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NCT Number: NCT01995578

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 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.

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Key information

Conditions

Myelodysplastic Syndromes (MDS) Acute Myelogenous Leukemia (AML) Bone Marrow Diseases Hematologic Diseases Hemic and Lymphatic Diseases Leukemia Leukemia, Myeloid Leukemia, Myeloid, Acute Myelodysplastic Syndromes Neoplasms Neoplasms by Histologic Type

Age range

1 year–75 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Memorial Sloan Kettering at Basking Ridge (Consent and Follow-up), Basking Ridge, New Jersey, United States

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

low dose 5'-azacitidine

Drug

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 outcomes

  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 outcomes

  1. Overall Survival

    Time frame: 2 years

    Kaplan-Meier methodology will be used to compare overall survival.

  2. Number of Participants Evaluated for Treatment 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.

Sponsors and collaborators

Lead sponsor

Memorial Sloan Kettering Cancer Center

Other

Registry information

Official study 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

Important dates

Study start
2013
Primary completion
2023
Study completion
2023
First posted
Nov 26, 2013
Registry last updated
May 22, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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