Oral Arsenic Trioxide for NPM1-mutated AML

A prospective open-label phase 2 study will be designed to assess the efficacy of oral arsenic trioxide plus azacitidine in preventing relapses in patients with NPM1-mutant AML. After screening and eligibility assessment, patients will receive treatment with oral arsenic trioxide plus azacitidine for 12 months. The recruitment period will last for 24 months and it will take approximately 36 months for study completion.

Sex

All

Minimum age

18 Years

Inclusion Criteria:

1. Adults ≥ 18 years
2. Diagnosis of AML with NPM1 mutation
3. Positive MRD for NPM1 mutation after completion of consolidation in transplant-ineligible patients
4. Positive MRD for NPM1 mutation after allogeneic HSCT
5. bilirubin ≤ 1.5 x upper limit normal (ULN); alanine aminotransferase (ALT) ≤ 2 x ULN or aspartate aminotransferase (AST) ≤ 2 x ULN; and prothrombin time versus control \<3 seconds at screening
6. Glomerular filtration rate ≥ 50 mL/min (by MDRD equation or Cockcroft-Gault formula)
7. Corrected QT interval (QTc) (by Framingham formula) \<500ms.
8. Able to give a written informed consent and fully comply to the requirements of the study.

Exclusion Criteria:

1. Patients on other investigational therapies
2. Prior exposure to azacitidine, decitabine or arsenic trioxide
3. Uncontrolled graft-versus-host disease (GVHD)
4. Eastern Cooperative Oncology Group (ECOG) performance status \> 2

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