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Biotin Labeled Red Blood Cells · 1 trial · 1 indication
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| Biotin Labeled Red Blood Cells | DRUG | On the day of transfusion, a 20 mL aliquot will be sterilely withdrawn from each RBC unit, washed and labeled with sulfo-NHS-biotin for 30 minutes, washed to stop the labeling reaction, then resuspended in plasma to a hematocrit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...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Novo Nordisk A/S Sponsored ADR Class B | NVO | 5 | PHASE3 | Etavopivat Low dose |
| Novartis AG Sponsored ADR | NVS | 4 | PHASE3 | Crizanlizumab |
| Sanofi SA Sponsored ADR | SNY | 2 | PHASE3 | PCV21, 20vPCV |
| Vertex Pharmaceuticals Incorporated | VRTX | 3 | PHASE3 | CTX001 |
| Agios Pharmaceuticals, Inc. | AGIO | 2 | PHASE2 | Mitapivat |
| Pfizer Inc. | PFE | 1 | PHASE2 | Osivelotor |
| Bristol-Myers Squibb Company | BMY | 1 | PHASE1 | BMS-986470, Famotidine, Pantoprazole |
| Fulcrum Therapeutics, Inc. | FULC | 1 | PHASE2 | Pociredir |
| Beam Therapeutics, Inc. | BEAM | 2 | PHASE1 | BEAM-101 |
| Editas Medicine, Inc. | EDIT | 2 | PHASE1 | EDIT-301 |
| Disc Medicine, Inc. | IRON | 1 | PHASE1 | DISC-3405 |
| Illumina, Inc. | ILMN | 1 | - | Undisclosed |
Biotin Labeled Red Blood Cells is an investigational small molecule being studied for use in Sickle Cell Disease. It is designed to label red blood cells so their survival can be measured. The drug is in Phase 1 clinical development and is not yet approved by the FDA.
Biotin Labeled Red Blood Cells targets red blood cells by attaching biotin to their surface. This labeling allows researchers to track the lifespan of red blood cells in patients with Sickle Cell Disease. The approach is being evaluated in a Phase 1 clinical trial to assess red blood cell survival.
Biotin Labeled Red Blood Cells is being developed by Cerus Corporation, a company traded on the NASDAQ under the ticker symbol CERS. The company is conducting clinical research on this product for Sickle Cell Disease.
Biotin Labeled Red Blood Cells is in Phase 1 clinical development. It is an investigational drug and has not been approved by the FDA. The Phase 1 trial has been completed, and the drug is not currently in active clinical trials.
Biotin Labeled Red Blood Cells was studied in a Phase 1 clinical trial with the identifier NCT04426591, titled 'Red Blood Cell Survival in Sickle Cell Disease.' The trial enrolled 22 participants in the United States and has been completed. The study was uncontrolled and not randomized or double-blinded.