Design of the VIALE-M phase III trial of venetoclax and oral azacitidine maintenance therapy in acute myeloid leukemia

Future Oncol. 2022 Aug;18(26):2879-2889. doi: 10.2217/fon-2022-0450. Epub 2022 Jul 19.


Prevention of relapse is a major therapeutic challenge and an unmet need for patients with acute myeloid leukemia (AML). Venetoclax is a highly selective, potent, oral BCL-2 inhibitor that induces apoptosis in AML cells. When combined with azacitidine, it leads to prolonged overall survival and rapid, durable remissions in treatment-naive AML patients ineligible for intensive chemotherapy. VIALE-M is a randomized, double-blind, two-arm study to evaluate the safety and efficacy of venetoclax in combination with oral azacitidine (CC-486) as maintenance therapy in patients in complete remission with incomplete blood count recovery after intensive induction and consolidation therapies. The primary end point is relapse-free survival. Secondary outcomes include overall survival, minimal residual disease conversion and improvement in quality-of-life. Trial Registration Number: NCT04102020 (

Keywords: BCL-2 inhibitor; CC-486 (oral azacitidine); acute myeloid leukemia; first remission; maintenance therapy; minimal residual disease conversion; phase III; relapse-free survival; venetoclax.

Publication types

  • Review

MeSH terms

  • Antineoplastic Combined Chemotherapy Protocols* / adverse effects
  • Azacitidine / adverse effects
  • Bridged Bicyclo Compounds, Heterocyclic
  • Cell Division
  • Clinical Trials, Phase III as Topic
  • Humans
  • Leukemia, Myeloid, Acute*
  • Randomized Controlled Trials as Topic
  • Sulfonamides


  • Bridged Bicyclo Compounds, Heterocyclic
  • Sulfonamides
  • Azacitidine
  • venetoclax

Associated data


Grant support