremic syndrome (aHUS) in adults and pediatric patients, aged one month and older.
Commenting on the CHAMPION-ALS clinical trial, John Orloff – Executive Vice President and Head of Research & Development at Alexion Pharmaceuticals, said: “Given the significant need for new and improved treatments for ALS, we are committed to advancing this clinical program with urgency.
“Based on preclinical data and the significant role complement activation is known to play in other neuromuscular diseases, we believe ULTOMIRIS has the potential to inhibit complement-mediated damage in people with ALS, which may slow disease progression. ”
The CHAMPION-ALS clinical trial will be a randomized, double-blind, placebo-controlled multicenter study, which will assess the efficacy and safety of ULTOMIRIS across a broad ALS population.
The adult patients to be enrolled in the clinical trial will have sporadic or familial ALS whose disease had begun within the prior 36 months. Also the patients should show a slow vital capacity (SVC) of at least 65% predicted, and do not need respiratory support.
Patients in the CHAMPION-ALS trial will be randomly grouped on a 2:1 basis to be subjected to ULTOMIRIS or placebo every eight weeks after an initial loading dose and could continue to have their existing standard of care treatment for ALS.
After 50 weeks, all the participating patients in the CHAMPION-ALS clinical study will be administered ULTOMIRIS in a two-year open-label extension phase of the trial. The ALS clinical trial will be held at nearly 90 clinical trial sites in North America, Europe, and Asia-Pacific.