Status:
COMPLETED
Single vs Double Umbilical Cord Blood Transplants in Children With High Risk Leukemia and Myelodysplasia (BMT CTN 0501)
Lead Sponsor:
Medical College of Wisconsin
Collaborating Sponsors:
National Heart, Lung, and Blood Institute (NHLBI)
Blood and Marrow Transplant Clinical Trials Network
Conditions:
Acute Myelogenous Leukemia
Acute Lymphocytic Leukemia
Eligibility:
All Genders
1-21 years
Phase:
PHASE3
Brief Summary
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 hem...
Detailed 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 hemato...
Eligibility Criteria
Inclusion
- 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:
- Having preceding myelodysplasia (MDS)
- High risk cytogenetics (high risk cytogenetics: del (5q) -5, -7, abn (3q), t (6;9) complex karyotype \[at least 5 abnormalities\],)the presence of a high FLT3 ITD-AR (\> 0.4)
- Requiring more than 1 cycle of chemotherapy to obtain complete remission (CR);
- FAB M6
- 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:
- Philadelphia chromosome-positive adult lymphoblastic leukemia (Ph+ ALL)
- Mixed lineage leukemia (MLL) rearrangement with slow early response (defined as having M2 \[5-25% blasts\] or M3 \[more than 25% blasts on bone marrow examination on Day 14 of induction therapy\])
- Hypodiploidy (less than 44 chromosomes or DNA index less than 0.81)
- End of induction M3 bone marrow
- End of induction M2 with M2-3 at Day 42
- Evidence of minimal residual disease (MRD). If a patient's only high risk criterion is MRD, approval by a protocol chair or protocol officer is required for enrollment. For COG centers, this will only be for MRD greater than 1 percent by flow MRD at the end of extended induction.
- High risk second remission, defined as one of the following conditions:
- Philadelphia chromosome-positive adult lymphoblastic leukemia (Ph+ ALL)
- Bone marrow relapse less than 36 months from induction
- T-lineage relapse at any time
- Very early isolated central nervous system (CNS) relapse (6 months from diagnosis)
- Slow reinduction (M2-3 at Day 28) after relapse at any time
- Evidence of minimal residual disease (MRD). If a patient's only high risk criterion is MRD, approval by a protocol chair or protocol officer is required for enrollment. For COG centers, this will only be for MRD greater than 1 percent by flow MRD at the end of extended induction.
- 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:
- Cardiac: Left ventricular ejection fraction greater than 40% or shortening fraction greater than 26%
- Hepatic: Bilirubin no more than 2.5 mg/dL; alanine aminotransferase (ALT), aspartate aminotransferase (AST), and alkaline phosphatase (ALP) no more than 5 times the upper limit of normal (ULN)
- Renal: Serum creatinine within normal range for age, or if serum creatinine is outside normal range for age, then renal function (creatinine clearance or GFR) greater than 70 mL/min/1.73 m\^2
- Pulmonary: Diffusing capacity of the lung for carbon monoxide (DLCO), forced expiratory volume in one second (FEV1), or forced vital capacity (FVC) greater than 50% of predicted value (corrected for hemoglobin); if unable to perform pulmonary function tests, then O2 saturation greater than 92% of room air
Exclusion
- Pregnant (β-positive human chorionic gonadotropin \[HCG\]) or breastfeeding
- Evidence of HIV infection or HIV positive serology
- Current uncontrolled bacterial, viral, or fungal infection (currently taking medication and progression of clinical symptoms)
- Autologous transplant less than 12 months prior to enrollment
- Prior autologous transplant for the disease for which the UCB transplant will be performed
- Prior allogeneic hematopoietic stem cell transplant
- Active malignancy other than the one for which the UCB transplant is being performed within 12 months of enrollment
- Inability to receive TBI
- Requirement of supplemental oxygen
- HLA-matched related donor able to donate
Key Trial Info
Start Date :
December 1 2006
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
October 1 2014
Estimated Enrollment :
224 Patients enrolled
Trial Details
Trial ID
NCT00412360
Start Date
December 1 2006
End Date
October 1 2014
Last Update
October 28 2021
Active Locations (38)
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1
University of Alabama
Birmingham, Alabama, United States, 35294
2
Phoenix Children's Hospital
Phoenix, Arizona, United States, 85016
3
City of Hope National Medical Center
Duarte, California, United States, 91010
4
Childrens Hospital at Oakland
Oakland, California, United States, 94609