An Open-Label, Multi-Center Clinical Trial of Eculizumab in Pediatric Patients With Atypical Hemolytic-Uremic Syndrome

Inclusion:

• Patient's parent/legal guardian must have been willing and able to give written informed consent and the patient must have been willing to give written informed assent (if applicable as determined by the central IRB/IEC).
• Pediatric patients with aHUS: Patients could have been newly diagnosed, or with previously diagnosed disease, or post-kidney transplant with the disease.
• Patients one month to 18 years and body weight ≥ 5kg.
• Platelet count at screening and baseline visit must have been below lower limit of normal ( 5 weeks prior to enrollment.
• Chronic dialysis (defined as dialysis on a regular basis as renal replacement therapy for end-stage renal disease [ESRD]).
• Patients with a confirmed diagnosis of sepsis defined as positive blood cultures within seven days of the screening visit and not treated with antibiotics to which the organism is sensitive.
• Presence or suspicion of active and untreated systemic bacterial infection that, in the opinion of the Investigator confounds an accurate diagnosis of aHUS or impedes the ability to manage the aHUS disease.
• Pregnancy or lactation.
• History of meningococcal/pneumococcal/gonococcal disease.
• Any medical or psychological condition that, in the opinion of the investigator, could increase the patient's risk by participating in the study or confound the outcome of the study.
• Patients receiving chronic intravenous immunoglobulin (IVIg) within eight weeks unless for unrelated medical condition (e.g., Hypogammaglobinemia), or chronic Rituximab therapy within 12 weeks of the screening visit.
• Patients receiving other immunosuppressive therapies such as steroids, mTOR inhibitors, calcineurin inhibitors (e.g., cyclosporine or tacrolimus are excluded unless: [1] part of an established post-transplant anti-rejection regime, or [2] patient has confirmed anti-Complement Factor antibodies antibody requiring immunosuppressive therapy or [3] steroids are being used for a condition other than aHUS (example asthma).
• Participation in any other investigational drug trial or device trial, or procedures beginning four weeks prior to screening and throughout the entire trial
• Prior use of eculizumab, hypersensitivity to eculizumab, to murine proteins or to one of the excipients.