Complement Inhibition in aHUS Dialysis Patients (ACCESS)

Inclusion Criteria:

• Age >18 years;
• Diagnosis of aHUS with or without identified genetic abnormalities in the complement system or thrombomodulin;
• Stable chronic extracorporeal or peritoneal dialysis therapy since at least 6 months;
• Written informed consent.

Exclusion Criteria:

• Women of childbearing potential or women who are breastfeeding;
• Shiga toxin-associated HUS or secondary forms of thrombotic microangiopathy;
• ADAMTS13 activity <10 % or circulating anti ADAMTS13 autoantibodies consistent with the diagnosis of thrombotic thrombocytopenic purpura;
• Need for specific intervention with plasma therapy and/or complement inhibitors as deemed clinically appropriate;
• Plasma therapy or treatment with complement inhibitors or antiplatelet and antithrombotic agents over the last two weeks;
• Liver function impairment (serum liver enzymes or bilirubin levels >3 x upper limit of normal);
• Neutrophil count < 2000/μL or lymphocyte count < 1000/μL;
• Infection requiring antibiotic treatment within the previous 4 weeks prior to screening;
• Participated in any clinical study of an investigational product within 30 days prior to screening or within 5 half-lives after taking the last dose;
• History or presence of any medical condition or disease which, in the opinion of the Investigator may place the subject at unacceptable risk for study participation;
• Inability to understand the potential risks and benefits of the study;
• Legal incapacity.