The Safety and Efficacy of Eculizumab in Japanese Patients With Atypical Hemolytic Uremic Syndrome (aHUS)
The Safety and Efficacy of Eculizumab in Japanese Patients With Atypical Hemolytic Uremic Syndrome (aHUS)
Protocol is intended to characterize the overall safety and tolerability of eculizumab in
 this population.
Inclusion Criteria:

 1. All patients with diagnosis of aHUS who have been receiving eculizumab by personal
 importation (specific eligibility criteria below do not apply) Or,

 2. Patients with current clinical manifestations of aHUS who meet the following criteria:

 1. Patient with diagnosis of aHUS with or without an identified complement
 regulatory protein genetic abnormality or anti-complement factor antibody and for
 whom other known etiologies of hemolytic uremic syndrome (HUS) have been ruled
 out as confirmed in the Exclusion Criteria

 2. Patient (and legal guardian if patient is not an adult) willing and able to give
 written informed consent and assent (or verbal assent if patient is unable to
 read or write)

 3. Patient at least 1 month of age and body weight ≥5 kg

 4. Platelet count at screening < lower limit of normal (LLN)

 5. Signs or symptoms of hemolysis (i.e., lactate dehydrogenase (LDH) ≥ 1.5x upper
 limit of normal (ULN) and Hemoglobin ≤ LLN) at start of current aHUS event

 6. Serum Creatinine (SrCr) level ≥ ULN at screening (patient requiring dialysis for
 acute renal failure also eligible)

 7. Female patient of childbearing potential practicing an effective, reliable and
 medically approved contraceptive regimen during the entire duration of the study,
 including the Follow-up Period. At the time of the last follow-up visit, patient
 must agree to continue to use adequate contraception methods for up to 5 months
 following discontinuation of eculizumab treatment

 8. Able and willing to comply with study procedures

 Exclusion Criteria:

 Shiga-toxin producing E. coli-HUS (STEC-HUS; shiga-toxin and/or STEC positive) History of
 malignancy within 5 years of screening Known human immunodeficiency virus (HIV) infection
 Identified drug exposure-related HUS Infection-related HUS HUS related to bone marrow
 transplant (BMT) HUS related to vitamin B12 deficiency Known Systemic Lupus Erythematosus
 (SLE) or antiphospholipid antibody positivity or syndrome 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 7 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 Unresolved systemic
 meningococcal disease Any medical or psychological condition that, in the opinion of the
 investigator, could increase patient's risk by participating in the study or confound the
 outcome of the study Patients receiving chronic intravenous immunoglobulin (IVIG) within 8
 weeks unless for unrelated medical condition (e.g., hypogammaglobulinemia) or chronic
 rituximab therapy within 12 weeks of the screening visit Patients receiving other
 immunosuppressive therapies such as steroids, calcineurin inhibitors (mTOR), (e.g.,
 cyclosporine or tacrolimus) are excluded unless: [1] part of an established post-transplant
 anti-rejection regimen, or [2] patient has confirmed anti-Complement Factor Antibodies
 requiring immunosuppressive therapy or [3] steroids are used for a condition other than
 aHUS (e.g., asthma) Prior eculizumab use, hypersensitivity to eculizumab, to murine
 proteins or to one of the excipients Inclusion in any other investigational intervention
 trial except this study