Open-Label, Multicenter Study to Assess the Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Eculizumab in Complement Inhibitor Treatment Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 25
- 试验地点
- 7
- 主要终点
- Percentage change from baseline in Lactate Dehydrogenase (LDH) at Week 12
研究概览
简要总结
This is a Phase 3b, single-arm, open-label, multicenter study to evaluate the efficacy, safety, pharmacokinetics (PK), pharmacodynamics (PD), and immunogenicity of eculizumab in adult participants with paraxysmal noturnal hemoglobinuria (PNH) in China.
详细描述
This is a Phase 3b, single-arm, open-label, multicenter study to evaluate the efficacy, safety, pharmacokinetics (PK), pharmacodynamics (PD), and immunogenicity of eculizumab in adult participants with PNH in China who previously have not been treated with complement inhibitors. Approximately 25 eligible participants in China will be enrolled.
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Adult C5 inhibitor naïve PNH patients (age>=18), which is confirmed by flow cytometry evaluation
- •Must be vaccinated against N meningitidis
排除标准
- •Meningitidis infection or unresolved meningococcal disease
- •Significant bone marrow failure
- •Other significant systemic diseases that might have impact on efficacy and safety assessment
研究组 & 干预措施
Eculizumab
Eculizumab will be administered by IV infusion.
干预措施: Eculizumab (Drug)
结局指标
主要结局
Percentage change from baseline in Lactate Dehydrogenase (LDH) at Week 12
时间窗: Baseline, Week 12
To assess efficacy of eculizumab in participants with PNH
次要结局
- Number(%) of participants achieving LDH Normalization(Baseline through Week 64)
- Number(%) of participants with Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)(Baseline through Week 64)
- Number(%) of participants needing Blood Transfusion(Baseline through Week 64)
- Change from baseline in functional assessment of Chronic Illness Therapy (FACIT)-Fatigue Score at Week 64(Baseline through Week 64)
- Pharmacokinetics (PK): Serum Eculizumab Concentration(Baseline through Week 64 (predose and postdose))
- Change from baseline in Serum Free and total Complement 5 (C5) Concentration(Baseline through week 64 (predose and postdose))
- Number(%) of participants with Antidrug Antibodies (ADAs) to Eculizumab(Baseline through Week 64)
- Number(%) of participants with Breakthrough Hemolysis(Baseline through Week 64)
- Number(%) of participants changes from baseline in vital signs and laboratory parameters at all scheduled visits.(Baseline through Week 64)