Kinetics of Pathogen Reduced Red Blood Cell Clearance in Subjects Undergoing Red Cell Exchange for Sickle Cell Disease
试验速览
- 阶段
- 2 期
- 状态
- 撤回
- 入组人数
- 5
- 主要终点
- Recovery
研究概览
简要总结
The primary objective of the study is to describe the recovery and survival of pathogen-reduced (PR) red blood cells (RBCs) prepared and stored with the INTERCEPT Blood System for RBCs® (hereafter referred to as INTERCEPT RBCs) in patients with sickle cell disease (SCD) undergoing red cell exchange (RCE) therapy, utilizing flow cytometry for biotin and acridine RBC surface markers.
详细描述
This trial is designed as a prospective, Phase 2, non-randomized, open-label, single arm, single center study in at least five (5) patients with sickle cell disease (SCD) undergoing red cell exchange (RCE) therapy.
Subjects receiving routine care for SCD utilizing regular RCE therapy at Grady Memorial Healthcare will be approached by Emory investigators and asked to enroll in the trial. Interested subjects will complete an informed consent form (ICF) and undergo pre-study screening, including a test for the presence of naturally-occurring antibodies with specificity for INTERCEPT RBCs and for antibodies with specificity for biotin. Subjects with a positive screen for INTERCEPT RBC antibodies or for biotin antibodies will be excluded from the study. Investigators will screen the current pool of patients receiving RCE treatment at Grady Memorial Healthcare to identify at least five (5) subjects who meet the study's enrollment criteria.
Enrolled and consented subjects will receive a full unit of INTERCEPT RBCs following each of two sequential RCE episodes (RCE #1 and RCE #2). The INTERCEPT RBCs will be administered by simple transfusion immediately following the end of each RCE. During RCE #1 subjects will also receive two biotinylated aliquots (~7 ml each) of RBCs drawn from the full INTERCEPT RBC unit before and after the PR process. The volume of each transfused aliquot will be assessed by weighing the syringe before and after transfusion. Each aliquot will be labeled at different biotin concentrations: (6 or 18 μg) to allow differentiation by flow cytometry. Subjects will be followed through a total of 4 sequential RCE episodes. Subjects will receive a full unit of INTERCEPT RBCs following RCE #2, with no biotinylated aliquots. No additional INTERCEPT RBCs will be transfused during RCE #3 or RCE #4.
To measure RBC recovery and blood volume, subject blood samples will be collected at the following time points:
- RCEs #1 and #2
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Supportive Care
- 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
- •Age ≥18 years
- •homozygous sickle cell disease (HbSS) or Hemoglobin S-β-thalassemia (HbSβ0) SCD
- •Receiving RCE for ≥3 months prior to enrollment and scheduled to undergo at least 4 sequential RCE episodes during the period of enrollment in the study
排除标准
- •Anticipated cessation of RBC transfusion therapy in the next ≤2 months
- •Planned stem cell transplant or gene therapy in the next 6 months
- •Delayed hemolytic transfusion reaction in the past 3 months
- •History of hyperhemolysis syndrome at any time
- •Consuming high-dose biotin or raw egg supplements
- •Current pregnancy
- •Antibody specific to INTERCEPT RBCs or biotinylated red blood cells (BioRBCs) at baseline
- •Patients with RBC alloantibodies that make it difficult to provide antigen matched blood.
研究组 & 干预措施
Intervention Arm
All subjects enrolled in CLI00185 will participate in the intervention arm which includes transfusion of a single pathogen reduced red blood cell unit in conjunction with two sequential red cell exchange episodes (as prescribed by subjects attending physician). Subjects will also receive aliquots of biotin labeled red blood cells during the first of the two red cell exchange episodes.
干预措施: Pathogen reduced red blood cells (Device)
结局指标
主要结局
Recovery
时间窗: 24 hour
The volume of distribution and 24-hour post transfusion recovery (PTR24) of the INTERCEPT RBC units
Correlation between the biotin and acridine markers
时间窗: 6 months
Correlation between the biotin and acridine markers for determining the number of circulating INTERCEPT RBCs
Survival
时间窗: 6 months
Comparison of Pre-PR and INTERCEPT RBC PTR24, and survival over the duration of the study with intervening RCE procedures
Acridine loss
时间窗: 6 months
Kinetics of acridine loss from the INTERCEPT RBC surface
次要结局
- Adverse Events(6 months)
- Treatment emergent antibodies(6 months)