NCT01995578

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

Completed Phase 2 Results posted Last updated 25 April 2024

What this trial tests

Phase 2 trial testing low dose 5'-azacitidine in Myelodysplastic Syndromes (MDS) in 32 participants. Completed in 27 September 2023.

Timeline

1 December 2013

Primary endpoint
27 September 2023

27 September 2023

Quick facts

Lead sponsor	Memorial Sloan Kettering Cancer Center
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	32
Start date	1 December 2013
Primary completion	27 September 2023
Estimated completion	27 September 2023
Sites	7 locations across United States

Drugs / interventions tested

• low dose 5'-azacitidine — full drug profile →

Conditions studied

• Myelodysplastic Syndromes (MDS) — all drugs for Myelodysplastic Syndromes (MDS) →
• Acute Myelogenous Leukemia (AML) — all drugs for Acute Myelogenous Leukemia (AML) →

Sponsor

Memorial Sloan Kettering Cancer Center — full company profile →

Who can join

Adults 1 to 75, any sex, with Myelodysplastic Syndromes (MDS) or Acute Myelogenous Leukemia (AML). Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• 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

Sponsor's own 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.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Maintenance therapy in acute myeloid leukemia: an evidence-based review of randomized trials.
   Rashidi A, Walter RB, Tallman MS, Appelbaum FR, et al · · 2016 · cited 38× · PMID 27354720 · DOI 10.1182/blood-2016-03-674127
2. Clinical Results of Hypomethylating Agents in AML Treatment.
   Cruijsen M, Lübbert M, Wijermans P, Huls G. · · 2014 · cited 35× · PMID 26237015 · DOI 10.3390/jcm4010001
3. Epimutational profile of hematologic malignancies as attractive target for new epigenetic therapies.
   Fratta E, Montico B, Rizzo A, Colizzi F, et al · · 2016 · cited 17× · PMID 27329599 · DOI 10.18632/oncotarget.10033
4. Novel agents targeting leukemia cells and immune microenvironment for prevention and treatment of relapse of acute myeloid leukemia after allogeneic hematopoietic stem cell transplantation.
   Shi W, Jin W, Xia L, Hu Y. · · 2020 · cited 15× · PMID 32837873 · DOI 10.1016/j.apsb.2020.06.012
5. Pharmacologic Strategies for Post-Transplant Maintenance in Acute Myeloid Leukemia: It Is Time to Consider!
   Abou Dalle I, El Cheikh J, Bazarbachi A. · · 2022 · cited 11× · PMID 35326641 · DOI 10.3390/cancers14061490
6. Post-Transplant Maintenance Therapy for Patients with Acute Myeloid Leukemia: Current Approaches and the Need for More Trials.
   Assi R, Masri N, Abou Dalle I, El-Cheikh J, et al · · 2021 · cited 8× · PMID 33531851 · DOI 10.2147/jbm.s270015
7. Following in the footsteps of acute myeloid leukemia: are we witnessing the start of a therapeutic revolution for higher-risk myelodysplastic syndromes?
   Bewersdorf JP, Zeidan AM. · · 2020 · cited 7× · PMID 32421403 · DOI 10.1080/10428194.2020.1761968
8. Advances in <i>Ex Vivo</i> T Cell Depletion - Where Do We Stand?
   Bryant AR, Perales MA. · · 2019 · cited 4× · PMID 31106295 · DOI 10.1002/acg2.29