Ravulizumab not efficacious in ALS

Key takeaways:

• Ravulizumab did not improve functional status or survival in patients with ALS.
• The phase 3 trial ended early due to “futility,” researchers said.

Researchers reported no differences in functional status or survival in patients with ALS treated with ravulizumab compared with placebo, according to a poster presented at the 2023 MDA Clinical & Scientific Conference.

“Given the unmet need for highly efficacious ALS treatments and the favorable benefit-risk profile of the anti-C5 antibody ravulizumab in other complement-mediated neurological diseases, we proceeded directly to a phase 3 clinical trial to evaluate the efficacy and safety of ravulizumab in ALS,” Glen Frick, MD, PhD, of AstraZeneca/Alexion Rare Disease, and colleagues wrote.

In the global, double-blind CHAMPION-ALS study, researchers randomly assigned adults with ALS who had received meningococcal vaccination 2:1 to ravulizumab (n = 255) or placebo (n = 127) for 50 weeks. The primary outcome was change in functioning, measured via the Combined Assessment of Function and Survival (CAFS). Secondary outcomes included functional and pharmacokinetic/pharmacodynamic measures, as well as prevalence of treatment-emergent adverse events.

After a planned interim analysis when 33% of participants reached week 26, the researchers ended the trial “for futility.” At that time, 15 participants on ravulizumab and five receiving placebo had reached the end of the 50-week trial period.

There were no significant differences in CAFS scores between groups, the researchers reported.

“Additionally, there were no observed differences in key efficacy-related secondary outcome measures and no notable differences in treatment-emergent adverse event rates between treatment groups,” Frick and colleagues wrote.