Atypical Hemolytic Uremic Syndrome (aHUS)


What is aHUS?

Atypical Hemolytic Uremic Syndrome (aHUS) is a rare, life-threatening, genetic disease. aHUS can damage vital organs such as the kidney, heart, and brain. In a person with aHUS, the complement system (part of the immune system) is uncontrolled. It is always "on" and can attack the body it normally protects.

aHUS is a genetic disease that is caused by a change, or mutation, in your DNA. This means that you will always have aHUS. These mutations make it difficult for your body to regulate, or “turn off,” the complement system.

When the complement system is overactive and does not shut off, the damage caused to the body is known as "thrombotic microangiopathy (TMA)."


    1. Noris M, Remuzzi G. Atypical hemolytic-uremic syndrome. N Engl J Med. 2009;361(17):1676-1687.

    2. Loirat C, Fremeaux-Bacchi V. Atypical hemolytic uremic syndrome. Orphanet J. Rare Dis. 2011;6:60.

    3. Kavanagh D, Goodship TH, Richards A. Atypical hemolytic uremic syndrome. Semin. Nephrol. 2013;33(6):508-530.

    4. Langman C. Systemic Multit-Organ Complications in Atypical Hemolytic Uremic Syndrome (aHUS): Retrospective Study in a Medical Practice Setting. In: European Hematology Association. Vol 97. s1. Amsterdam, The Netherlands: Haematologica; 2012:195-196.

    5. Noris M, Remuzzi G. Cardiovascular complications in atypical haemolytic uraemic syndrome. Nat Rev Nephrol 2014;10(3):174-180.


ID: NCT02949128

An investigation into a potential treatment for adults and adolescents with aHUS

The purpose of the study is to assess the efficacy of an investigational medication to control disease activity in adolescent and adult patients with aHUS who have not previously used a complement inhibitor.

If you are a patient who has been diagnosed with aHUS and are interested in information about the CHAMPION aHUS-311 study, you can email Alexion at Have a discussion with your physician if you are interested in participating in the CHAMPION aHUS-311 study.

For a location of the nearest study center:

Who is Eligible to Participate?

To participate in the CHAMPION aHUS-311 Study you must meet the following requirements:

Age: 12 years or older, weighing at least 40kg (88lbs)

Evidence of TMA: (thrombotic microangiopathy) including:

Lab tests that show decreased kidney function, low platelets and increased LDH (lactate dehydrogenase) indicating destruction of red blood cells

Recent meningococcal vaccine no more than 3 years prior to starting the study

If you are under 18, you will also require a vaccination against streptococcus pneumoniae and haemophilus influenzae

Female patients of childbearing potential must use highly effective contraception from the start, at screening, and continuing until at least 8 months after the last dose

If you have previously or are currently being treated with eculizumab, or a treatment that works in a similar way, you will NOT be eligible to participate in this study.

For a more detailed list of eligibility criteria, please go to

Download and print this checklist to review with your doctor

What Does Participation Involve?

The first step is a discussion with your physician about your interest in participating in the CHAMPION aHUS-311 study. Your physician can refer you or your adolescent child to a study site, where investigators will determine your eligibility to participate in the study. If eligible, the dosing of the investigational medication will be based on weight. The investigational medication will be given intravenously (IV) at a doctor’s office or infusion center for a period of 26 weeks.

There are potential side effects. The study doctor will discuss these with you and they will be described in the informed consent document you will be asked to sign prior to beginning the study.

Your condition will be carefully monitored throughout the study, with ongoing communication between the study doctor and your personal doctor.

Resources for aHUS-311