US Study of ECT-001-CB in Pediatric and Young Adult Patients With High-Risk Myeloid Malignancies

Inclusion Criteria

• •1. Acute Myeloid Leukemia
• •1. Chemo-refractory relapse (MRD+)
• •2. Primary induction failure (no CR or CRi after \>= 2 courses of intensive induction therapy): \< 30% blasts in evaluable marrow.
• •3. Relapse after previous allogeneic (or autologous) transplant (\>4 months)
• •4. Secondary or therapy-related MDS/AML
• •5. Poor response to induction (5-30% blasts) or MDR+ after induction
• •2. Myelodysplastic syndrome (MDS)
• •1. Relapse after allogeneic or autologous transplant (\>4 months)
• •2. ≥10 % blasts within 30 days of start of conditioning regimen
• •3. Poor and very poor cytogenetics abnormalities
• •3. Chronic myelogenous leukemia: Patients who progressed to blast crisis
• •4. Mixed Phenotype Acute Leukemia: MRD+ or relapse after previous transplant (\>4 months).
• •5. JMML (Juvenile Myelo-Monocytic Leukemia)
• •6. Availability of 2 ≥ 4/8 HLA matched CBU (allele level: A, B, C and DRB1)
• •1. Cord to be expanded: CD34+ cell count ≥ 0.5 x 10\^5/kg and TNC ≥ 1.5 x 10\^7/kg (pre-cryo)
• •2. Back up cord: Pre-freeze TNC ≥ 2 x 10\^7/kg with CD34+ cells ≥ 1.5 x 10\^5/kg. If a single cord does not meet this criterion 2 back up cords will be an acceptable alternative with a minimum for each of 1.5 x 10\^7 TNC/kg with 1.0 x 10\^5 CD34+/kg. Another acceptable HSC back up source could be a haploidentical with medical clearance prior to starting conditioning regimen.
• •7. Lansky / Karnofsky \>60%
• •8. Bilirubin \< 2 x upper limit of normal (ULN) unless felt to be related to Gilbert's disease or hemolysis; AST and ALT \< 3 x ULN; alkaline phosphatase \< 5 x ULN
• •9. Estimated or measured creatinine clearance ≥ 50ml/min/1.73m2
• •10. Left ventricular ejection fraction of ≥ 40%
• •11. FVC, FEV1 and DLCO ≥ 50% of predicted
• •12. Signed written informed consent
• •13. Female patients of childbearing potential must have a negative serum pregnancy test within 7 days of enrolment and mush be willing to use an effective contraceptive method while enrolled in the study.