This phase II trial studies how well donor umbilical cord blood transplant with ex-vivo expanded cord blood progenitor cells (dilanubicel) works in treating patients with blood cancer. Before the transplant, patients will receive chemotherapy (fludarabine, cyclophosphamide and in some cases thiotepa) and radiation therapy. Giving chemotherapy and total-body irradiation before a donor umbilical cord blood transplant helps stop the growth of cells in the bone marrow, including normal blood-forming cells (stem cells) and cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. The donated stem cells may also replace the patient's immune cells and help destroy any remaining cancer cells.
OUTLINE: Patients receive either regimen A or regimen B. REGIMEN A: Patients (10 through 45 years old) receive fludarabine intravenously (IV) over 30 minutes on days -8 to -6 and cyclophosphamide IV on days -7 and -6. Patients undergo total body irradiation (TBI) twice daily (BID) on days -4 to -1. Patients receive unmanipulated cord blood unit IV followed by dilanubicel IV within the next 24 hours on day 0. REGIMEN B: Patients (10 through 65 years old) receive fludarabine IV over 30-60 minutes on days -6 to -3 and IV over 30 minutes on day -2, cyclophosphamide IV on day -6, and thiotepa IV over 2-4 hours on days -5 and -4. Patients undergo TBI once daily (QD) on days -2 and -1. Patients receive unmanipulated cord blood unit IV followed by dilanubicel IV within the next 24 hours on day 0. All patients undergo bone marrow aspirate and biopsy as clinically indicated during screening and on study. Patients undergo multigated acquisition scan (MUGA) or echocardiography (ECHO), and computed tomography (CT) during screening. Patients also undergo blood sample collection on study. After completion of study treatment, patients are followed up at 180 days, 1 year, and 2 years.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
31
Given IV
Given IV
Given IV
Given IV
Undergo TBI
Given IV
Correlative studies
Undergo blood sample collection
Undergo bone marrow aspirate and biopsy
Undergo bone marrow aspirate and biopsy
Undergo MUGA
Undergo ECHO
Undergo CT
Fred Hutch/University of Washington Cancer Consortium
Seattle, Washington, United States
Incidence of graft failure
Primary graft failure/rejection as defined by no neutrophil recovery (regardless of donor chimerism) or autologous recovery (neutrophil recovery but \< 10% donor chimerism in blood and bone marrow \[BM\]).
Time frame: Up to day 45 post-transplant
Time to neutrophil engraftment
The day of neutrophil recovery will be the 1st day of 2 consecutive days of absolute neutrophil count at or above 500 after the 1st post-cord blood transplant nadir.
Time frame: Up to day 45 post-transplant
Time to platelet engraftment
Measured by the number of participants with a platelet count \> 20,000/ul without subsequent transfusions for 7 days
Time frame: Up to day 100 post-transplant
Incidence of adverse events
Toxicities will be graded using the National Cancer Institute Common Terminology Criteria for Adverse Events Version 5.0.
Time frame: Up to day 100 post-transplant
Incidence of non-relapse mortality
Time frame: At day 100 post-transplant
Incidence of non-relapse mortality
Time frame: At 1 year post-transplant
Incidence and severity of acute graft versus host disease (GVHD)
Assessed using the Acute GVHD Grading Scale (reference: Przepiorka D, Weisdorf D, Martin P, et al. 1994 Consensus Conference on Acute GVHD Grading. Bone Marrow Transplant 1995; 15: 825-8).
Time frame: At day 100 post-transplant
Incidence and severity of chronic graft versus host disease (GVHD)
Assessed using the Chronic GVHD Grading Scale (reference: Filipovich AH, Weisdorf D, Pavletic S, et al. National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease. I. Diagnosis and Staging Working Group report. Blood Marrow Transplant 2005; 11: 945-56).
Time frame: 1 year post-transplant
Incidence and severity of chronic graft versus host disease (GVHD)
Assessed using the Chronic GVHD Grading Scale (reference: Filipovich AH, Weisdorf D, Pavletic S, et al. National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease. I. Diagnosis and Staging Working Group report. Blood Marrow Transplant 2005; 11: 945-56).
Time frame: Up to approximately 2 years post-transplant
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.