A Phase 3, Single-arm, Open-label, Multicenter Study to Assess the Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Ravulizumab 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
- 18
- 试验地点
- 8
- 主要终点
- Percentage Change in Lactate Dehydrogenase (LDH) From Baseline to Day 183 (Week 26)
研究概览
简要总结
The primary objective of this study is to evaluate the efficacy of ravulizumab in adult participants with 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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Adult C5 inhibitor naive PNH patients (age>=18), which is confirmed by flow cytometry evaluation.
- •Must be vaccinated againast N meningitidis.
排除标准
- •Meningitidis infection or unresolved meningococcal disease
- •History of bone marrow transplantation
- •Other significant systemic diseases that might have impact on efficacy and safety assessment
研究组 & 干预措施
Ravulizumab
During the Primary Treatment Period, participants will receive a weight-based loading dose of ravulizumab on Day 1 followed by weight-based maintenance dose of ravulizumab on Day 15 and once every 8 weeks (q8w) thereafter for a total of 26 weeks. On Day 183, all participants will enter a 32-week Extension Treatment Period and receive ravulizumab. Beginning on Day 183, participants will receive a maintenance dose of ravulizumab q8w for an additional 32 weeks.
干预措施: Ravulizumab (Drug)
结局指标
主要结局
Percentage Change in Lactate Dehydrogenase (LDH) From Baseline to Day 183 (Week 26)
时间窗: Baseline, Day 183 (Week 26)
LDH is an indicator of intravascular hemolysis that occurs in participants with paroxysmal nocturnal hemoglobinuria. A decrease in LDH indicated reduction (improvement) in hemolysis. Baseline was defined as the average of all available on-study assessments prior to the first dose of study drug. The percent change in LDH was analyzed using a mixed-effect model for repeated measures (MMRM) with the fixed categorical effect of visit, fixed continuous effect of the LDH baseline value as covariates, and participant as random effect.
次要结局
- Number of Participants Achieving LDH <1.5 × Upper Limit of Normal (ULN) at Day 183 (Week 26)(Day 183 (Week 26))
- Number of Participants Achieving Transfusion Avoidance (TA) Through Day 183 (Week 26)(Day 183 (Week 26))
- Number of Participants Experiencing Breakthrough Hemolysis Through Day 183 (Week 26)(Day 183 (Week 26))
- Change in Functional Assessment of Chronic Illness Therapy-Fatigue Scale (FACIT-Fatigue) Score From Baseline to Day 183 (Week 26)(Day 183 (Week 26))
- Change in Hemoglobin (Hgb) From Baseline to Day 183 (Week 26)(Baseline, Day 183 (Week 26))