Adult Double Cord Blood Transplant Study

Study Overview
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Brief Summary
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The hypothesis of the study is double unit umbilical cord blood transplantation in adults will be associated with a one year survival rate of at least 40%.

Official Title
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A Phase II Multicenter Trial of Myeloablative Double Unit Umbilical Cord Blood Transplantation (UCBT) in Adults With Hematologic Malignancy

Conditions
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Cord Blood Stem Cell Transplantation, Hematologic Malignancies

Intervention / Treatment
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* Procedure: Cord blood transplantation


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Age 22 - 50 years Patients will have one of the following hematological malignancies: Acute myelogenous leukemia (AML): Complete first remission (CR1) at high risk for relapse or Complete second remission (CR2) Acute lymphoblastic leukemia (ALL): Complete first remission (CR1) at high risk for relapse or Complete second remission (CR2) Acute undifferentiated leukemia (AUL) or biphenotypic leukemia: CR1 or CR2 Myelodysplastic Syndrome (MDS) with one of the following: Low and Intermediate-1 International Prognostic Scoring System (IPSS) score with Life-threatening neutropenia or thrombocytopenia; or Platelet transfusion dependence Intermediate-2 or High IPSS score Therapy-related disease: patient with history of chemotherapy and current evidence of MDS Patients with adequate organ function and performance status criteria Two Suitable Umbilical Cord Blood Units: a cryopreserved dose of at least 1.5 x 107 TNC/kg. If the unit contains red cells at time of cryopreservation, the cryopreserved dose must be at least 2.0 x 107 TNC/kg. Each unit must be at least 4/6 HLA-A and B antigen, and DRB1 allele matched with the recipient. Each unit must be at least 3/6 HLA-A, B DRB1 antigen matched to each other. Exclusion Criteria: Patient with suitable related donor AML, ALL, AUL, biphenotypic leukemia beyond CR2 AML evolved from myelofibrosis Any acute leukemia with: Morphologic relapse or persistent disease in the BM Active extra-medullary leukemia including active CNS leukemia Requiring greater than two cycles of chemotherapy to obtain present remission status Bone marrow aplasia (defined as BM cellularity < 5% at transplant work-up) MDS with 10% or greater bone marrow blasts at pre-transplant workup Prior autologous or allogeneic HSC transplant at any time Prior radiation therapy rendering patient ineligible for TBI Any uncontrolled infection at time of study enrollment Seropositive or NAT positive for HIV or HTLV1 Females who are pregnant or breast feeding Patient unable to give informed consent or unable to comply with the treatment protocol including appropriate supportive care, follow-up, and research tests

Ages Eligible for Study
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Minimum Age: 22 Years
Maximum Age: 50 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Other: Myeloablative double unit UCBT<br>Myeloablative preparative regimen of chemotherapy and radiation followed by double unit umbilical cord blood transplantation | Procedure: Cord blood transplantation<br>* Myeloablative preparative regimen of chemotherapy and radiation followed by double unit umbilical cord blood transplantation<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Measure overall survival of double unit UCBT in adult patients with hematologic malignancies |  | One year | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Measure incidence of donor-derived neutrophil and platelet recovery |  | 100 Days | 
| Measure contribution of each unit to initial and sustained engraftment |  | 2 years | 
| Measure incidence and severity of acute graft-versus-host disease |  | 100 Days | 
| Measure incidence and severity of chronic GVHD |  | 1 year | 
| Measure incidence of transplant-related mortality |  | 6 months | 
| Measure incidence of malignant relapse |  | 2 years | 
| Measure incidence of serious infectious complications |  | 1 year | 
| Measure incidence of immune reconstitution |  | 2 years | 
| Measure probability of overall and disease-free survival |  | 2 years | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Cord Blood Stem Cell Transplantation, Hematologic Malignancies, Leukemia, Myelodysplastic Syndrome