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This study is a Phase III, randomized, open-label, multi-center, prospective study of single umbilical cord blood (UCB) transplantation versus double UCB transplantation in pediatric patients with hematologic malignancies.
Full description
BACKGROUND:
In nearly every large single center or registry analysis of outcomes after UCB transplantation, cell dose is identified as an important factor influencing the incidence and rate of hematopoietic recovery, risk of transplant-related mortality, and probability of survival. Pilot data suggest that infusion of two partially human leukocyte antigen (HLA)-matched UCB units, which always augments the graft cell dose, is safe and may improve neutrophil recovery and survival. To determine whether the infusion of two UCB units enhances survival, a multi-center, open-label, randomized trial is proposed. As adequate single UCB units can be identified for more than 80% of pediatric recipients (in contrast to less than 30% for adults), this study will be open only to pediatric patients. The population will be restricted to patients with high-risk hematologic malignancy, the most common indication of UCB transplantation in children.
DESIGN NARRATIVE:
Participants will include patients 1 to 21 years of age with a diagnosis of hematological malignancy and with two partially HLA-matched UCB units. Units must be HLA-matched at 3 of 6 HLA-A and B (intermediate resolution molecular typing) and DRB1 (high resolution molecular typing) with each other and 4 of 6 with the recipient. Two appropriately HLA-matched units must be available such that one unit delivers a pre-cryopreserved, nucleated cell dose of at least 2.5 x 10^7 per kilogram and the second unit delivers at least 1.5 x 10^7 per kilogram.
Patients will be randomized no more than 14 days prior to initiation of conditioning. UCB units will be shipped prior to initiation of conditioning.
The preparative regimen will consist of the following:
Patients will be followed for at least 24 months post-transplant.
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Inclusion criteria
Two partially HLA-matched UCB units. Units must be HLA-matched minimally at 4 of 6 HLA-A and B (at intermediate resolution by molecular typing) and DRB1 (at high resolution by molecular typing) loci with the patient, and the units must be HLA-matched at 3 of 6 HLA- A, B, DRB1 loci with each other (using same resolution of molecular typing as indicated above). Two appropriately HLA-matched units must be available such that one unit delivers a pre-cryopreserved nucleated cell dose of at least 2.5 x 10^7 per kilogram and the second unit at least 1.5 x 10^7 per kilogram.
Acute myelogenous leukemia (AML) at the following stages:
High risk first complete remission (CR1), defined as the following:
Second or greater CR
First relapse with less than 25% blasts in bone marrow
Morphologic complete remission with incomplete blood count recovery
Therapy-related AML for which prior malignancy has been in remission for at least 12 months
Acute lymphocytic leukemia (ALL) at the following stages:
High risk first remission, defined as one of the following conditions:
High risk second remission, defined as one of the following conditions:
Any third or subsequent CR
NK cell lymphoblastic leukemia in any CR
Biphenotypic or undifferentiated leukemia in any CR or if in first relapse must have less than 25% blasts in bone marrow (BM)
Myelodysplastic syndrome (MDS) at any stage
Chronic myelogenous leukemia (CML) in chronic or accelerated phase
All patients with evidence of CNS leukemia must be treated and be in CNS CR to be eligible for study.
Patients 16 years old or older must have a Karnofsky score of at least 70% and patients younger than 16 years old must have a Lansky score of at least 70%.
Patients with adequate physical function as measured by:
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224 participants in 2 patient groups
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Data sourced from clinicaltrials.gov
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