Multi-center, Open-label Randomized Study of Single or Double Myeloablative Cord Blood Transplantation With or Without Infusion of Off-the-shelf ex Vivo Expanded Cryopreserved Cord Blood Progenitor Cells in Patients With Hematologic Malignancies
I. Compare the time to neutrophil engraftment (absolute neutrophil count [ANC] >= 500) in
patients receiving a standard of care myeloablative cord blood transplant (CBT) augmented
with an off-the-shelf pre-expanded and cryopreserved cord blood product to those who do not
receive the product.
I. Provide initial data on clinical and economic benefit, such as time to platelet
engraftment, duration of initial hospitalization, day 200 transplant related mortality (TRM)
and incidence of severe infections in the first 100 days post transplant.
II. The kinetics of immune system recovery will also be evaluated in both arms.
OUTLINE: Patients are randomized to 1 of 2 treatment arms.
Standard of Care Arm:
CONDITIONING REGIMEN: Patients receive fludarabine phosphate intravenously (IV) over 30
minutes on days -8 to -6 and cyclophosphamide IV on days -7 to -6. Patients also undergo
total-body irradiation (TBI) twice daily (BID) on days -4 to -1.
TRANSPLANT: Patients undergo single-unit or double-unit unmanipulated umbilical cord blood
(UCB) transplant on day 0.
GRAFT-VERSUS-HOST DISEASE (GVHD) PROPHYLAXIS: Patients receive cyclosporine IV over 1 hour
twice daily (adults) or three times a day (children) on days -3 to 100 with taper beginning
on day 101. Patients also receive mycophenolate mofetil (MMF) IV three times a day on days
0-7 then may receive MMF orally (PO) three times a day. Patients remain on MMF three times a
day for a minimum of 30 days, and then may begin taper if there is no evidence of
graft-versus-host disease (GVHD) and are well-engrafted from one donor unit.
CONDITIONING REGIMEN: Patients receive fludarabine phosphate IV and cyclophosphamide IV, and
undergo TBI as in Standard of Care Arm.
TRANSPLANT: Patients undergo single-unit or double-unit unmanipulated UCB transplant on day
0. Patients also receive an infusion of ex vivo-expanded cord blood progenitors at least 4
hours after completion of UCB transplant.
GVHD PROPHYLAXIS: Patients receive cyclosporine IV and mycophenolate mofetil IV or PO as in
Standard of Care Arm.
After completion of study treatment, patients are followed up periodically for 2 years.
Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Time to engraftment (ANC greater than or equal to 500) in both arms (standard myeloablative CBT with and without off-the-shelf expanded cord blood progenitors)
The log-rank test will be used.
Up to 2 years
Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium
United States: Food and Drug Administration
|Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium||Seattle, Washington 98109|
|University of Colorado||Denver, Colorado 80217|
|City of Hope Medical Center||Duarte, California 91010|
|Duke University Medical Center||Durham, North Carolina 27710|
|Dana-Farber Cancer Institute/Boston Children's Hospital||Boston, Massachusetts 02115|