An Open-label, Multi-center Clinical Trial of Eculizumab in Adult Patients With Atypical Hemolytic-uremic Syndrome
An Open-label, Multi-center Clinical Trial of Eculizumab in Adult Patients With Atypical Hemolytic-uremic Syndrome
The record Primary purpose is to assess the efficacy of eculizumab in adult patients with
 Atypical Hemolytic- Uremic Syndrome (aHUS) to control Thrombotic Microangiopathy (TMA) as
 characterized by thrombocytopenia, hemolysis and renal impairment.
Inclusion:

 1. Patient must be willing and able to give written informed consent.

 2. Patient's age > 18 years.

 3. Patients exhibit thrombocytopenia, hemolysis and elevated Serum Creatinine.

 4. Patients with diagnosis of aHUS with or without an identified complement regulatory
 protein genetic abnormality or anti-complement factor antibody and for whom etiologies
 of hemolytic uremic syndrome have been ruled out as confirmed in the exclusion
 criteria

 5. Female patients of childbearing potential must be 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, patients must
 agree to continue to use adequate contraception methods for up to 5 months following
 discontinuation of eculizumab treatment.

 6. Able and willing to comply with study procedures

 Exclusion:

 1. Chronic dialysis.

 2. Prior eculizumab use or hypersensitivity to eculizumab, to murine proteins or to one
 of the excipients.

 3. Known familial a disintegrin and metalloproteinase with a thrombospondin type 1 motif,
 member 13 (ADAMTS-13) deficiency (ADAMTS-13 <5%).

 4. Typical Hemolytic-Uremic Syndrome (HUS) (known Shiga toxin +).

 5. History of malignancy within 5 years of screening.

 6. Known human immunodeficiency virus (HIV) infection.

 7. Identified drug exposure-related hemolytic-uremic syndrome (HUS).

 8. Infection-related HUS.

 9. HUS related to bone marrow transplant (BMT).

 10. HUS related to vitamin B12 deficiency.

 11. Known Systemic Lupus Erythematosus (SLE) or antiphospholipid antibody positivity or
 syndrome.

 12. 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.

 13. 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.

 14. Pregnancy or lactation.

 15. Unresolved systemic meningococcal disease.

 16. 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.

 17. Patients receiving chronic intravenous immunoglobulin (IVIg) within 8 weeks or chronic
 Rituximab therapy within 12 weeks of screening visit.

 18. Patients receiving other immunosuppressive therapies such as steroids, mechanist
 target of rapamycin (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
 requiring immunosuppressive therapy, or [3] steroids are being used for a condition
 other than aHUS (example asthma).

 19. Participation in any other investigational drug trial or device trial, or procedures
 beginning 4 weeks prior to screening and throughout the entire trial.