An open-label, multi-center clinical trial of eculizumab in adult patients with atypical hemolytic-uremic syndrome - ND

Conditions: Adult patients with Atypical Hemolytic Uremic Syndrome (aHUS)
MedDRA version: 9.1Level: LLTClassification code 10019515

Registration Number: EUCTR2010-020326-18-IT

Lead Sponsor: ALEXION PHARMACEUTICALS

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 30

Inclusion Criteria

1. Patient must be willing and able to give written informed consent. 2. Patient’s age = 18 years. 3. Platelet count at screening must be < lower limit of normal range (LLN) 4. Signs or symptoms of hemolysis (i.e, LDH=1.5 ? upper limit or normal range (ULN) and hemoglobin = LLN) at start of current aHUS event. 5. Serum Creatinine (SrCr) level = ULN (patients requiring dialysis for acute renal failure are also eligible). 6. Patients with diagnosis of aHUS with or without an identified complement regulatory protein genetic abnormality or anti-complement factor antibody and for whom known etiologies of hemolytic uremic syndrome (HUS) have been ruled out as confirmed in the Exclusion Criteria. 7. 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. 8. Able and willing to comply with study procedures
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Known familial ADAMTS-13 deficiency (ADAMTS-13<5%) 2. Typical HUS (known Shiga toxin +). 3. History of malignancy within 5 years of screening. 4. Known human immunodeficiency virus (HIV) infection. 5. Identified drug exposure-related HUS. 6. Infection-related HUS. 7. HUS related to bone marrow transplant (BMT). 8. HUS related to vitamin B12 deficiency. 9. Known Systemic Lupus Erythematosus (SLE) or antiphospholipid antibody positivity or syndrome. 10. Chronic dialysis (defined as dialysis on a regular basis as renal replacement therapy for end-stage renal disease). 11. 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. 12. 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. 13. Pregnancy or lactation. 14. Unresolved systemic meningococcal disease. 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 receiving chronic intravenous immunoglobulin (IVIg) within 8 weeks (e.g., hypogammaglobinemia). or chronic rituximab therapy within 12 weeks of the screening visit unless for unrelated medical condition 17. 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 requiring immunosuppressive therapy or [3] steroids are being used for a condition other than aHUS (example asthma). 18. Participation in any other investigational drug trial or device trial, or procedures beginning 4 weeks prior to screening and throughout the entire trial. 19. Prior eculizumab use, hypersensitivity to eculizumab, to murine proteins or to one of the excipients