A Phase III Study of Azacitidine with and without APR-246 to Treat TP53-Mutant Myelodysplastic Syndromes


Full Title

A Phase III Multicenter, Randomized, Open Label Study of APR-246 in Combination with Azacitidine Versus Azacitidine Alone for the Treatment of TP53 Mutant Myelodysplastic Syndromes (WIRB)


The purpose of this study is to compare treatment combining azacitidine with the investigational drug APR-246 versus azacitidine alone in patients with myelodysplastic syndromes (MDS) containing TP53 mutations. APR-246 was designed to target a protein present in cells called mutant p53. In many cases where tumors have become resistant to standard chemotherapy, the p53 protein is found to be mutated and deactivated.

Early research showed that APR-246 may be able to reactivate mutant p53 in tumor cells, which could potentially make these cells respond better to therapies such as azacitidine. Patients will be randomly assigned to receive azacitidine plus APR-246 or azacitidine alone. Azacitidine is given intravenously (by vein) and APR-246 is given by 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 TP53-mutant MDS.
  • Patients may not have received intensive (induction) chemotherapy, azacitidine, decitabine, or similar medications for MDS.
  • Any prior MDS treatment must have been completed 2 or more weeks before entering the study.
  • 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. Virginia Klimek at 212-639-6519.





Disease Status

Newly Diagnosed