NL-OMON33473已完成2 期
An Open-Label, Multi-Center Controlled Clinical Trial of Eculizumab in Adult Patients with Plasma Therapy-Sensitive Atypical Hemolytic-Uremic Syndrome (AHUS) - C08-003A
适应症
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 1
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 18 至 99（—）
入选标准
- •1. Male or female patients *18 years of age who have been diagnosed with Atypical Hemolytic-Uremic Syndrome (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 immediately prior to first dose of Investigational Product and (ii) receive the same volume of PP or PE and units of FFP for at least 8 weeks immediately prior to first dose of Investigational Product.
- •3. Platelet Count Pre-PT Baseline Set-Point (collected immediately prior to 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 1 (MCP-1) 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) Complement Protein 3 or factor H or factor I functional
- •deficiency or abnormal factor interaction (C3/CFH/CFI FFP Group);
- •* (Category 2) Factor B Gain of Function ;
- •* (Category 3) Anti-CFH Antibody (Anti-CFH Group);
- •* (Category 4) MCP-1 deficiency (MCP-1 Group);
- •5. Patients diagnosed with HUS of the atypical type without documented complement regulatory protein genetic abnormality or known anti-CFH antibody are eligible if other etiologies of 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 HIV positivity, and anti-phospholipid antibody negative). Thrombotic thrombocytopenic purpura also must be ruled out (i.e., ADAMTS-13 activity must be > 5%; see Exclusion Criteria). 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 * ULN.
- •7. Creatinine level * ULN for age.
- •8. Sexually active women of childbearing potential must be practicing an effective,
- •reliable and medically acceptable contraceptive regimen during the entire duration of the study, including the follow-up period.
- •9. Able to give written informed consent.
- •10. Able and willing to comply with study procedures.
排除标准
- •1. ADAMTS-13 inhibitor or deficiency (i.e., ADAMTS-13 activity <5%) as measured at
- •the screening visit.
- •2. Malignancy.
- •3. Typical HUS (Shiga toxin +).
- •4. Known HIV infection.
- •5. Identified drug exposure-related HUS.
- •6. Infection-related HUS.
- •7. 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.
- •8. 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.
- •9. Pregnancy or lactation.
- •10. Unresolved meningococcal disease.
- •11. Known Systemic Lupus Erythematosus (SLE) or antiphospholipid antibody positivity or syndrome.
- •12. 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.
- •13. Patients receiving IVIg or Rituximab therapy.
- •14. Patients receiving other immunosuppressive therapies such as steroids, mTOR
- •inhibitors or FK506 inhibitors are excluded unless: [1] part of a post-transplant antirejection 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.
- •15. Patients receiving Erythrocyte Stimulating Agents (ESAs) unless already on a stable dose for at least 4 weeks prior to the screening period.
- •16. 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.
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