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临床试验/EUCTR2008-006952-23-SE
EUCTR2008-006952-23-SE进行中（未招募）不适用

AN OPEN-LABEL, MULTI-CENTER CONTROLLED CLINICAL TRIAL OF ECULIZUMAB IN ADULT PATIENTS WITH PLASMA THERAPY-RESISTANT ATYPICAL HEMOLYTIC-UREMIC SYNDROME (AHUS)

ALEXION PHARMACEUTICALS, INC.0 个研究点目标入组 8 人开始时间: 2009年3月4日最近更新:
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试验速览

阶段
不适用
状态
进行中（未招募）
入组人数
8
研究概览入排标准研究者记录与标识符相似试验

研究概览

简要总结

暂无简介。

研究设计

研究类型
Interventional clinical trial of medicinal product

入排标准

性别
All

入选标准

  • •1. Male or female patients = 18 years who have been diagnosed with aHUS. Patients may
  • •be newly diagnosed, experiencing a relapse of the disease, or having a post-transplant
  • •recurrence of the disease.
  • •2. Patients must exhibit a decrease in platelet count despite at least 4 PT treatments in the 1
  • •week immediately prior to screening. At screening, platelet count must be < 150 x
  • •109/L and at least 25% lower than the average of 3 platelet counts obtained during the
  • •most recent TMA remission and at least 1 month apart during that remission prior to
  • •screening (designated the average remission platelet count”).
  • •3. If historical counts are not available, platelet count at onset of the current aHUS episode
  • •must be = 75 x 109/L, and platelet count at screening must be = 100 x 109/L despite PT
  • •treatment administration of at least 4 PT treatments in the 1 week immediately prior to
  • •4. Known complement regulatory protein genetic abnormality, i.e., a mutation in
  • •Complement Protein 3, factor H or associated factor, factor I, or membrane cofactor
  • •protein (MCP) or known Factor B gain-of-function mutation, or known Anti-CFH
  • •antibody (aHUS lesions”).
  • •Patients diagnosed with aHUS with any of these aHUS lesions are eligible and will
  • •be assigned to one of the following parallel categories during the treatment period of
  • •- (Category 1) Factor H or factor I functional deficiency, abnormal factor
  • •interaction (CFH/CFI FFP Group), or deletions of the CFHR1 and CFHR3
  • •- (Category 2) Complement Protein 3, abnormal factor interaction (C3) or Factor
  • •B Gain of Function;
  • •- (Category 3) Anti-CFH Antibody (anti-CFH Group);
  • •- (Category 4) MCP deficiency (MCP Group);
  • •5. Patients diagnosed with aHUS without documented complement regulatory protein
  • •genetic abnormality or known anti-CFH antibody are eligible if other etiologies of
  • •hemolytic uremic syndrome (HUS) have been ruled out as confirmed in the Exclusion
  • •Criteria (i.e., including Shiga-toxin negative, non-infectious, non-drug-exposure-related
  • •[e.g., cyclosporine]), no known human immunodeficiency syndrome (HIV) positivity,
  • •and anti-phospholipid antibody negative). Patients meeting these conditions will be
  • •assigned to Category 5. In addition, these patients will undergo genetic testing to
  • •determine if a mutation can be identified. If a mutation is identified, the patient will be
  • •reassigned to the appropriate category.
  • •6. Lactate dehydrogenase (LDH) level = upper limit of normal (ULN) unless the patient
  • •has been receiving plasma exchange (PE) and LDH at the onset of the current aHUS
  • •episode was at least the ULN. If LDH is normal at screening, other markers indicative
  • •of ongoing hemolysis should be evaluated, such as haptoglobin, schistocytes, and
  • •discussed with the Sponsor;
  • •7. Creatinine level = ULN for age (patients requiring acute dialysis for acute renal failure
  • •also eligible).
  • •8. 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 will be
  • •counseled by the principal investigator (PI) or designated study staff that they must
  • •continue to use adequate contraception methods for up to 5 months following
  • •discontinuation of eculizumab treatment.
  • •9. 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) y

排除标准

  • •1. Thrombotic Thrombocytopenic Purpura (TTP), defined as ADAMTS13 activity <5% from an historical observation (prior to initiation of Plasma Therapy) or as tested at the screening visit by the central laboratory.
  • •2. History of malignancy within 5 years of screening.
  • •3. Typical HUS (known Shiga toxin +).
  • •4. Known HIVinfection.
  • •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. Renal function status requiring chronic dialysis (defined as dialysis on a regular basis as
  • •renal replacement therapy).
  • •10. 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
  • •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
  • •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
  • •16. Patients who have received previous treatment with eculizumab.
  • •17. Patients receiving intravenous immunoglobulin (IVIg) within 8 weeks or Rituximab
  • •therapy within 12 weeks of the screening visit.
  • •18. Patients receiving other immunosuppressive therapies such as steroids, calcineurin
  • •inhibitors (mTOR) or tacrolimus are excluded unless: [1] part of an established posttransplant
  • •anti-rejection regime and dose of such medications have been unchanged for
  • •at least 4 weeks prior to the screening period, or [2] patient has confirmed anti-CFH
  • •antibody requiring immunosuppressive therapy and dose of such medications have been
  • •unchanged for at least 4 weeks prior to the screening period or [3] 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.

研究者

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