A phase 3 trial evaluating ravulizumab (Ultomiris) in adults with amyotrophic lateral sclerosis (ALS) has been discontinued.

The multicenter, randomized, double-blind, placebo-controlled CHAMPION-ALS trial (ClinicalTrials.gov Identifier: NCT04248465) evaluated the efficacy and safety of ravulizumab, a long-acting complement inhibitor, in 382 adults with sporadic or familial ALS who had disease onset within the prior 36 months. Patients were randomly assigned 2:1 to receive either ravulizumab or placebo via intravenous infusion every 8 weeks following an initial loading dose. The primary endpoint was the change in ALS function rating scale-revised score.

Following a prespecified interim analysis, the decision to halt the study was made based on the recommendation from the Independent Data Monitoring Committee due to a lack of efficacy. The safety profile of ravulizumab was consistent with its established profile and no new safety data was observed. Patients who are enrolled in the trial will discontinue ravulizumab and will complete any necessary follow-up evaluations. 

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Gianluca Pirozzi, MD, PhD, Senior Vice President, Head of Development and Safety, Alexion, said, “We are disappointed by this outcome and what it means for patients with this devastating disease. We would like to thank the entire ALS community as well as investigators and healthcare professionals who dedicated their time and expertise to this trial. We continue to be confident in the potential of targeting C5 for complement-driven diseases and remain fully committed to our efforts to serve the rare disease community.”  


Update on CHAMPION-ALS phase III trial of Ultomiris in amyotrophic lateral sclerosis. News release. AstraZeneca. Accessed August 20, 2021. https://www.astrazeneca.com/media-centre/press-releases/2021/update-on-ultomiris-phase-iii-als-trial.html

This article originally appeared on MPR