The purpose of this study is to determine a safe dose of BPX-501 gene modified T cells infused after a haplo-identical stem cell transplant to facilitate engraftment and the safety of Rimiducid (AP1903) on day 7 to prevent GVHD.
This is a single arm dose finding study evaluating the safety and efficacy of a BPX 501 infusion (T cells genetically modified with the inducible Caspase 9 suicide gene) of 3x10E6 to 1X10E7 cells/kg followed by a Rimiducid infusion on day 7 after a partially mismatched, related, T cell-depleted hematopoietic cell transplantation (HCT) in patients with non-malignant diseases. The purpose of this clinical trial is to determine the dose of BPX 501 T cell infusion with subsequent planned infusion of Rimiducid which can facilitate engraftment and prevent the occurrence of GVHD.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
1
Single administration of BPX-501 T cells post partially-mismatched, related T cell depleted HCT followed by Rimiducid infusion on day 7
Fred Hutchinson Cancer ResearchCenter
Seattle, Washington, United States
Adverse Events
To determine the safety (as defined by non-responsive Grade III-IV GVHD to rimiducid) of HCT with HLA-haploidentical CD34+ selected peripheral blood stem cell (PBSC) grafts and BPX 501 T cells followed by scheduled rimiducid infusion on Day 7. this outcome measure is reported as number of patients who experienced the AE of Grade III-IV GVHD that was not non-responsive to rimiducid (safety switch) administration.
Time frame: 24 months
Engraftment
Determine the engraftment rate (defined as \>50% donor CD3 chimerism) on day 28 after HCT with HLA-haploidentical CD34+ selected PBSC grafts per dose cohort of BPX 501 T cells followed by Rimiducid infusion on Day 7. NOTE: only one patient was enrolled who received the dose of 5x 10\^6cell/kg dose of BPX-501
Time frame: Day 28
GvHD
To determine the incidence and severity of acute and chronic GVHD
Time frame: Month 24
Immune Reconstitution
Measure immune reconstitution
Time frame: Month 24
Infection Rates
Determine the risk for severe infections
Time frame: Day 200
Graft Rejection
Incidence of graft rejection
Time frame: Month 24
Rimiducid Activity
Time to resolution of acute and chronic GvHD following administration of Rimiducid
Time frame: Month 24
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High Grade Toxicity
Rate of high grade toxicity
Time frame: Month 24