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Our mission is to provide healthcare professionals with unbiased clinical research information, easily.

Currently, you can access the following clinical trials being conducted worldwide:

359,057 studies
219 countries
Clinical trial information and results are updated daily from ClinicalTrials.gov. The latest data update was conducted on 12/04/2020.
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Clinical trial information and results are updated daily from ClinicalTrials.gov. The latest data update was conducted on 12/04/2020.
Our Science
  • azacitidine (DNA Methyltransferase Inhibitor)

    The safety and efficacy of the agents and/or uses under investigation have not been established. There is no guarantee that the agents will receive health authority approval or become commercially available in any country for the uses being investigated.


    Proposed Mechanism of Action

    Azacitidine is a cytidine nucleoside analogue1 that is incorporated into newly synthesized DNA as well as RNA.1 Preclinical studies suggest that the antileukemic effects of azacitidine include direct cytotoxicity due to inhibition of protein synthesis and DNA damage and re-expression of aberrantly silenced tumor suppressor genes due to DNA hypomethylation.1-3

    Azacitidine Hypothesized Mechanism of Action

    Azacitidine is an epigenetic modifying therapy hypothesized to re-activate aberrantly silenced tumor suppressor genes and induce malignant cell death in acute myeloid leukemia.

    azacitidine by Disease State

    azacitidine in Acute Myeloid Leukemia

    View Trials Investigating azacitidine in Acute Myeloid Leukemia
    View Rationale for Clinical Development

    Rationale for Clinical Development

    Patients with acute myeloid leukemia (AML) have a poor prognosis, intensified by age-related factors and adverse disease characteristics.4-7 Intensive chemotherapy induction is standard treatment for AML but depends on patient fitness.6-8 The majority of patients who are ineligible or unfit for intensive chemotherapy are elderly,4,6-8 and their prognosis remains dismal.5 There is an unmet need for additional treatment options for patients with AML who are ineligible or unwilling to receive intensive chemotherapy.5 Preclinical studies in leukemia cell lines have demonstrated that treatment with azacitidine resulted in DNA demethylation, which supports its clinical investigation in AML.9

    View Related Pathways


    1. Raj K, Mufti GJ. Ther Clin Risk Manag. 2006;2:377-388. PMID: 18360650
    2. Hollenbach PW, et al. PLoS One. 2010;5:e9001. PMID: 20126405
    3. Kaminskas E, et al. Oncologist. 2005;10:176-182. PMID: 15793220
    4. Pollyea DA, et al. Br J Haematol. 2011;152:524-542. PMID: 21314823
    5. Thein MS, et al. Cancer. 2013;119:2720-2727. PMID: 23633441
    6. Döhner H, et al. Blood. 2010;115:453-474. PMID: 19880497
    7. Milligan DW, et al. Br J Haematol. 2006;135:450-474. PMID: 17054678
    8. NCCN Clinical Practice Guidelines in Oncology. Acute Myeloid Leukemia. V2.2019.
    9. Hagemann S, et al. PLoS ONE. 2011;6:e17388. PMID: 21408221