NCT04085601已完成3 期
A Phase 3, Randomized, Multicenter, Open-Label, Controlled Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 53
- 试验地点
- 29
- 主要终点
- Change From Baseline in Lactate Dehydrogenase (LDH) Concentration At Week 26
研究概览
简要总结
Evaluation of the Efficacy and Safety of Pegcetacoplan in Patients with Paroxysmal Nocturnal Hemoglobinuria .
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 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
- •Be at least 18 years old (inclusive).
- •Have LDH ≥1.5 x ULN at the screening visit.
- •Have PNH diagnosis, confirmed by high sensitivity flow cytometry (granulocyte or monocyte clone >10%).
- •Have Hb less than the lower limit of normal (LLN) at the screening visit.
- •Have ferritin greater than/equal to the LLN, or total iron binding capacity (TIBC) less than/equal to ULN at the screening visit, based on central laboratory reference ranges. If a subject is receiving iron supplements at screening, the Investigator must ensure that the subject's dose has been stable for 4 weeks prior to screening, and it must be maintained throughout the study. Subjects not receiving iron at screening must not start iron supplementation during the course of the study.
- •Body mass index (BMI) ≤ 35 kg/m2 at the screening visit.
- •Have a platelet count of >50,000/mm3 at the screening visit.
- •Have an absolute neutrophil count >500/mm3 at the screening visit.
排除标准
- •Treatment with any complement inhibitor (eg, eculizumab) within 3 months prior to screening.
- •Hereditary complement deficiency.
- •History of bone marrow transplantation.
- •Concomitant use of any of the following medications is prohibited if not on a stable regimen for the time period indicated below prior to screening:
- •Erythropoietin or immunosuppressants for at least 8 weeks
- •Systemic corticosteroids for at least 4 weeks
- •Vitamin K antagonists (eg, warfarin) with a stable international normalized ratio (INR) for at least 4 weeks
- •Iron supplements, vitamin B12, or folic acid for at least 4 weeks
- •Low-molecular-weight heparin for at least 4 weeks
研究组 & 干预措施
1,080mg APL-2 administered subcutaneously twice weekly
Experimental
干预措施: APL-2 (Drug)
结局指标
主要结局
Change From Baseline in Lactate Dehydrogenase (LDH) Concentration At Week 26
时间窗: Baseline (Day 1) and Week 26
The LDH concentration was analyzed using an analysis of covariance (ANCOVA) model with a last observation carried forward (LOCF) and a baseline observation carried forward (BOCF) approach for handling missing data. Baseline was defined as average of measurements prior to first dose of pegcetacoplan or on or prior to randomization of SoC. Post baseline missing values are imputed using multiple imputation method with Markov Chain Mont Carlo method.
Number of Subjects Who Achieved Hemoglobin (Hb) Stabilization
时间窗: From Baseline (Day 1) up to Week 26
The Hb stabilization was defined as avoidance of a \>1 gram per deciliter (g/dL) decrease in Hb concentration from Baseline in the absence of transfusion through Week 26.
次要结局
- Change From Baseline in Absolute Reticulocyte Count (ARC) at Week 26(Baseline and Week 26)
- Change From Baseline in Functional Assessment of Chronic Illness Therapy- (FACIT-Fatigue) Scale Score at Week 26(Baseline and Week 26)
- Percentage of Subjects With Hb Normalization Levels at Week 26(Baseline and Week 26)
- Change From Baseline in Linear Analog Assessment (LASA) Scales Score at Week 26(Baseline and Week 26)
- Number of Subjects With Failure of Hb Stabilization(Up to Week 26)
- Time to First PRBC Transfusion(Up to Week 26)
- Number of Subjects With an Hb Response in the Absence of Transfusions(Baseline and Week 26)
- Change From Baseline in Hb Concentration at Week 26(Baseline and Week 26)
- Percentage of Subjects Who Received Transfusion or Decrease of Hb >2 g/dL From Baseline(At Week 26)
- Percentage of Subjects With Transfusion Avoidance(At Week 26)
- Number of PRBC Units Transfused From Baseline Through Week 26(Up to Week 26)
- Percentage of Subjects With LDH Normalization at Week 26(At Week 26)
- Change From Baseline in European Organization for Research and Treatment of Cancer (EORTC) 30-item Core Quality of Life Questionnaire (QLQ-C30) Scores at Week 26(Baseline and Week 26)
- Percentage of Subjects With ARC Normalization(At Week 26)
研究者
研究点 (29)
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