Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Plerixafor · 1 trial · 1 indication
The percentage of participants who are alive at post-transplantation Day 100 will be calculated using the Kaplan-Meier estimate.
The percentage of participants who are alive at post-transplantation Week 52 will be calculated using the Kaplan-Meier estimate.
The percentage of participants who are alive at post-transplantation Week 104 will be calculated using the Kaplan-Meier estimate.
Successful engraftment is defined by absolute neutrophil count (ANC) greater than or equal to \>=500 cells/microliter (mL) for 3 consecutive days.
An AE is any untoward medical occurrence in a participant administered a pharmaceutical product that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product.
An SAE is any untoward medical occurrence that at any dose: Results in death, in the view of the Investigator, places the participant at immediate risk of death (a life-threatening event); however, this does not include an event that, had it occurred in a more severe form, might have caused death, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, results in a congenital anomaly/birth defect or is a medically important event.
| Arm | Type | Description |
|---|---|---|
| BIVV003 | EXPERIMENTAL | Participants will receive plerixafor as subcutaneous (SQ) administration followed by myeloablative conditioning therapy with intravenous (IV) busulfan. BIVV003 will then be administered as a 1-time IV infusion of autologous Cluster of Differentiation 34 + Hematopoietic Stem/Progenitor Cell (CD34+HSPC) transfected ex vivo with zinc finger nuclease (ZFN) messenger ribonucleic acid (mRNAs) targeting the B-cell lymphoma/leukemia 11A (BCL11A) locus. |
| Name | Type | Description |
|---|---|---|
| Plerixafor | BIOLOGICAL | Plerixafor subcutaneous injection will be administered prior to apheresis. |
| Busulfan | DRUG | Busulfan IV infusion will be administered as myeloablative conditioning therapy. |
| BIVV003 | GENETIC | BIVV003 will be administered as an IV infusion following myeloablative conditioning with busulfan. |
Inclusion Criteria * Ages 18 to 40 * Confirmation of sickle cell disease (SCD) diagnosis (HbSS or HbS\[beta\]0 genotype) * Severe SCD, defined as having 1 or more of the following manifestations: Clinically significant neurologic event (example \[e.g.\], stroke) or any neurological deficit lasting ...
| 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 |
Plerixafor is being studied for use in Sickle Cell Disease as part of an autologous hematopoietic stem cell transplantation procedure. It is intended to mobilize stem cells for collection, which are then used in the transplant. The drug is currently in Phase 1 clinical development for this indication.
Plerixafor targets the CXCR4 receptor, which plays a role in stem cell mobilization. By blocking this receptor, it helps release hematopoietic stem cells from the bone marrow into the bloodstream, where they can be collected for transplantation. This mechanism is being evaluated in the context of Sickle Cell Disease treatment.
Plerixafor is being developed by Sangamo Therapeutics, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol SGMO. The company is conducting clinical research to evaluate the drug's safety and efficacy for use in Sickle Cell Disease.
Plerixafor is in Phase 1 clinical development for Sickle Cell Disease. It is an investigational drug and has not been approved by regulatory authorities for this indication. The Phase 1 trial has been completed, and the drug remains under investigation for this use.
Plerixafor has one completed clinical trial, identified as NCT03653247, titled "A Study to Assess the Safety, Tolerability, and Efficacy of BIVV003 for Autologous Hematopoietic Stem Cell Transplantation in Patients With Severe Sickle Cell Disease." This Phase 1 study enrolled 7 participants in the United States.
Plerixafor is also known as BIVV003. In the clinical trial NCT03653247, the drug is referred to as BIVV003, which is the investigational name used during the study. Both names refer to the same drug being developed for use in Sickle Cell Disease.