Phase 2
N=32
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
Myelodysplastic Syndromes (MDS) · Acute Myelogenous Leukemia (AML)
Bottom Line
View on ClinicalTrials.gov: NCT01995578 ↗Enrolled (actual)
32
Serious AEs
31.3%
Results posted
May 2024
Primary outcome: Primary: Relapse Rate — 20; 2; 10 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- low dose 5'-azacitidine (Drug)
- Age
- Pediatric, Adult, Older Adult · 1+ yrs
- Sex
- All
- Sponsor
- Memorial Sloan Kettering Cancer Center
- Primary completion
- Sep 2023
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Relapse Rate |
20; 2; 10 | — |
| SECONDARY Overall Survival |
20; 12 | — |
| SECONDARY Number of Participants Evaluated for Treatment Safety |
32 | — |
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 ( =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 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.
Data sourced from ClinicalTrials.gov (NCT01995578). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.