Observational, Multicenter Study of Subjects With Pure Red Cell Aplasia Associated With r-HuEPO Treatment
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 52
- 主要终点
- Number of participants with pure red cell aplasia outcome (Extended observation phase)
研究概览
简要总结
The purpose of this study is to investigate the relationship of anti-erythropoietin antibodies to the clinical course and outcome of pure red cell aplasia (PRCA) in participants currently or previously treated with recombinant human erythropoietin.
详细描述
This is a multicenter (study conducted at multiple sites), observational (study in which the investigators/physicians observe the participant's data and measure their outcomes) study. Approximately 150 participants will be enrolled in this study. The study consists of an initial observation phase and extended observation period. An initial observation phase starting at enrollment and ending when 24 months have elapsed since the date of loss of efficacy (LOE), supplemented with retrospective data collection for the period between LOE date and date of enrollment in the study. Participants remaining epoetin alfa (EPO-Ab) positive 24 months after LOE will enter a 2-year extended observation period. Study visits will take place every month during the initial observation phase and data will be collected every 6 months during the extended observation phase. Safety evaluations will include assessment of adverse events, clinical laboratory tests, vital signs, and physical examination which will be monitored throughout the study. The total study duration for each participant will be approximately for 4 years.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Pure red cell aplasia (PRCA) associated with recombinant human erythropoietin (r-HuEPO) treatment
- •Anemia unresponsive to r-HuEPO treatment
- •PRCA associated with erythropoietin treatment followed by a sudden decrease (more than or equal to 2 gram per deciliter within 30 days) in a previously stable hemoglobin level
- •Exclusion criteria:
- •Participants who are not fulfilling the inclusion criteria
排除标准
- 未提供
结局指标
主要结局
Number of participants with pure red cell aplasia outcome (Extended observation phase)
时间窗: Up to 2 years after the enrollment in the extended observation phase
Participants remaining anti-epoetin alfa positive 24 months after loss of efficacy will enter in the extended observation period.
Number of participants with Pure Red Cell Aplasia (PRCA) outcome (Initial observation phase)
时间窗: Up to 24 months after the date of loss of efficacy
The PRCA outcome is measured by anti-epoetin alfa qualitative test. Persistence of PRCA is defined as: 1) absolute reticulocyte count less than 30,000 per cubic millimeter; and/or 2) no reversal of erythroblastopenia on repeated bone marrow testing. Resolution of PRCA is defined as: 1) absolute reticulocyte count greater than or equal to 30,000 per cubic millimeter; and/or 2) reversal of erythroblastopenia on repeated bone marrow testing.
Overall clinical outcome of pure red cell aplasia (Initial observation phase)
时间窗: Up to 24 months after the date of loss of efficacy
The overall clinical outcome is evaluated by anti-epoetin alfa qualitative test. Overall clinical status will be recorded at each visit in the initial and extended observation phases using a categorical scale (improved, same, worsened, death). In case of death, the date and cause of death along with the the date and cause of death will be recorded.
Overall clinical outcome of pure red cell aplasia (Extended observation phase)
时间窗: Up to 2 years after the enrollment in the extended observation phase
次要结局
- Different treatment modalities with pure red cell aplasia outcome (Initial observation phase)(Up to 24 months after the date of loss of efficacy)
- Different treatment modalities with pure red cell aplasia outcome (Extended observation phase)(Up to 2 years after the enrollment in the extended observation phase)
- Risk factors for Loss of Efficacy (LOE) and pure red cell aplasia (PRCA) outcome(Period between LOE date and date of enrollment in the study)