NCT03030183已完成2 期
A Phase 2 Multicenter, Open-Label, Uncontrolled Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of RA101495 in Subjects With Paroxysmal Nocturnal Hemoglobinuria Who Have an Inadequate Response to Eculizumab
适应症
干预措施
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 3
- 试验地点
- 1
- 主要终点
- Change-from-baseline in Serum Lactate Dehydrogenase (LDH) Levels.
研究概览
简要总结
The purpose of the study is to evaluate the safety and efficacy of RA101495 in patients with paroxysmal nocturnal hemoglobinuria (PNH) who have an inadequate response to eculizumab. Patients will be treated with RA101495 for 12 weeks.
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
- •Diagnosis of PNH by flow cytometry
- •Inadequate response to eculizumab defined as having received eculizumab for at least 6 months plus a documented LDH level ≥ 1.5 x the upper limit of normal (ULN) and/or the presence of a known C5 mutation conferring resistance to eculizumab
排除标准
- •History of meningococcal disease
- •Current systemic infection or suspicion of active bacterial infection
研究组 & 干预措施
Zilucoplan (RA101495)
Experimental
Subjects will receive RA101495 at the dose of 0.3 mg/kg subcutaneously (SC) at Day 1 (loading dose) followed by a starting maintenance dose of 0.1 mg/kg daily SC
干预措施: Zilucoplan (RA101495) (Drug)
结局指标
主要结局
Change-from-baseline in Serum Lactate Dehydrogenase (LDH) Levels.
时间窗: Through Week 12 of the study
Change-from-baseline through Week 12 in serum lactate dehydrogenase (LDH) levels
次要结局
- Change-from-baseline Bilirubin Values(Through week 12)
- Change-from-baseline Hemoglobinuria Values(Through week 12)
- Change-from-baseline Free Hemoglobin Values(Through week 12)
- Change-from-baseline Haptoglobin Values(Through week 12)
- Change-from-baseline Reticulocyte Values(Through week 12)
- Change-from-baseline Total Hemoglobin Values(Through week 12)
研究者
研究点 (1)
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