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A Phase 1b/2 Study to Evaluate the Safety and Efficacy of APR-246 in Combination with Azacitidine for the Treatment of TP53 Mutant Myeloid Neoplasms

Clinical Trial Details

This is a Phase 1b/2 study for men and women with a myeloid neoplasm: myelodysplastic syndrome (MDS), a myeloproliferative neoplasm (MPN), or chronic myelomonocytic leukemia (CMML), who are positive for a TP53 mutation. 

This research study is being done because there is a great need for more effective treatments for subjects with myeloid neoplasms. In particular, the mutational status of TP53 is thought to be an important prognostic factor in MDS patients. Therefore, there is a great need to develop new therapies for TP53 mutated patients. It is thought that APR-246 treats myeloid neoplasms facilitating cell death in TP53 mutant cancer cells. Azacitidine is a hypomethylating agent that kills abnormal blood cells. 

The study drugs will be administered following the below schedule:

Lead in phase: 

  • Subjects will receive APR-245 intravenously (IV) on Days -14, -13, -12, and -11. 

Treatment Phase (Each Cycle): 

  • Subjects will receive APR-245 IV on the following Days: 1, 2, 3, 4. 
  • Subjects will receive azacitidine either subcutaneously (SC) or IV over 7 days on the following Days: Day 4, 5, 6, 7, 8, 9, 10 (7 consecutive days) OR Days 4-5 and Days 8-12 (2 days + 5 days) 

Subjects will continue on treatment as long as they are responding to therapy and not experiencing unacceptable side effects

Key Eligibility: 
  • Open to men and women age 18 years or older with a diagnosis of myelodysplastic syndrome (MDS), MDS/ myeloproliferative neoplasm (MPN) or chronic myelomonocytic leukemia (CMML) 
  • Documentation of a TP53 gene mutation by next-generation sequencing (NGS) 
  • Detailed eligibility reviewed when you contact the study team. 

Study contact by location

Upper East Side - Manhattan


Tania J. Curcio, NP, FNP-BC
(212) 746-2571

Primary Investigator(s)

Protocol ID(s)

Weill Cornell Medicine IRB #:




Open to Enrollment

Age Group