临床试验/NCT01192399

NCT01192399已完成2 期

Phase II Clinical Study of Eculizumab (h5G1.1-mAb) in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients

Alexion Pharmaceuticals, Inc.0 个研究点目标入组 29 人开始时间: 2007年11月最近更新: 2018年3月13日

适应症

干预措施

试验速览

阶段: 2 期
状态: 已完成
发起方: Alexion Pharmaceuticals, Inc.
入组人数: 29
主要终点: Change From Baseline in Lactate Dehydrogenase

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

A study to assess the safety and efficacy of eculizumab in Japanese patients with hemolytic PNH.

详细描述

The objective of this study was to assess the safety and efficacy of eculizumab in Japanese patients with hemolytic PNH. In addition, the pharmacokinetics (PK) and pharmacodynamics (PD) of eculizumab in Japanese patients were assessed and compared to that of non-Japanese patients who had participated earlier studies of eculizumab treatment in hemolytic PNH.

研究设计

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

入排标准

年龄范围: 12 Years 至 —（Child, Adult, Older Adult）
性别: All
接受健康志愿者: 否

入选标准

•Japanese individuals at least 12 years of age
•Diagnosis of PNH > 6 months
•At least one transfusion in the past 2 years for anemia or anemia-related symptoms
•LDH level ≥ 1.5 x upper limit of normal within 12 weeks
•Presence of a glycosylphosphatidylinositol (GPI)-deficient red blood cell (RBC) clone (type III cells) by flow cytometry of ≥10%
•Negative serum pregnancy test for women of child-bearing potential

排除标准

•Platelet count < 30,000/µL
•Absolute neutrophil count ≤ 500/µL
•Known or suspected hereditary complement deficiency
•History of hematopoietic stem cell transplant
•History of meningococcal disease

研究组 & 干预措施

Eculizumab

Experimental

Eculizumab intravenous infusions every week x 4 doses, then 900 mg 1 week later for 1 dose, then 900 mg every 2 weeks for 4 doses

干预措施: Eculizumab (Biological)

结局指标

主要结局

Change From Baseline in Lactate Dehydrogenase

时间窗: Baseline, Week 12

次要结局

Change From Baseline in Plasma Free Hemoglobin(Baseline, Week 12)
Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Total Score(Baseline, Week 12)
Change From Baseline in Paroxysmal Nocturnal Hemoglobinuria (PNH) Red Blood Cell (RBC) Count(Baseline, Week 12)
Number of Units of Packed Red Blood Cells (pRBCs) Transfused(12 weeks pre-treatment, baseline, 12 weeks post-treatment)
Change From Baseline in Lactate Dehydrogenase (LDH) Area Under the Curve (AUC)(Baseline to Week 12)
Change From Baseline in European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire (QLQ)-C30 Score (Global Health Status)(Baseline, Week 12)

研究者

发起方

Alexion Pharmaceuticals, Inc.

申办方类型

Industry

责任方

Sponsor

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