Rejuvesol® Washed RBC in Sickle Cell Patients Requiring Frequent Transfusions

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.

Study Overview

• Status: Completed
• Conditions: Sickle Cell Disease
• Intervention / Treatment: Drug: Rejuvesol; Procedure: Blood transfusion

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.

• Study Type: Interventional
• Enrollment (Actual): 4
• Phase: Phase 4

Contacts and Locations

Study Locations

• United States
  • North Carolina
    • Chapel Hill, North Carolina, United States
      • University of North Carolina at Chapel Hill
    • Durham, North Carolina, United States, 27710
      • Duke Univeristy Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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%.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Other
• Allocation: Non-Randomized
• Interventional Model: Crossover Assignment
• Masking: Single

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Transfusion with rejuvenated red blood cells (RBCs); 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.	Drug: Rejuvesol; Procedure: Blood transfusion; Only transfusions necessary for the treatment of the sickle cell disease will be given for the purpose of the study.
Active Comparator: Transfusion with standard red blood cells; 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.	Procedure: Blood transfusion; Only transfusions necessary for the treatment of the sickle cell disease will be given for the purpose of the study.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Average Percent Hemoglobin (HbA) Decrement Per Day	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.	6 months

Secondary Outcome Measures

Outcome Measure	Time Frame
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 RBC Microparticles (MP) Counts	6 months
Change in RBC/RBC-MP-mediated Thrombin Generation	6 months
Change in p50 Pre- and Post-transfusion	6 months

Collaborators and Investigators

• Sponsor: Duke University
• Investigators: Principal Investigator: Jay Raval, MD, University of North Carolina

Study record dates

Study Major Dates

• Study Start (Actual): December 1, 2016
• Primary Completion (Actual): December 31, 2017
• Study Completion (Actual): December 31, 2017

Study Registration Dates

• First Submitted: April 2, 2016
• First Submitted That Met QC Criteria: April 6, 2016
• First Posted (Estimate): April 7, 2016

Study Record Updates

• Last Update Posted (Actual): January 25, 2019
• Last Update Submitted That Met QC Criteria: December 31, 2018
• Last Verified: December 1, 2018

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Genetic Diseases, Inborn; Anemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Anemia, Sickle Cell
• Other Study ID Numbers: Pro00069955