Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT04426591 | Red Blood Cell Survival in Sickle Cell Disease | PHASE1 | COMPLETED | 22 | — | — | Oct 29, 2021 | Apr 30, 2025 | Jun 8, 2025 | 3 | United States |
Survival of the transfused biotin labeled RBCs will be assessed as the count of biotinylated RBCs per sample.
Survival of transfused biotin labeled RBCs will be assessed as the half-life of biotinylated RBCs.
The long-term lifespan of transfused biotin labeled RBCs is assessed as the linearly extrapolated as mean potential lifespan (MPL) of biotinylated RBCs.
| Arm | Type | Description |
|---|---|---|
| Biotin labeled Red Blood Cells | EXPERIMENTAL | Participants receiving a transfusion with biotin labeled RBCs. Samples will be taken for 12 weeks after the biotinylated transfusion. During this time participants will continue to receive regular monthly transfusions (non-biotinylated) as part of CTT. |
| Name | Type | Description |
|---|---|---|
| Biotin Labeled Red Blood Cells | DRUG | On the day of transfusion, a 20 mL aliquot will be sterilely withdrawn from each RBC unit, washed and labeled with sulfo-NHS-biotin for 30 minutes, washed to stop the labeling reaction, then resuspended in plasma to a hematocrit of \~60%. The biotin-labeled RBC (BioRBC) will be transfused along with the remainder of the RBC unit (unlabeled volume). Standard blood bank and CTT protocols and minor antigen matching for SCD patients will be followed. Exact transfusion volume will be determined based on pre-transfusion hemoglobin (Hb), sickle cell hemoglobin (HbS), and body weight, per clinical protocol. |
| INTERCEPT Blood System | DEVICE | In addition to the blood drawn for the main study, individuals participating in this optional intervention will have additional tubes of peripheral venous blood will be drawn for evaluating treatment-emergent antibodies specific to INTERCEPT RBCs and acridine surface label monitoring. Tests for treatment-emergent antibodies specific to INTERCEPT RBCs will be performed according to procedures developed by Cerus Corporation. |
Inclusion Criteria: * Hemoglobinopathy: * Any sickle cell disease genotype, or * Transfusion-dependent thalassemia (TDT) * Receiving CTT for ≥3 months prior to enrollment. * For participants with past BioRBC transfusion exposure, BioRBC antibody screens must have been conducted through at leas...