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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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. |
|
| 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Rejuvesol | Drug |
| ||
| Blood transfusion |
| 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 |
| Measure | Description | 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 | |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Ian J Welsby, BSc MBBS | Duke University | Principal Investigator |
| Jay Raval, MD | University of North Carolina | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of North Carolina at Chapel Hill | Chapel Hill | North Carolina | United States | |||
| Duke Univeristy Medical Center |
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| ID | Title | Description |
|---|---|---|
| FG000 | All Study Participants | Each participant in this 4 subject pilot study acted as their own control. Red cell exchanges (RCEs) occurred approximately every month and, over a 6 month period, the sequence of exchanges for each patient was: SRRRSS, where S=standard and R=rejuvenated RCEs. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| First Standard RCE |
| |||||||||||||
| First Rejuvenated RCE |
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| Second Rejuvenated RCE |
| |||||||||||||
| Third Rejuvenated RCE |
| |||||||||||||
| Second Standard RCE |
| |||||||||||||
| Third Standard RCE |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | All Study Participants | Each participant in this 4 subject pilot study acted as their own control. Red cell exchanges (RCEs) occurred approximately every month and, over a 6 month period, the sequence of exchanges for each patient was: SRRRSS, where S=standard and R=rejuvenated RCEs. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | Each participant acted as their own control with a sequence of rejuvenated and standard exchanges over 6 months. One subject ended further study participation after only 1 rejuvenated RBC exchange; limited data available. | Posted | Mean | Full Range | %HbA per day | 6 months |
|
6 months
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Transfusion With Standard RBCs | Each participant received three standard red cell exchanges. |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Ian Welsby, M.D. | Duke University | 919-681-6752 | ian.welsby@duke.edu |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Feb 26, 2017 | Nov 9, 2018 | Prot_SAP_000.pdf |
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| ID | Term |
|---|---|
| D000755 | Anemia, Sickle Cell |
| ID | Term |
|---|---|
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
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| ID | Term |
|---|---|
| D001803 | Blood Transfusion |
| ID | Term |
|---|---|
| D001691 | Biological Therapy |
| D013812 | Therapeutics |
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| Procedure |
Only transfusions necessary for the treatment of the sickle cell disease will be given for the purpose of the study. |
|
| 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 |
| Durham |
| North Carolina |
| 27710 |
| United States |
|
| Participants |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Count of Participants | Participants |
|
| OG001 | 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. Blood transfusion: Only transfusions necessary for the treatment of the sickle cell disease will be given for the purpose of the study. |
|
|
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| Secondary | Actual HbA Decrement (g/dl) With Indexing to Calculated Circulating Blood Volume | Data not collected. | Posted | 6 months |
|
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| Secondary | Actual HbA Decrement (g/dl) Without Indexing to Calculated Circulating Blood Volume | Data not collected. | Posted | 6 months |
|
|
| Secondary | Change in RBC Microparticles (MP) Counts | Data not collected. | Posted | 6 months |
|
|
| Secondary | Change in RBC/RBC-MP-mediated Thrombin Generation | Data not collected. | Posted | 6 months |
|
|
| Secondary | Change in p50 Pre- and Post-transfusion | Data not collected. | Posted | 6 months |
|
|
| 0 |
| 4 |
| 0 |
| 4 |
| 0 |
| 4 |
| EG001 | Transfusion With Rejuvenated RBCs | Each participant received three rejuvenated red cell exchanges. | 0 | 4 | 0 | 4 | 0 | 4 |
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| D006425 |
| Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |