The purpose of this study is to investigate mobilization and collection of HSPCs in patients with bone marrow failure syndromes (BMFS) using granulocyte-colony stimulating factor (otherwise known as Filgrastim) with plerixafor to demonstrate safety and feasibility of collecting HSPCs to advance gene therapy.
Primary objective:
\- To characterize the safety of Filgrastim plus plerixafor in participants with life threatening blood disorders as determined by the incidence of adverse events (AEs).
Secondary Objectives:
* To measure the mobilization effects of Filgrastim plus plerixafor in the peripheral blood in participants as determined by peak peripheral blood CD34+ counts. * To characterize the feasibility of HSPC mobilization using Filgrastim plus plerixafor as determined by peripheral blood CD34+ counts. * To estimate efficacy of Filgrastim plus plerixafor for HSPC mobilization and apheresis collection in participants as determined by the yield of CD34+ cells (CD34+ cells/kg).
Participants with a bone marrow failure syndrome with an identified genetic cause willing to donate autologous HSPCs for advancing gene therapy
Age ≥ 18 years - 25 years
The following hematological parameters need to be met (regardless of transfusion or growth factor support)
Hb > 8 g/dL
Participant with sickle cell disease
Participant who has had a prior autologous or allogeneic HSCT
Active viral, bacterial, fungal, or parasitic infection
Total bilirubin >2.5x ULN or transaminases >5x ULN
Single group
Filgrastim
DrugAdministered twice daily dose starting on day 1 for 5 days.
Plerixafor
DrugAdministered on day 5 via IV.
Leukapheresis
Procedure/SurgeryPeripheral venous access or through a central venous catheter approximately 4-5 hours after the dose of plerixafor is given.
Incidence of treatment-emergent adverse events following filgrastim plus plerixafor administration
Safety will be assessed by the incidence, type, and severity of adverse events occurring after administration of filgrastim plus plerixafor in participants with bone marrow failure syndromes.
Number of participants achieving peripheral blood CD34+ counts ≥5 cells/µL
Feasibility of hematopoietic stem and progenitor cell mobilization will be assessed by peripheral blood CD34+ cell counts measured after plerixafor administration and prior to or during apheresis.
Peripheral blood CD34+ kinetics following filgrastim plus plerixafor administration
Peripheral blood CD34+ cell counts will be measured after plerixafor administration and prior to or during apheresis.
Observed CD34+ cell yield after 1 blood volume apheresis
CD34+ cell yield (CD34+ cells/kg) collected after processing 1 blood volume during apheresis following filgrastim plus plerixafor administration.
Estimated total CD34+ cell yield from projected full-volume apheresis
Estimated total CD34+ cell yield (CD34+ cells/kg) projected from the observed yield after processing 1 blood volume during apheresis.
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