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.
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
Drug | Synonyms | Arms |
---|
low dose 5'-azacitidine | | low 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
Name | Type | Description | Interventions |
---|
low dose 5'-azacitidine | Experimental | 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. | |
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: | Active, not recruiting |
Lead Sponsor: | Memorial Sloan Kettering Cancer Center |
Trial Keywords
- 5'-Azacitidine
- Stem cell transplant
- 13-192
Last Updated
November 6, 2020