Venetoclax in Combination With Azacitidine


  1. SUB-TYPE OF MDS:  All Intermediate 2 or High Risk patients who have never had treatment for MDS
  2. SEVERITY OF MDS: High risk
  3. NAME OF DRUG: Venetoclax (a drug that encourages the death of cancer cells by blocking a marker on the cells called BCL-2) plus Azacitidine.
  4. Aims and benefits: To determine the safety and effectiveness of Venetoclax in combination with Azacitidine compared with Azacitidine alone. This study is designed for patients who have never had treatment for higher risk MDS.
      Primary outcome measures:

    • Overall response rate
    • Recommended dose and dosing schedule of Venetoclax in combination with Azacitidine
    • Percentage of patients with side effects
      Secondary outcome measures:

    • Complete response rate
    • Improved blood counts
    • Reduced need for blood and platelet transfusions
    • Reduction in bone marrow blasts
    • Time to transformation to acute myeloid leukaemia
  5. Basic inclusion criteria:
    • Diagnosis of de novo MDS (no identifiable cause for the MDS) with IPSS risk Intermediate-2 or high
    • Between 5 and 30% blasts in the bone marrow
    • ECOG performance status of 0, 1 or2
    • Patient has not received previous treatment (excluding supportive care) for MDS
    • Patient is not a candidate to undergo intensive chemotherapy or allogeneic hematopoietic stem cell transplantation (HSCT)
  6. Basic exclusion criteria:
    • MDS related to previous medications
    • MDS evolving from a pre-existing myeloproliferative neoplasm (MPN)
    • Diagnosis of MDS/MPN overlap
    • Patients who have received allogeneic HSCT or solid organ transplantation.
  7. Trial sites/locations and name of physician in charge of trial: 
  8. More information:

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