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 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 it causes to the body is known as "thrombotic microangiopathy (TMA).


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    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: NCT03131219

An investigation into a potential treatment for pediatric aHUS

The purpose of the CHAMPION aHUS-312 study is to assess the efficacy of an investigational medication to control disease activity in children and adolescents with aHUS who have not previously used a complement inhibitor.

If you are a parent or guardian of a child under the age of 18 who has been diagnosed with aHUS and are interested in information about this study, you can email Alexion at Have a discussion with your physician if you are interested in having your child participate in the CHAMPION aHUS-312 study.

For more information about this study:

Who is Eligible to Participate?

To participate in the CHAMPION aHUS-312 Study you must meet the following requirements:

Age: Under 18 years old, weighing at least 5kg (11lbs) at the time of consent

Evidence of TMA (thrombotic microangiopathy) including:

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

Must have had a vaccination not more than 3 years prior to entering the study, and vaccination against streptococcus pneumoniae and haemophilus influenzae

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

If your child has previously or is currently being treated with eculizumab, or a treatment that works in a similar way, the child will not be eligible to participate in this study. In addition, if your child has had a bone marrow transplant within the past 6 months they cannot participate in the study.

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 child participating in the CHAMPION aHUS-312 study. Your physician can refer you to a study site, where investigators will determine if your child is eligible to participate in the study. If eligible, during the treatment period the dosing of the investigational medication will be based on the child's 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.

The child's condition will be carefully monitored throughout the study, with ongoing communication between the study doctor and your personal doctor.

Resources for aHUS-312