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
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|ClinicalTrials.gov Identifier: NCT01995578|
Recruitment Status : Active, not recruiting
First Posted : November 26, 2013
Last Update Posted : November 6, 2020
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|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||32 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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|
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.
- 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.
- overall survival [ Time Frame: 2 years ]Kaplan-Meier methodology will be used to compare overall survival.
- 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.
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 ClinicalTrials.gov identifier (NCT number): NCT01995578
|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|
|Principal Investigator:||Roni Tamari, MD||Memorial Sloan Kettering Cancer Center|