Clinical trial 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
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 type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Myelodysplastic Syndromes (MDS)
Acute Myelogenous Leukemia (AML)
Keywords: 5'-Azacitidine
Stem cell transplant
13-192
Lead sponsor: Memorial Sloan Kettering Cancer Center
Sponsor class: OTHER
First posted: 2013-11-26
Enrollment: 32
Sex: ALL
Minimum age (years): 1
Maximum age (years): 75
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


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.

Primary outcomes:
measure: Relapse Rate
timeFrame: 2 years
Secondary outcomes:
measure: Overall Survival
timeFrame: 2 years
measure: Number of Participants Evaluated for Treatment Safety
timeFrame: 2 years
Other outcomes:
Locations:
city: Basking Ridge
country: United States
facility: Memorial Sloan Kettering at Basking Ridge (Consent and Follow-up)
state: New Jersey
city: Middletown
country: United States
facility: Memorial Sloan Kettering Monmouth (Consent and Follow-up)
state: New Jersey
city: Montvale
country: United States
facility: Memorial Sloan Kettering Bergen (Consent and Follow-up)
state: New Jersey
city: Commack
country: United States
facility: Memorial Sloan Kettering Commack (Consent and Follow-up)
state: New York
city: Harrison
country: United States
facility: Memorial Sloan Kettering Westchester (Consent and Follow-up)
state: New York
city: New York
country: United States
facility: Memorial Sloan Kettering Cancer Center
state: New York
city: Rockville Centre
country: United States
facility: Memorial Sloan Kettering Nassau (Consent and Follow-up)
state: New York
Study officials:
affiliation: Memorial Sloan Kettering Cancer Center
name: Roni Tamari, MD
role: PRINCIPAL_INVESTIGATOR
References: