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临床试验/NCT01193348
NCT01193348已完成2 期

An Open-Label, Multi-Center Clinical Trial of Eculizumab in Pediatric Patients With Atypical Hemolytic-Uremic Syndrome

Alexion Pharmaceuticals, Inc.0 个研究点目标入组 22 人开始时间: 2010年9月最近更新:
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适应症
干预措施
相关药物

试验速览

阶段
2 期
状态
已完成
入组人数
22
主要终点
Proportion of Patients With Complete TMA Response
研究概览研究设计入排标准研究组 & 干预措施结局指标研究者记录与标识符相似试验

研究概览

简要总结

The primary purpose is to assess the efficacy and safety of eculizumab in pediatric patients with aHUS to control TMA as characterized by thrombocytopenia, hemolysis and renal impairment.

研究设计

研究类型
Interventional
分配方式
Non Randomized
干预模型
Single Group
主要目的
Treatment
盲法
None

入排标准

年龄范围
1 Month 至 18 Years（Child, Adult）
性别
All
接受健康志愿者
否

入选标准

  • 未提供

排除标准

  • 未提供

研究组 & 干预措施

Eculizumab

Experimental

干预措施: Eculizumab (Drug)

结局指标

主要结局

Proportion of Patients With Complete TMA Response

时间窗: Through 26 weeks

Proportion of Patients with Complete TMA response was determined and defined by normalization of hematological parameters (platelet count and LDH) and ≥ 25% improvement in serum creatinine from baseline which was sustained for at least two consecutive measurements obtained at least four weeks apart).

次要结局

  • Proportion of Patients With Complete Hematologic Response(Through End of Study, Median Exposure 55 Weeks)
  • Proportion of Patients With Platelet Count Normalization(Through End of Study, Median Exposure 55 Weeks)
  • Proportion of Patients With Estimated Glomerular Filtration Rate (eGFR) Improvement(Through End of Study, Median Exposure 55 Weeks)
  • Platelet Count Change From Baseline to 26 Weeks(Through 26 weeks)
  • Proportion of Patients With Complete TMA Response(Through End of Study, Median Exposure 55 Weeks)
  • Platelet Count Change From Baseline to 52 Weeks(Through 52 Weeks)
  • Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 5 - <10kg) N=3(Induction Phase was between 1 and 4 weeks in length depending on patient weight cohort.Maintenance Phase was started 1 week after induction phase and dosing of eculizumab administration was every 2 weeks or every 3 weeks depending on patient weight cohort)
  • Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 10 - <20kg) N=7(Induction Phase was between 1 and 4 weeks in length depending on patient weight cohort.Maintenance Phase was started 1 week after induction phase and dosing of eculizumab administration was every 2 weeks or every 3 weeks depending on patient weight cohort)
  • Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 20 - <30kg)(Induction Phase was between 1 and 4 weeks in length depending on patient weight cohort. Maintenance Phase was started either 2 weeks or 3 weeks after induction phase depending on patient weight cohort)
  • Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort 30 - <40kg) N=1(Induction Phase was between 1 and 4 weeks in length depending on patient weight cohort. Maintenance Phase was started either 2 weeks or 3 weeks after induction phase depending on patient weight cohort)
  • Pharmacokinetics (PK) and Pharmacodynamics (PD); Minimum and Maximum Blood Concentration (Body Weight Cohort ≥40kg) N=5(Induction Phase was between 1 and 4 weeks in length depending on patient weight cohort. Maintenance Phase was started either 2 weeks or 3 weeks after induction phase depending on patient weight cohort)

研究者

申办方类型
Industry
责任方
Sponsor

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