EUCTR2008-006955-28-SE进行中（未招募）不适用
AN OPEN-LABEL, MULTI-CENTER CONTROLLED CLINICAL TRIAL OF ECULIZUMAB IN ADOLESCENT PATIENTS WITH PLASMA THERAPY-SENSITIVE ATYPICAL HEMOLYTIC UREMIC SYNDROME (AHUS)
适应症
相关药物
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 15
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.
- •2. Patients must be receiving PT for aHUS and must be observed to (i) receive =1 PT
- •treatment every two weeks and no more than 3 PT treatments/week (at an unchanged
- •frequency) for at least 8 weeks before the first dose of Investigational Product.
- •3. Platelet Count Pre-PT Baseline Set-Point (collected in the hours before the
- •Qualifying PT Episode) is within 75% of the average of the pre-PT platelet counts
- •collected at Screening and during the Observation Period.
- •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 the trial:
- •(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-drugexposure-
- •related [e.g., cyclosporine]), no known HIV positivity, and antiphospholipid
- •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) at screening or at the onset of the current aHUS
- •episode was = 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.
- •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 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 must be willing to give written informed assent (if applicable as
- •determined by the IRB/IEC).
- •10. Able and willing to comply with study procedures.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- 另有 1 项未显示
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 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. 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.
- •10. 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.
- •11. Pregnancy or lactation.
- •12. Unresolved meningococcal disease.
- •13. Known Systemic Lupus Erythematosus (SLE) or antiphospholipid antibody
- •positivity or syndrome.
- •14. 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
- •15. Patients who have received previous treatment with eculizumab.
- •16. Patients receiving intravenous immunoglobulin (IVIg) within 8 weeks or Rituximab
- •therapy within 12 weeks of the screening visit.
- •17. 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 and
- •throughout the Observation Period or [3] patient is experiencing an acute aHUS
- •relapse immediately after transplant.
- •18. 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.
- •19. 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.
- •20. Hypersensitivity to eculizumab, to murine proteins, or to one of the excipients.
- •21. Patients between the ages from 12 and up to 18 years weighing < 40 kg.
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