Open Label Controlled Trial of Eculizumab in Adult Patients With Plasma Therapy-Resistant aHUS
Open Label Controlled Trial of Eculizumab in Adult Patients With Plasma Therapy-Resistant aHUS
The purpose of this study is to determine whether eculizumab is safe and effective in the
 treatment of adult patients with plasma therapy-resistant Atypical Hemolytic-Uremic Syndrome
 (aHUS).
Exclusion Criteria:

 1. TTP, (defined as ADAMTS-13 activity <5%) from an historical observation (prior to
 initiation of plasma therapy) or as tested at the screening visit by the central
 laboratory

 2. Malignancy within 5 years of screening

 3. Typical HUS (Shiga toxin +)

 4. Known HIV infection

 5. Identified drug exposure-related HUS.

 6. Infection-related HUS

 7. HUS related to bone marrow transplant

 8. HUS related to vitamin B12 deficiency

 9. Renal function status requiring chronic dialysis

 10. Patients with a confirmed diagnosis of sepsis

 11. 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

 12. Pregnancy or lactation

 13. Unresolved meningococcal disease

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

 15. 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

 16. Patients who have received previous treatment with eculizumab

 17. Patients receiving IVIG within 8 weeks or Rituximab therapy within 12 weeks of
 screening.

 18. Patients receiving other immunosuppressive therapies such as steroids, mTOR inhibitors
 or tacrolimus are excluded unless: [1] part of an established post-transplant
 anti-rejection regime, [2] patient has confirmed anti-CFH antibody requiring
 immunosuppressive therapy, and [3] dose of such medications have been unchanged for at
 least 4 weeks prior to the screening period or [4] patient is experiencing an acute
 aHUS relapse immediately after transplant

 19. Patients receiving Erythrocyte Stimulating Agents (ESAs) unless already on a stable
 dose for at least 4 weeks prior to the screening period, or a washout period of at
 least 2 weeks from the last dose of ESA therapy.

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

 21. Hypersensitivity to eculizumab, to murine proteins or to one of the excipients