Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
BIVV003 · 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 ...
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| Vertex Pharmaceuticals Incorporated | VRTX | 3 | PHASE3 | CTX001 |
| Pfizer Inc. | PFE | 1 | PHASE3 | Osivelotor |
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| Beam Therapeutics, Inc. | BEAM | 2 | PHASE2 | BEAM-101 |
| Editas Medicine, Inc. | EDIT | 2 | PHASE2 | EDIT-301 |
| Disc Medicine, Inc. | IRON | 1 | PHASE1 | DISC-3405 |
BIVV003 is an investigational gene therapy being developed for severe sickle cell disease. It is designed for autologous hematopoietic stem cell transplantation, meaning a patient's own stem cells are used. Sangamo Therapeutics, Inc. is the developer. BIVV003 is in Phase 1/2 clinical development and is not described as approved.
BIVV003 is being studied for use in patients with severe sickle cell disease, in the setting of autologous hematopoietic stem cell transplantation. The single clinical trial enrolled patients 18 years of age and older with severe sickle cell disease. It is an investigational gene therapy, not an established treatment.
BIVV003 is developed by Sangamo Therapeutics, Inc., which trades under the ticker SGMOQ. The company is the sponsor associated with the Phase 1/2 study of BIVV003 in severe sickle cell disease. Sangamo Therapeutics is a gene therapy and genomic medicine company.
BIVV003 is in Phase 1/2 clinical development. Its single study, NCT03653247, is listed as a Phase 1/Phase 2 trial and has been completed. BIVV003 is an investigational gene therapy for severe sickle cell disease and is not described as FDA approved.
BIVV003 has been studied in one clinical trial, 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. The trial was a completed Phase 1/Phase 2 study conducted in the United States with 7 enrolled patients.
The BIVV003 trial, NCT03653247, was an uncontrolled, open-label Phase 1/Phase 2 study without randomization or double blinding. It enrolled 7 patients aged 18 years and older with severe sickle cell disease at sites in the United States and did not include healthy volunteers. The study has been completed.