跳至主要内容
临床试验/NCT02731157
NCT02731157已完成4 期

Rejuvesol® Washed RBC in Sickle Cell Patients Requiring Frequent Transfusions

Duke University2 个研究点 分布在 1 个国家目标入组 4 人开始时间: 2016年12月最近更新:
适应症
干预措施
相关药物

试验速览

阶段
4 期
状态
已完成
入组人数
4
试验地点
2
主要终点
Average Percent Hemoglobin (HbA) Decrement Per Day

研究概览

简要总结

The objective of this proposal is to test the feasibility of red blood cell (RBC) rejuvenation to chronic transfusion in sickle cell disease (SCD) and the potential benefit of RBC rejuvenation in this population to determine if a larger clinical trial powered to definitively characterize the benefits of rejuvenation is warranted.

This is a small pilot study is to see if restoring important energy molecules (ATP and 2,3,DPG) in stored red blood cells before they are transfused, with a rejuvenating solution (Rejuvesol), offers any advantages to individuals over standard blood transfusion. Subjects will receive either rejuvenated (R) or standard (S) RBCs with each transfusion for 6 transfusions (over approximately a 6-month period) in a pre-defined order to maximize detection of any signal.

详细描述

Blood transfusion is part of the standard care for individuals that have sickle cell disease. Often these transfusions become needed quite frequently. The purpose of the red blood cells in the blood is to deliver oxygen to the organs and tissues of the body. People with sickle cell disease have abnormal red blood cells. Stored blood undergoes some changes that may make it less effective in achieving this goal.

The purpose of this study is to see if restoring important energy molecules (ATP and 2,3,DPG) in stored red blood cells before they are transfused, with a rejuvenating solution (Rejuvesol), offers any advantages to individuals over standard blood transfusion. This is a Food and Drug Administration (FDA) approved process that is described by the American Association of Blood Banks for prolonging blood storage but not used for everyday transfusions. The investigators want to use this process to improve blood transfused to individuals who need frequent transfusions. Potential advantages include better delivery of oxygen by the transfused red blood cells and easier release of oxygen to the tissues. In addition the study will assess how using the rejuvenated blood affects the interval between transfusions. This means possibly that transfusions may not be needed as often.

Although Rejuvesol has been previously approved by the FDA, it is not routinely used to prepare standard blood transfusions to individuals who have sickle cell disease. Use of Rejuvesol in this study is considered investigational.

研究设计

研究类型
Interventional
分配方式
Non Randomized
干预模型
Crossover
主要目的
Other
盲法
Single (Outcomes Assessor)

入排标准

年龄范围
18 Years 至 —(Adult, Older Adult)
性别
All
接受健康志愿者

入选标准

  • Stable, compliant, chronically transfused sickle cell disease (SCD) patients
  • Currently maintained crisis-free with repeated RBC therapy for at least 3 consecutive sessions
  • ≥18 years old
  • Have Hb SS disease
  • Have the capacity to give informed consent

排除标准

  • Baseline need for washed RBCs
  • Pre-treatment SaO2 < 92%.

研究组 & 干预措施

Transfusion with rejuvenated red blood cells (RBCs)

Experimental

Subjects with sickle cell disease will receive RBCs treated with Rejuvesol®. Scheduled red cell exchanges performed with the last 4 units of the exchange having been incubated with Rejuvesol® solution. Only transfusions necessary for the treatment of the sickle cell disease will be given for the purpose of the study.

干预措施: Rejuvesol (Drug)

Transfusion with rejuvenated red blood cells (RBCs)

Experimental

Subjects with sickle cell disease will receive RBCs treated with Rejuvesol®. Scheduled red cell exchanges performed with the last 4 units of the exchange having been incubated with Rejuvesol® solution. Only transfusions necessary for the treatment of the sickle cell disease will be given for the purpose of the study.

干预措施: Blood transfusion (Procedure)

Transfusion with standard red blood cells

Active Comparator

Subjects with sickle cell disease will receive standard RBCs. Only transfusions necessary for the treatment of the sickle cell disease will be given for the purpose of the study.

干预措施: Blood transfusion (Procedure)

结局指标

主要结局

Average Percent Hemoglobin (HbA) Decrement Per Day

时间窗: 6 months

The %HbA decrement is the current pre-treatment HbA - previous post treatment HbA in %. The average %HbA decrement per day was calculated using matched pairs.

次要结局

  • Actual HbA Decrement (g/dl) With Indexing to Calculated Circulating Blood Volume(6 months)
  • Actual HbA Decrement (g/dl) Without Indexing to Calculated Circulating Blood Volume(6 months)
  • Change in p50 Pre- and Post-transfusion(6 months)
  • Change in RBC/RBC-MP-mediated Thrombin Generation(6 months)
  • Change in RBC Microparticles (MP) Counts(6 months)

研究者

申办方类型
Other
责任方
Sponsor

研究点 (2)

Loading locations...

相似试验