A Randomized, Double-Blind, Phase 3 Study Evaluating the Safety and Efficacy of Venetoclax in Combination With Azacitidine in Patients Newly Diagnosed With Higher-Risk Myelodysplastic Syndrome (Higher-Risk MDS)


The purpose of this study is to see how safe and effective venetoclax and azacitidine (AZA) combination are when compared to AZA and a placebo (contains no medicine), in participants with newly diagnosed higher-risk MDS.

Study Arms

  • Experimental: Arm 1: Venetoclax + Azacitidine (AZA)
  • Active Comparator: Arm 2: Placebo + Azacitidine


  • Participants with a diagnosis of Myelodysplastic Syndrome (MDS) according to the 2016 World Health Organization (WHO) classification with presence of < 20% bone marrow blasts per marrow biopsy/aspirate at screening.
  • Participants must meet the following disease activity criteria:
    • Overall Revised International Prognostic Scoring System (IPSS-R) score > 3 (intermediate, high or very high).
    • Eastern Cooperative Oncology Group (ECOG) performance status of <= 2.
    • Hematopoietic stem cell transplant (HSCT) eligible with no pre-arranged HSCT at the time of Study Day 1, or HSCT ineligible without plan for HSCT at the time of Study Day 1.