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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT03653247 | A Study to Assess the Safety, Tolerability, and Efficacy of BIVV003 for Autologous Hematopoietic Stem Cell Transplantation in Patients With Severe Sickle Cell Disease | PHASE1 | COMPLETED | 7 | — | — | Mar 6, 2019 | Jul 17, 2025 | Sep 12, 2025 | 5 | United States |
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 ...