A Phase 1 Trial of a Single ProHema® CB Product for Pediatric Patients With Hematologic Malignancies
Condition: Hematologic Malignancies
MeSH terms: Neoplasms; Hematologic Neoplasms
Intervention: Biological: ProHema-CB
Intervention description: Each subject will receive one administration of ProHema-CB unit transplant.
Phase: Phase 1
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 3.0
Sex: All
Minimum age: 1 Year
Maximum age: 18 Years
Healthy volunteers: No

Summary:

      This is an open-label, safety study of a single ProHema-CB product administered following
      myeloablative conditioning regimen in pediatric subjects with hematologic malignancies.
    

Eligibility criteria:

        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).

        Exclusion Criteria:

          1. Female subjects that are pregnant or breastfeeding.

          2. Evidence of HIV infection or HIV positive serology.

          3. Current uncontrolled bacterial, viral or fungal infection.

          4. Prior allogeneic hematopoietic stem cell transplant.

          5. Autologous transplant < 12 months prior to enrollment.

          6. Prior autologous transplant for the disease for which the UCB transplant is being
             performed.

          7. Active malignancy other than the one for which the UCB transplant is being performed
             within 12 months of enrollment.

          8. Inability to receive TBI.

          9. Requirement of supplemental oxygen.

         10. HLA-matched related donor able to donate.

         11. Use of an investigational drug within 30 days prior to screening.

         12. Subject is unlikely to comply with the protocol requirements, instructions and
             study-related restrictions