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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT02193191 | Escalation of Plerixafor for Mobilization of CD34+ Hematopoietic Progenitor Cells and Evaluation of Globin Gene Transfer in Patients With Sickle Cell Disease | PHASE1 | COMPLETED | 25 | — | — | Sep 17, 2014 | Mar 27, 2025 | Mar 30, 2025 | 2 | United States |
Safety is assessed using a dose limiting toxicity (DLT) endpoint. Definition of a DLT is the occurrence of any of the below events that meets the following criteria: The occurrence of a vasoocclusive crisis requiring hospitalization, acute chest syndrome, CNS acute event, or any other disease related ischemic-based adverse event (AE) should be considered as a DLT, if occurring in the 48 hours DLT observation period.
Efficacy is defined as 100% of evaluable patients reaching a PB CD34 concentration ≥ 30/uL.
| Arm | Type | Description |
|---|---|---|
| Plerixafor | EXPERIMENTAL | Patients will receive a single dose of subcutaneous plerixafor with peripheral blood studies at approximately 0-2 hours before, approximately 6-12 hours after, and approximately 20-48 hours after plerixafor administration, with leukapheresis in the last 3 patients on the protocol. Collected HPCs will be transferred to the MSKCC CTCEF to determine if the HPCs are amenable to transduction with a lentiviral vector encoding the normal ß- globin gene. |
| Name | Type | Description |
|---|---|---|
| Plerixafor | DRUG | - |
Inclusion Criteria: * Patients must have confirmed and measurable Sickle Cell Disease, defined by SS or Sβ thalassemia confirmed by hemoglobin fractionation. * ≥ 18 to 65 years of age * Patient must have a ECOG performance status ≤2 or Karnofsky score \> 70% * Patients must have acceptable organ an...