Clinical trial NCT02731157: Rejuvesol® Washed RBC in Sickle Cell Patients Requiring Frequent Transfusions
Official title: Rejuvesol® Washed RBC in Sickle Cell Patients Requiring Frequent Transfusions
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Sickle Cell Disease
Lead sponsor: Duke University
Sponsor class: OTHER
First posted: 2016-04-07
Enrollment: 4
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: OTHER

Brief summary: 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.

Detailed description: 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.

Eligibility criteria: Inclusion Criteria:

* 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

Exclusion Criteria:

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

Primary outcomes:
measure: Average Percent Hemoglobin (HbA) Decrement Per Day
timeFrame: 6 months
Secondary outcomes:
measure: Actual HbA Decrement (g/dl) With Indexing to Calculated Circulating Blood Volume
timeFrame: 6 months
measure: Actual HbA Decrement (g/dl) Without Indexing to Calculated Circulating Blood Volume
timeFrame: 6 months
measure: Change in RBC Microparticles (MP) Counts
timeFrame: 6 months
measure: Change in RBC/RBC-MP-mediated Thrombin Generation
timeFrame: 6 months
measure: Change in p50 Pre- and Post-transfusion
timeFrame: 6 months
Other outcomes:
Locations:
city: Chapel Hill
country: United States
facility: University of North Carolina at Chapel Hill
state: North Carolina
city: Durham
country: United States
facility: Duke Univeristy Medical Center
state: North Carolina
Study officials:
affiliation: Duke University
name: Ian J Welsby, BSc MBBS
role: PRINCIPAL_INVESTIGATOR
affiliation: University of North Carolina
name: Jay Raval, MD
role: PRINCIPAL_INVESTIGATOR
References: