A Phase II Study of Pevonedistat and Azacitidine in Patients with Myelodysplastic Syndromes (MDS) or MDS/Myeloproliferative Neoplasms that Persist Despite Therapy


Full Title

A Phase II trial of Pevonedistat and Azacitidine in MDS or MDS/MPN Patients Who Fail Primary Therapy with DNA Methyl Transferase Inhibitors


DNA methyltransferase inhibitors, such as azacitidine, are drugs used to treat myelodysplastic syndromes (MDS). However, in some patients the MDS continues to grow. In this study, researchers are evaluating the safety and effectiveness of combination treatment with azacitidine plus the investigational drug pevonedistat (also known as MLN4924) in patients with MDS or MDS/myeloproliferative neoplasms (MPN) that persist despite DNA methyltransferase inhibitor treatment.

Pevonedistat works against cancer by inhibiting an enzyme that cancer cells use to grow. It may work better in combination with azacitidine. It is given intravenously (by vein). Azacitidine is given via subcutaneous (under the skin) injection.


To be eligible for this study, patients must meet several criteria, including but not limited to the following:

  • Patients must have MDS or MDS/MPN that continues to grow despite DNA methyltransferase inhibitor treatment (such as azacitidine, decitabine, or guadecitabine).
  • Patients must be able to walk and do routine activities for more than half of their normal waking hours.
  • This study is for patients age 18 and older.

For more information about this study and to inquire about eligibility, please contact Dr. Raajit Rampal at 212-639-2194.