A Phase 1 Trial of a Single ProHema® CB Product for Pediatric Patients With Hematologic Malignancies

Inclusion Criteria

• •1. Male and female subjects aged 1 to 18 years, inclusive.
• •2. Subjects with hematologic malignancies for whom allogeneic stem cell transplantation is deemed clinically appropriate.
• •1. Acute Myelogenous Leukemia (AML) in high risk 1st or subsequent CR
• •2. Acute Lymphoblastic Leukemia (ALL) in CR
• •3. NK cell lymphoblastic leukemia in any CR
• •4. Biphenotypic or undifferentiated leukemia in 1st or subsequent CR
• •5. Myelodysplastic Syndrome (MDS) at any stage.
• •6. Chronic Myelogenous Leukemia (CML) All subjects with evidence of CNS leukemia must be treated and be in CNS CR to be eligible for trial.
• •3. Lack of 5-6/6 HLA matched related or 8/8 HLA A, B, C, DRß1 matched unrelated donor; or unrelated donor not available within appropriate timeframe, as determined by the transplant physician.
• •4. Availability of suitable primary and secondary umbilical cord blood (UCB) units.
• •5. Adequate performance status, defined as:
• •1. Subjects ≥ 16 years: Karnofsky score ≥ 70%.
• •2. Subjects \< 16 years: Lansky score ≥ 70%.
• •6. Cardiac: Left ventricular ejection fraction at rest must be \> 40%, or shortening fraction \> 26%.
• •7. Pulmonary:
• •1. Subjects \> 10 years: DLCO (diffusion capacity) \> 50% of predicted (corrected for hemoglobin)
• •2. FEV1, FVC \> 50% of predicted; Note: If unable to perform pulmonary tests, then O2 saturation \> 92% on room air.
• •8. Renal: Serum creatinine within normal range for age, or if serum creatinine outside normal range for age, then renal function (creatinine clearance or GFR) \> 70mL/min/1.73m2.
• •9. Hepatic: Bilirubin ≤ 2.5 mg/dL (except in the case of Gilbert's syndrome or ongoing hemolytic anemia); and ALT, AST and Alkaline Phosphatase ≤ 5 × ULN.
• •10. Signed IRB approved Informed Consent Form (ICF).