Clinical Trials /

Maintenance Low Dose 5'-Azacitidine Post T Cell Depleted Allogeneic Stem Cell Transplantation for Patients With Myelodysplastic Syndrome and Acute Myelogenous Leukemia With High Risk for Post-Transplant Relapse

NCT01995578

Description:

The purpose of this study is to learn if 5'-Azacitidine will help to lower the risk of the disease coming back after a stem cell transplant in patients with MDS and AML. This study will also be looking at the side effects of this medicine. 5'-Azacitidine is an FDA approved drug for treatment of MDS and AML, as well as patients whose disease came back after transplant, where it helped going into remission. It is unclear if 5'-Azacitidine can prevent the disease from coming back after transplant. This study will help show if getting 5'-Azacitidine soon after transplant can lower the risk of your disease coming back.

Related Conditions:
  • Acute Myeloid Leukemia
  • Myelodysplastic Syndromes
Recruiting Status:

Recruiting

Phase:

Phase 2

Trial Eligibility

Document

Title

  • Brief Title: Maintenance Low Dose 5'-Azacitidine Post T Cell Depleted Allogeneic Stem Cell Transplantation for Patients With Myelodysplastic Syndrome and Acute Myelogenous Leukemia With High Risk for Post-Transplant Relapse
  • Official Title: A Single Arm Phase II Trial of Maintenance Low Dose 5'-Azacitidine Post T Cell Depleted Allogeneic Stem Cell Transplantation for Patients With Myelodysplastic Syndrome and Acute Myelogenous Leukemia With High Risk for Post-Transplant Relapse

Clinical Trial IDs

  • ORG STUDY ID: 13-192
  • NCT ID: NCT01995578

Conditions

  • Myelodysplastic Syndromes (MDS)
  • Acute Myelogenous Leukemia (AML)

Interventions

DrugSynonymsArms
low dose 5'-azacitidinelow dose 5'-azacitidine

Purpose

The purpose of this study is to learn if 5'-Azacitidine will help to lower the risk of the disease coming back after a stem cell transplant in patients with MDS and AML. This study will also be looking at the side effects of this medicine. 5'-Azacitidine is an FDA approved drug for treatment of MDS and AML, as well as patients whose disease came back after transplant, where it helped going into remission. It is unclear if 5'-Azacitidine can prevent the disease from coming back after transplant. This study will help show if getting 5'-Azacitidine soon after transplant can lower the risk of your disease coming back.

Trial Arms

NameTypeDescriptionInterventions
low dose 5'-azacitidineExperimentalThis is a single arm phase II trial to assess the efficacy and confirm the safety of maintenance therapy with 5'-azacitadine compared to historical control after TCD allogeneic hematopoietic stem cell transplant for patients with MDS and AML who are at high risk of relapse.
  • low dose 5'-azacitidine

Eligibility Criteria

        Inclusion Criteria:

        Patients who have undergone T cell depleted allogeneic hematopoietic stem cell
        transplantation at MSKCC for:

          -  De novo myelodysplastic syndromes (MDS): IPSS-1 with poor risk cytogenetics or higher
             IPSS.

          -  Acute myelogenous leukemia (AML) in first remission that required more than 1 cycle of
             treatment to achieve remission or with the following cytogenetic abnormalities: FLT3
             mutation, deletion/monosomy of chromosome 5 or 7, MLL gene rearrangement, or more than
             or equal to 3 cytogenetics abnormalities. Also patients in second or greater
             remission.

          -  Patients with Secondary MDS/AML.

          -  Patients will be considered eligible for the study if after transplant they achieved
             hematologic (<5% blasts) and cytogenetic remission.

          -  Patients will be eligible to enter the study between 60-120 days post transplant.

          -  Age: pediatrics and adults patients - 1 year old-75 years old.

          -  Karnofsky performance status >=60% for patients >16yo and Lansky performance status
             >=60% for patients ≤16yo

          -  Stable blood counts (ANC>1000/uL, Hb>8gr/dL, Plt>50,000/ uL) not supported by
             transfusions.

          -  Renal: Serum creatinine <1.5 ULN

          -  Hepatic: <3xULN ALT and <1.5 total serum bilirubin, unless there is congenital benign
             hyperbilirubinemia.

          -  Cardiac: Adequate cardiac function measured by LVEF>50%. If asymptomatic,
             pretransplant echocardiogram is adequate. If symptomatic, echocardiogram needs to be
             repeated.

          -  Each patient must be willing to participate as a research subject and must sign an
             informed consent form.

        Exclusion Criteria:

        Patients will be excluded from the trial if at time of enrollment:

          -  Active uncontrolled bacterial, fungal or viral infection.

          -  Evidence of uncontrolled graft-versus-host disease.

          -  Pulmonary: new onset hypoxia

          -  Known or suspected hypersensitivity to 5'-azacitadine or mannitol.

               -  Evidence of residual disease either by increased blasts count (>5%) or
                  persistence of previous known cytogenetics abnormalities.

          -  Peripheral blood neutrophil chimerism: less than 95% donor.
      
Maximum Eligible Age:75 Years
Minimum Eligible Age:1 Year
Eligible Gender:All
Healthy Volunteers:No

Primary Outcome Measures

Measure:relapse rate
Time Frame:2 years
Safety Issue:
Description:Relapse of MDS or AML will be analyzed as to type and genetic origin of the leukemic cells. These will be defined by morphologic and/or cytogenetic criteria: an increasing number of blasts in the marrow over 5%, by presence of circulating blasts, or by presence of blasts in any extramedullary site as well as presence of previous cytogenetic abnormalities. Other studies assessing for MRD, FACS and FISH assays will be evaluated but would not be considered disease relapse if positive since they are experimental.

Secondary Outcome Measures

Measure:overall survival
Time Frame:2 years
Safety Issue:
Description:Kaplan-Meier methodology will be used to compare overall survival.
Measure:safety
Time Frame:2 years
Safety Issue:
Description:The safety will be described by tabulating the number of transfusions, frequencies of bleeding and serious infections, and the use of G-CSF support.

Details

Phase:Phase 2
Primary Purpose:Interventional
Overall Status:Recruiting
Lead Sponsor:Memorial Sloan Kettering Cancer Center

Trial Keywords

  • 5'-Azacitidine
  • Stem cell transplant
  • 13-192

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