Phase II Study of Myeloablative Double Unit Umbilical Cord Blood Transplantation in Adult Patients

Study Overview
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Brief Summary
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This study aimed to evaluate the efficacy and safety of myeloablative double unit umbilical cord blood transplantation (dUCBT) in adult patients with hematologic malignancies. Two myeloablative conditioning regimens will be used in this study: (1) total body irradation (TBI), cyclophosphamide, and cytarabine, or (2) thiotepa, busulfan, and fludarabine.

Official Title
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Prospective Multicenter Phase II Study of Myeloablative Double Unit Umbilical Cord Blood Transplantation in Adult Patients With Hematologic Malignancies

Conditions
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Hematologic Malignancy

Intervention / Treatment
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* Procedure: Myeloablative double unit cord blood transplantation


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients with hematologic malignancies requiring hematopoietic stem cell transplantation: (1) high risk acute myeloid leukemia in remission, (2) high risk acute lymphoblastic leukemia in remission, or (3) IPSS intermediate-2, high, or transfusion-dependent myelodysplastic syndrome Patients who have suitable cord blood units for dUCBT Patients with ECOG performance status 0-1 Patients with adequate organ function: lung, kidney, liver, heart, etc. Informed consent Exclusion Criteria: Patients who can receive hematopoietic stem cell transplantation from HLA-matched sibling or unrelated donors Patients who have anti-HLA antibodies Patients with any evidence of central nervous system (CNS) involvement of disease Patients with uncontrolled diabetes Patients with uncontrolled hypertension Patients with any evidence of active infection Positive for human immunodeficiency virus (HIV) Previous history of other malignancy within 5 years (Basal cell carcinoma of skin, cervical carcinoma in situ, and differentiated thyroid carcinoma with curative resection are permitted.) Women who are pregnant or breastfeeding Patients with previous history of allogeneic stem cell transplantation Patients with major psychotic disorder or drug/alcohol abuser Inappropriate patients according to the investigators' opinion

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 65 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 |
| --- | --- |
| Experimental: Myeloablative dUCBT<br>Myeloablative conditioning with (1) TBI, cyclophosphamide, and cytarabine, or (2) thiotepa, busulfan, and fludarabine, followed by double unit umbilical cord blood transplant | Procedure: Myeloablative double unit cord blood transplantation<br>* Two myeloablative conditioning regimens will be used in this study: (1) total body irradation (TBI, 12Gy), followed by cyclophosphamide (60 mg/kg/day on day -3 and -2) and cytarabine (3 g/m2 every 12 hours on day -5 and -4), or (2) thiotepa (5 mg/kg/day on day -7 and -6), busulfan (3.2 mg/kg/day on day -5, -4, and -3), and fludarabine (50 mg/m2/day on day -5, -4, and -3).<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Overall survival |  | 2 years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Progression-free survival |  | 2 years | 
| Time to relapse or progression |  | 2 years | 
| Time to engraftment |  | 2 years | 
| Acute graft-versus-host disease |  | 2 years | 
| Chronic graft-versus-host disease |  | 2 years | 
| Non-relapse mortality |  | 2 years | 
| Other transplant-related serious adverse events |  | 2 years |