MND Australia


Phase 3


The CHAMPION ALS Study is 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 is sponsored by Alexion Pharmaceuticals.

What is involved?

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

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 recognize 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.

How far off is a treatment?

Ravulizumab is an investigational medication not currently approved for use in patients with ALS in any country worldwide. A possible treatment resulting from this study is likely years away.

Contact details

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

Further information is available here