This phase I trial studies the side effects and the best dose of plerixafor in increasing cluster of differentiation 34 (CD34) blood forming cell (hematopoietic progenitor cell) count in patients with sickle cell disease. Plerixafor may help move CD34 cells from the bone marrow to the blood. These cells are then collected, changed in the laboratory to make normal oxygen carrying molecules (hemoglobin) in red blood cells and then given back to the patient.
Study sponsor and potential other locations can be found on ClinicalTrials.gov for NCT02193191.
PRIMARY OBJECTIVES:
I. To establish the safety of plerixafor mobilization of hematopoietic progenitor cells (HPC) in patients with sickle cell.
II. To determine the efficacy of plerixafor mobilization of HPC in patients with sickle cell disease.
SECONDARY OBJECTIVES:
I. To determine the ability to transduce plerixafor mobilized CD34+ HPCs with a lentiviral vector encoding the beta (B) - globin gene and possibly expand CD34+HPCs from patients with sickle cell disease in the second phase of the protocol.
II. To perform correlative studies of neutrophil, endothelial cell, and platelet activation with plerixafor administration in the sickle cell host during phase 1 of the protocol.
OUTLINE: This is a dose-escalation study.
Patients receive a single dose of plerixafor subcutaneously (SC).
After completion of study treatment, patients are followed up for 1 week and then at day 30.
Trial PhasePhase I
Trial Typesupportive care
Lead OrganizationMemorial Sloan Kettering Cancer Center
Principal InvestigatorRoni Tamari