EUCTR2008-006953-41-AT进行中（未招募）1 期
AN OPEN-LABEL, MULTI-CENTER CONTROLLED CLINICAL TRIAL OF ECULIZUMAB IN ADOLESCENT PATIENTS WITH PLASMA THERAPY-RESISTANT ATYPICAL HEMOLYTIC-UREMIC SYNDROME (AHUS)
适应症
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 7
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 between ages from 12 and up to 18 year weighing = 40 kg 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 screening.
- •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 genes;
- •- (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. Patient’s parents/legal guardian must be willing and able to give written informed
- •consent and patients mus
排除标准
- •1. Thrombotic Thrombocytopenic Purpura (TTP)
- •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 sensitive.
- •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 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.
- •22. Patients between the ages from 12 and up to 18 years weighing < 40 kg.
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