MND Australia


Phase 3 complete


The CHAMPION ALS Study was a Phase III, double-blind, randomised, placebo-controlled, parallel-group, multicentre study with an open-label extension period, to evaluate the efficacy and safety of ravulizumab (also known as Ultomiris) in adult patients with amyotrophic lateral sclerosis (ALS). The study was sponsored by Alexion Pharmaceuticals.

What is Ultomiris?

Ultomiris contains an antibody that is injected into the bloodstream and that inhibits a protein called C5, a member of the complement system. The complement system is part of the immune system and is thought to be implicated in neurodegeneration in ALS.

Under normal conditions, the complement system acts as part of the innate immune system to enhance the function of antibodies and the immune cells that recognise threats like infections. The complement system is activated at a low level at all times. Under some circumstances, however, an ALS-triggering event, for example, such as increased inflammation around nerve cells in the brain, the complement system overreacts.

By inhibiting C5, Ultomiris interrupts the overreaction of the complement system. In that way, the therapy may be able to slow disease progression in ALS.

The Phase 3 Champion trial

Treatment was delivered intravenously (into-the-vein) every eight weeks for a total of 50 weeks, after which all patients would have received Ultomiris in a two-year open-label extension phase. Patients were allowed to continue to receive their standard of care treatment for ALS.

The CHAMPION-ALS Phase III trial was a randomised, double-blind, placebo-controlled multicentre global trial designed to evaluate the efficacy and safety of Ultomiris across a broad ALS population. The trial enrolled 382 adults with sporadic or familial ALS who had disease onset (in the form of first motor symptoms) within the prior 36 months, demonstrated a slow vital capacity (SVC) of at least 65 percent predicted, and were not dependent on respiratory support. The primary endpoint was change in ALS functional rating scale-revised (ALSFRS-R) score. Trial participants were randomised on a 2:1 basis to receive Ultomiris or placebo every eight weeks following an initial loading dose. The trial was conducted at approximately 90 clinical trial sites across North America, Europe and Asia.

How far off is a treatment?

Alexion announced on the 20th August 2021 that it was discontinuing CHAMPION-ALS in adults with amyotrophic lateral sclerosis (ALS).

This decision is based on the recommendation of the Independent Data Monitoring Committee (IDMC), following their review of data from a pre-specified interim analysis. The IDMC recommended that the trial be discontinued due to lack of efficacy. No new safety findings were observed and the data were consistent with the established safety profile of Ultomiris.

Contact details

Any questions from clinicians can be directed to Alexion’s Medical Information department on 1800 788 189 or

Further information is available here