Rejuvesol® Washed RBC in Sickle Cell Patients Requiring Frequent Transfusions

NCT ID: NCT02731157

Last Updated: 2019-01-25

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE4
• Total Enrollment: 4 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2016-12-31
• Study Completion Date: 2017-12-31

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.

Conditions

Sickle Cell Disease

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: CROSSOVER
• Primary Study Purpose: OTHER
• Blinding Strategy: SINGLE

Study Groups

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.

Group Type: EXPERIMENTAL

Rejuvesol

Intervention Type: DRUG

Blood transfusion

Intervention Type: PROCEDURE

Only transfusions necessary for the treatment of the sickle cell disease will be given for the purpose of the study.

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.

Group Type: ACTIVE_COMPARATOR

Blood transfusion

Intervention Type: PROCEDURE

Only transfusions necessary for the treatment of the sickle cell disease will be given for the purpose of the study.

Interventions

Rejuvesol

Intervention Type: DRUG

Blood transfusion

Only transfusions necessary for the treatment of the sickle cell disease will be given for the purpose of the study.

Intervention Type: PROCEDURE

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%.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Duke University

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Ian J Welsby, BSc MBBS

Role: PRINCIPAL_INVESTIGATOR

Duke University

Jay Raval, MD

Role: PRINCIPAL_INVESTIGATOR

University of North Carolina

Locations

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, United States

Duke Univeristy Medical Center

Durham, North Carolina, United States

Countries

United States

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

Pro00069955

Identifier Type: -

Identifier Source: org_study_id