STAT Inhibitor OPB-111077 and Decitabine in Treating Patients With Acute Myeloid Leukemia That Is Refractory or Newly Diagnosed and Ineligible for Intensive Chemotherapy

PRIMARY OBJECTIVES:

I. To determine the safety and tolerability of STAT inhibitor OPB-111077 (OPB-111077) in
combination with decitabine.

SECONDARY OBJECTIVES:

I. To describe any preliminary efficacy of OPB-111077 in combination with decitabine in
patients with acute myeloid leukemia (AML).

II. To measure adenosine triphosphate (ATP) generation and perform metabolomics in patients
with AML who are receiving OPB-111077 and decitabine.

III. To assess apoptosis and proliferation assays in patients with AML who are receiving
OPB-111077 and decitabine.

Inclusion Criteria:

- Patients must have histologic evidence of high risk acute myeloid leukemia defined as
one of the following:

- Primary refractory non-M3 AML

** Evidence of leukemia after any therapy which, in the opinion of the
investigator, would be appropriate for therapy with OPB-111077 and decitabine

- Newly diagnosed non-M3 AML not eligible for intensive induction chemotherapy

- Subjects must have an Eastern Cooperative Oncology Group (ECOG) performance status of
2 or less

- Subjects must have a life expectancy of at least 4 weeks

- Subjects must be able to consume oral medication

- Subjects must have recovered from the toxic effects of any prior chemotherapy to <
grade 1 (except alopecia)

- Creatinine =< 2.0mg/dL

- Total bilirubin =< 2 x upper limit of normal (ULN)

- Serum glutamate pyruvate transaminase (SGPT) alanine aminotransferase (ALT) =< 2 x ULN

- Negative pregnancy test for women with child-bearing potential

- Patients must be able to sign consent and be willing and able to comply with scheduled
visits, treatment plan and laboratory testing

Exclusion Criteria:

- Subjects with FAB M3 (t(15;17)(q22;q21)[PML-RARalpha]) are not eligible

- Subjects must not be receiving any chemotherapy agents (except hydroxyurea);
intrathecal methotrexate and cytarabine are permissible

- Subjects must not be receiving growth factors, except for erythropoietin

- Subjects with a "currently active" second malignancy, other than non-melanoma skin
cancer, carcinoma in situ of the cervix, resected incidental prostate cancer (staged
pT2 with Gleason score =< 6 and postoperative prostate-specific antigen [PSA] < 0.5
ng/mL), or other adequately treated carcinoma-in-situ are ineligible; patients are not
considered to have a "currently active" malignancy if they have completed therapy and
are free of disease for >= 1 year

- Subjects with uncontrolled high blood pressure, unstable angina, symptomatic
congestive heart failure (New York Heart Association [NYHA] class 3), myocardial
infarction within the past 6 months or serious uncontrolled cardiac arrhythmia are not
eligible

- Subjects with other severe concurrent disease which in the judgment of the
investigator would make the patient inappropriate for entry into this study are
ineligible

- Subjects must not have evidence of active leukemia in the central nervous system (CNS)

- Subjects must not have received any investigational agents within 30 days of study
entry

- Subjects must not be pregnant or breastfeeding; pregnancy tests must be obtained for
all females of child-bearing potential; pregnant or lactating patients are ineligible
for this study; males or women of childbearing potential may not participate unless
they have agreed to use an effective contraceptive method (defined as hormonal
contraceptives, intrauterine devices, surgical contraceptives, or condoms)

- Subjects who have uncontrolled infection are not eligible; patients must have any
active infections under control; fungal disease must be stable for at least 2 weeks
before study entry

- Subjects with bacteremia must have documented negative blood cultures prior to study
entry