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The PUMA Trial is a Trial of a Single ProHema Modulated-CB Unit as Part of a Double CB Transplant in Patients With Hematologic Malignancies.

NCT01627314

Description:

This study is an open-label randomized, prospectively and historically controlled trial of the safety and efficacy of a single ProHema-CB unit used as part of a double CB transplant following myeloablative or reduced intensity conditioning for subjects age 15-65 years with hematologic malignancies. A maximum of 60 eligible subjects will be enrolled and treated in the trial at approximately 10 centers within the U.S.

Related Conditions:
  • Acute Myeloid Leukemia
Recruiting Status:

Terminated

Phase:

Phase 2

Trial Eligibility

Document

Title

  • Brief Title: The PUMA Trial is a Trial of a Single ProHema Modulated-CB Unit as Part of a Double CB Transplant in Patients With Hematologic Malignancies.
  • Official Title: A Phase 2 Controlled Trial of a Single ProHema®-CB Unit (Ex Vivo Modulated Human Cord Blood) As Part of a Double Umbilical Cord Blood Transplant Following Myeloablative or Reduced Intensity Conditioning For Patients Age 15-65 Years With Hematologic Malignancies.

Clinical Trial IDs

  • ORG STUDY ID: FT1050-03
  • NCT ID: NCT01627314

Conditions

  • Hematologic Malignancies

Interventions

DrugSynonymsArms
ProHema-CBProHema-CB
Untreated CBControl Arm

Purpose

This study is an open-label randomized, prospectively and historically controlled trial of the safety and efficacy of a single ProHema-CB unit used as part of a double CB transplant following myeloablative or reduced intensity conditioning for subjects age 15-65 years with hematologic malignancies. A maximum of 60 eligible subjects will be enrolled and treated in the trial at approximately 10 centers within the U.S.

Detailed Description

      All subjects will receive a myeloablative or reduced intensity conditioning regimen, after
      which they will receive 2 HLA-matched UCB units. A total of 40 subjects will receive one
      ProHema-CB as part of a double CB transplant and an additional 20 subjects will be enrolled
      as concurrent controls. The determination of which CB unit will be the ProHema-CB unit will
      be made based primarily upon the degree of HLA match.
    

Trial Arms

NameTypeDescriptionInterventions
ProHema-CBExperimental
  • ProHema-CB
Control ArmActive Comparator
  • Untreated CB

Eligibility Criteria

        Inclusion Criteria:

          1. Subjects with hematologic malignancies for whom allogeneic stem cell transplantation
             is deemed clinically appropriate. Eligible diseases and stages include:

               -  Acute lymphoblastic leukemia (including T lymphoblastic leukemia) in complete
                  remission.

               -  Remission is defined as < 5% blasts with no morphological characteristics of
                  acute leukemia in a bone marrow with > 20% cellularity.

               -  Acute myelogenous leukemia in high risk first CR or second or subsequent CR.

               -  High risk first CR is defined by but is not limited to at least one of the
                  following factors: greater than 1 cycle of induction chemotherapy to achieve CR,
                  prior myelodysplastic syndrome (MDS), presence of Flt3 abnormalities, FAB M6 or
                  M7 subtypes of leukemia, or adverse cytogenetics.

               -  Remission is defined as < 5% blasts with no morphological characteristics of
                  acute leukemia (e.g. Auer Rods) in a bone marrow with > 20% cellularity.

               -  Biphenotypic/Undifferentiated leukemia in first or subsequent CR (same definition
                  of CR as for ALL/AML).

               -  Non-Hodgkin's lymphoma (T-cell, large cell or mantle cell) or Hodgkin's lymphoma
                  in second or subsequent complete remission (CR) or in partial remission (PR) with
                  documented chemosensitivity. In addition, marginal zone lymphoma or follicular
                  lymphoma that has progressed after ≥ 2 therapies (excluding single-agent
                  rituximab). No history of prior myeloablative procedure.

          2. Lack of suitable 5-6/6 HLA-matched related or (if institutional guidelines dictate)
             suitable 8/8 HLA-A, B, C, DRß1 matched unrelated donor; or unrelated donor not
             available within appropriate timeframe.

          3. Age 15-65 years.

          4. Eastern Cooperative Oncology Group (ECOG) performance status 0-2.

          5. Signed IRB approved Informed Consent Form (ICF).

        Exclusion Criteria:

          1. History of prior allogeneic transplantation

          2. Cardiac disease: symptomatic congestive heart failure or evidence of left ventricular
             dysfunction (Ejection fraction < 40%) as measured by gated radionuclide ventriculogram
             or echocardiogram; active angina pectoris, or uncontrolled hypertension; history of
             myocardial infarction with depressed ejection fraction.

          3. Pulmonary disease: symptomatic chronic obstructive lung disease, symptomatic
             restrictive lung disease, or corrected DLCO of < 50% of predicted, corrected for
             hemoglobin.

          4. Renal disease: serum creatinine > 2.0 mg/dl and calculated creatinine clearance < 40
             mL/min.

          5. Hepatic disease: serum bilirubin > 2.0 mg/dl (except in the case of Gilbert's syndrome
             or ongoing hemolytic anemia), SGOT or SGPT > 5 x upper limit of normal.

          6. Neurologic disease: symptomatic leukoencephalopathy, active CNS malignancy or other
             neuropsychiatric abnormalities believed to preclude transplantation.

          7. HIV antibody.

          8. Uncontrolled infection.

          9. Pregnancy or breast feeding mother.

         10. Inability to comply with the requirements for care after allogeneic stem cell
             transplantation.
      
Maximum Eligible Age:65 Years
Minimum Eligible Age:15 Years
Eligible Gender:All
Healthy Volunteers:No

Primary Outcome Measures

Measure:Neutrophil engraftment/chimerism
Time Frame:before Day 60
Safety Issue:
Description:To determine the rate of neutrophil engraftment after a single ProHema-CB unit is used as part of a double CB transplant.

Secondary Outcome Measures

Measure:Neutrophil engraftment
Time Frame:before Day 180
Safety Issue:
Description:To define measures of engraftment, including time to neutrophil engraftment, cumulative incidence of neutrophil engraftment by Day 42, time to platelet engraftment (> 20K and > 50K), cumulative incidence of platelet engraftment, and rates of primary and secondary graft failure

Details

Phase:Phase 2
Primary Purpose:Interventional
Overall Status:Terminated
Lead Sponsor:Fate Therapeutics

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