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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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01995578
Recruitment Status : Active, not recruiting
First Posted : November 26, 2013
Last Update Posted : November 6, 2020
Information provided by (Responsible Party):
Memorial Sloan Kettering Cancer Center

Brief Summary:

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.

Condition or disease Intervention/treatment Phase
Myelodysplastic Syndromes (MDS) Acute Myelogenous Leukemia (AML) Drug: low dose 5'-azacitidine Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 32 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Study Start Date : December 2013
Estimated Primary Completion Date : November 2021
Estimated Study Completion Date : November 2021

Arm Intervention/treatment
Experimental: low dose 5'-azacitidine
This 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.
Drug: low dose 5'-azacitidine
5'-azacitadine will be given at a low dose of 32mg/m2 S.C for 5 days every 28 days (a cycle). Dose de-escalation will be permitted for hematologic and non- hematologic toxicities. Patients will start taking the study drug between days 60-120 post TCD allogeneic hematopoietic stem cell transplant and up to a year post-transplant or until there is a toxicity that requires cessation of therapy. Therefore patients will get between 8-10 cycles. Patients who come off-study for reasons unrelated to toxicities before completing 4 cycles will be replaced Since most cases of relapse occur early post transplant, in the first year, this is the most appropriate time to intervene. Treatment will start as soon as possible.

Primary Outcome Measures :
  1. relapse rate [ Time Frame: 2 years ]
    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 :
  1. overall survival [ Time Frame: 2 years ]
    Kaplan-Meier methodology will be used to compare overall survival.

  2. safety [ Time Frame: 2 years ]
    The safety will be described by tabulating the number of transfusions, frequencies of bleeding and serious infections, and the use of G-CSF support.

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   1 Year to 75 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01995578

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United States, New Jersey
Memorial Sloan Kettering at Basking Ridge (Consent and Follow-up)
Basking Ridge, New Jersey, United States, 07920
Memorial Sloan Kettering Monmouth (Consent and Follow-up)
Middletown, New Jersey, United States, 07748
Memorial Sloan Kettering Bergen (Consent and Follow-up)
Montvale, New Jersey, United States, 07645
United States, New York
Memorial Sloan Kettering Commack (Consent and Follow-up)
Commack, New York, United States, 11725
Memorial Sloan Kettering Westchester (Consent and Follow-up)
Harrison, New York, United States, 10604
Memorial Sloan Kettering Cancer Center
New York, New York, United States, 10065
Memorial Sloan Kettering Nassau (Consent and Follow-up)
Rockville Centre, New York, United States
Sponsors and Collaborators
Memorial Sloan Kettering Cancer Center
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Principal Investigator: Roni Tamari, MD Memorial Sloan Kettering Cancer Center
Additional Information:
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Responsible Party: Memorial Sloan Kettering Cancer Center Identifier: NCT01995578    
Other Study ID Numbers: 13-192
First Posted: November 26, 2013    Key Record Dates
Last Update Posted: November 6, 2020
Last Verified: November 2020
Keywords provided by Memorial Sloan Kettering Cancer Center:
Stem cell transplant
Additional relevant MeSH terms:
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Leukemia, Myeloid
Leukemia, Myeloid, Acute
Myelodysplastic Syndromes
Pathologic Processes
Neoplasms by Histologic Type
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Antimetabolites, Antineoplastic
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Enzyme Inhibitors