Clinical Trials /

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

NCT02354417

Description:

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

Related Conditions:
  • Acute Myeloid Leukemia
Recruiting Status:

Terminated

Phase:

Phase 1

Trial Eligibility

Document

Title

  • Brief Title: A Phase 1 Trial of a Single ProHema® CB Product for Pediatric Patients With Hematologic Malignancies
  • Official Title: A Phase 1 Trial of a Single ProHema® CB Product (Ex Vivo Modulated Human Cord Blood Cells) Following Myeloablative Conditioning for Pediatric Patients With Hematologic Malignancies

Clinical Trial IDs

  • ORG STUDY ID: FT1050-04
  • NCT ID: NCT02354417

Conditions

  • Hematologic Malignancies

Interventions

DrugSynonymsArms
Biological: ProHema-CBProHema-CB

Purpose

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

Detailed Description

      A maximum of 18 eligible male and female subjects (1 to 18 years old, inclusive) will be
      enrolled and treated in the trial at approximately 3 to 5 centers within the U.S. These 18
      subjects will consist of 3 cohorts of 6 subjects each. The cohorts will be defined by age: 1
      to 4 years; > 4 to 12 years; and > 12 to 18 years. These cohorts will be enrolled
      simultaneously.

      All subjects will be admitted to the hospital, per institutional practice, and will receive a
      myeloablative conditioning regimen, after which they will receive an HLA-matched or partially
      matched ProHema-CB unit on study Day 0.
    

Trial Arms

NameTypeDescriptionInterventions
ProHema-CBExperimentalAll subjects will receive treatment with ProHema-CB (ex-vivo modulated human cord blood cells) transplant. ProHema-CB (the prostaglandin derivative, 16,16-dimethyl prostaglandin E2 also referred to as FT1050) will be prepared and administered in one of two formulations, based upon subject weight: For subjects > 35 kg, ProHema-CB will be administered as 150 mL product in a blood bag via gravity infusion. It will be infused at 10 mL to 15 mL per minute, for a total infusion time of 10 to 15 min. For subject's ≤ 35 kg, ProHema-CB will be administered as a 50 mL product in a syringe via syringe pump.o It will be infused at 5 mL/kg per hour for a total infusion time of up to ~1 hour.
  • Biological: ProHema-CB

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
      
Maximum Eligible Age:18 Years
Minimum Eligible Age:1 Year
Eligible Gender:All
Healthy Volunteers:No

Primary Outcome Measures

Measure:Safety Profile, Primarily Assessed by Neutrophil Engraftment
Time Frame:Neutrophil engraftment by Day 42
Safety Issue:
Description:To describe the safety profile of ProHema-CB after myeloablative conditioning in pediatric patients with hematologic malignancies. The safety profile will primarily be assessed by neutrophil engraftment.

Details

Phase:Phase 1
Primary Purpose:Interventional
Overall Status:Terminated
Lead Sponsor:Fate Therapeutics

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