Umbilical Cord Blood (UCB) Transplantation in Pediatric Patients With High Risk Leukemia and Myelodysplasia

Completed · Not applicable

Conditions studied: Leukemia, Myelodysplasia, Acute Lymphocytic Leukemia, Acute Myelogenous Leukemia

In brief

Unrelated Cord Blood (UCB) transplant in children is a viable stem cell transplant modality for patients with leukemia and myelodysplasia. UCB is now considered "Standard Of Care" in cases where a suitable living bone marrow donor is not available. The survival of UCB is similar to Matched Unrelated Marrow Transplant. This study is considered "Research" since UCB is not a licensed product and requires investigational new drug (IND). THERE ARE NO SPECIFIC RESEARCH QUESTIONS IN THIS PROTOCOL. This protocol merely provides UCB as a stem cell treatment modality to pediatric patients who may require it after a conditioning regimen that excludes Total Body Irradiation.

Key facts

Study ID

NCT02007863

Run by

University of Miami

People needed

2

Starts

2008-08-01

Expected to finish

2014-06-01

Last updated by the study team

2015-12-28

Who can join

Age: any, up to 21. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Patients must be up to 21 years of age
• Patients cannot receive total body irradiation (TBI) because of:
• Young age - < 2 years at diagnosis of leukemia resulting in an age < 4 years at transplantation (due to risk of severe growth retardation and brain damage).
• Inability to tolerate TBI because of prior radiation or organ toxicity.
• Refractory/multiply relapsed leukemia, for which a traditional TBI/cyclophosphamide regimen would unlikely lead to a successful outcome.
• Patients must have a partially human leukocyte antigen (HLA)-matched UCB unit. Unit 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. The unit must deliver a pre-cryopreserved nucleated cell dose of at least 2.5 x 10\^7 per kilogram.
• Acute myelogenous leukemia (AML) at the following stages:
• High risk first complete remission (CR1), defined as:
• Having preceding myelodysplasia (MDS)
• High risk cytogenetics (High-risk cytogenetics: del (5q) -5, -7, abn (3q), t (6;9) complex karyotype (>= 5 abnormalities)
• Requiring > 2 cycles chemotherapy to obtain CR;
• Second or greater CR.
• First relapse with < 25% blasts in bone marrow.
• Patients with therapy-related AML whose 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:
• Ph+ ALL; or,
• Myeloid/lymphoid leukemia (MLL) rearrangement with slow early response [defined as having M2 (5-25% blasts) or M3 (>25% blasts on bone marrow examination on Day 14 of induction therapy)]; or,
• Hypodiploidy (< 44 chromosomes or DNA index < 0.81); or,
• End of induction M3 bone marrow; or,
• End of induction M2 with M2-3 at Day 42.
• High risk second remission, defined as:
• Bone marrow relapse < 36 months from induction; or,
• T-lineage relapse at any time; or,
• Very early isolated central nervous system (CNS) relapse (6 months from diagnosis); or,

You may not qualify if…

• Pregnant (B-positive 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 < 6 months prior to enrollment.
• Prior autologous transplant for the disease for which the UCB transplant will be performed.
• Allogeneic hematopoietic stem cell transplant < 6 months prior to enrollment.
• Active malignancy other than the one for which the UCB transplant is being performed within 12 months of enrollment
• Active CNS leukemia.
• Requirement of supplemental oxygen.
• HLA-matched related donor able to donate.

Where it is running

• Jackson Memorial Hospital — Miami, Florida, United States
• University of Miami Sylvester Comprehensive Cancer Center — Miami, Florida, United States

Full record on ClinicalTrials.gov

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