Arsenic Trioxide in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia

PRIMARY OBJECTIVES:

I. Determine the complete remission rate of relapsed and refractory acute myeloid leukemia
(AML) patients with Mutated Nucleophosmin 1 (NPM1) gene.

SECONDARY OBJECTIVES:

I. Determine the duration of remission in these patients. II. Determine the in vivo
biological effect of arsenic trioxide in AML with mutated NPM1.

OUTLINE:

Patients receive arsenic trioxide intravenously (IV) over 1-2 hours daily for up to 45 days.
Patients achieving complete remission, receive arsenic trioxide IV over 1-2 hours daily 5
days a week for 4 weeks. Treatment repeats every 8 weeks for up to 4 courses in the absence
of disease progression or unacceptable toxicity.

Inclusion Criteria:

- AML, any French- American- British (FAB) subtype except M3, with confirmed mutation
in the NPM1 gene

- Relapsed and/or refractory AML from any duration of complete remission (CR); any
number of prior therapies allowed

- Eastern Cooperative Oncology Group (ECOG) performance status 0-2, life expectancy > 3
months

- Serum creatinine =< 2.0 mg/dL

- Bilirubin =< 2.0 mg/dL

- Alanine aminotransferase (ALT)/aspartate aminotransferase (AST) =< 3 x upper limit of
normal (ULN)

- Ability to understand and the willingness to sign a written informed consent document

Exclusion Criteria:

- Pregnant or breast-feeding women will not be entered on this study due to risks of
fetal and teratogenic adverse events as seen in animal/human studies; pregnancy tests
must be obtained in women; sexually active males or females may not participate
unless they have agreed to use an effective contraceptive method

- Patients who are currently receiving another investigational drug

- Patients who are currently receiving other anti-cancer agents

- Uncontrolled systemic fungal, bacterial, viral, or other infection (defined as
exhibiting ongoing signs/symptoms related to the infection and without improvement,
despite appropriate antibiotics or other treatment)

- Known hypersensitivity to arsenic trioxide