5-Azacitidine and Decitabine Epigenetic Therapy for Myeloid Malignancies

  • End date
    Dec 1, 2023
  • participants needed
  • sponsor
    Benjamin Tomlinson
Updated on 24 March 2021
myeloproliferative disorder
chronic myelomonocytic leukemia
granulocyte colony stimulating factor
direct bilirubin
bone marrow procedure
colony stimulating factor
blood transfusion
conjugated bilirubin
refractory cytopenia with multilineage dysplasia


Another term for myelodysplastic syndrome is bone marrow failure. The bone marrow is where components of blood such as red cells, platelets and white cells are made. In bone marrow failure, the ability for bone marrow to make these cells is decreased. In myelodysplastic syndrome, this decreased bone marrow function is believed to result from abnormalities that prevent the normal maturation process by which bone marrow cells develop into red blood cells, white blood cells and platelets. In myelodysplastic syndrome, these abnormal bone marrow cells occupy space in the bone marrow and prevent the function of remaining normal bone marrow cells.

One approach to treating the abnormal growth of immature cells is to give chemotherapy which damages DNA within these cells and causes their death. Unfortunately, such therapy has side-effects, since even normal cells can be affected by the treatment. Both 5-azacitidine (5AZA) and decitabine (DEC) are FDA-approved to treat MDS. In this study, 5AZA and DEC will be administered using an alternating low doses schedule in an attempt to overcome the known mechanisms of resistance to the administration of 5AZA or DEC as single agents caused by automatic adaptive shifts in DNA metabolism.


This will be a single arm, open label pilot study of 5AZA-alt-DEC. Participants will be treated for a minimum of 24 weeks in the absence of clear evidence of progressive disease. Participants who have any response will be permitted to continue treatment until relapse or progression of disease that is not sensitive to protocol defined dose escalation.

The primary objective of this study is to determine Overall Response Rate (ORR) of 5AZA-alt-DEC. The combined response endpoint will include complete response (CR), partial response (PR), and hematologic improvement (HI), with HI criteria specifically as defined by IWG criteria

The secondary endpoints of this study include:

  • Cumulative incidence of response for both CR and overall response
  • Duration of response (DOR)
  • Safety evaluation by tabulation of adverse events of grade 3 and higher

Correlative endpoints include:

  • Correlation of DNMT1 depletion with clinical response criteria
  • Correlation of clinical response with disease biological phenotype measured by morphology and cytogenetics
  • Exploratory measurements of pyrimidine metabolism pre-treatment and on-therapy

Condition Bone marrow disorder, Preleukemia, MYELODYSPLASTIC SYNDROME, Myelodysplastic Syndromes (MDS), MDS/MPN Crossover Syndromes, MDS/MPN Crossover Syndromes, myelodysplastic syndromes, myelodysplastic syndrome (mds), MDS/MPN Crossover Syndromes
Treatment Decitabine, 5-azacytidine
Clinical Study IdentifierNCT04187703
SponsorBenjamin Tomlinson
Last Modified on24 March 2021

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