A Phase I/II Trial of the MUC1 Inhibitor, GO-203-2C in Patients With Relapsed or Refractory Acute Myeloid Leukemia

Inclusion Criteria:

To be considered eligible for enrollment into this study, all of the following inclusion criteria must be met during the screening period:
Documented AML by peripheral blood and bone marrow analyses meeting WHO criteria, excluding patients with acute promyelocytic leukemia (APL)
Patients with AML refractory to primary induction chemotherapy, relapsed disease, or age ≥ 60 and not appropriate for standard cytotoxic therapy due to age, performance status, and/or adverse risk factors according to the treating physician
Age ≥ 18 years
Karnofsky performance status ≥ 50% or ECOG performance status 0-2
Life expectancy ≥ 6 weeks
Able to understand the investigational nature of this study and to provide written consent to participate in it
Signed written IRB-approved Informed Consent document

Adequate hepatic and renal function:

serum bilirubin ≤ 1.5 X institutional ULN OR serum direct bilirubin ≤ 2 X institutional ULN
serum ALT and AST ≤ 2.5 X institutional ULN
serum alkaline phosphatase < 5 X institutional ULN
serum creatinine ≤ 2.0 mg/dL
corrected calcium level ≥ institutional LLN
Negative pregnancy test in women of child-bearing potential
Women and men of child-producing potential must agree to use effective contraceptive methods during the study period (including post-treatment observation period)

Exclusion Criteria:

A patient will be considered not eligible for enrollment into this study if any of the following criteria are met during the screening period:
Evidence of leukemic meningitis or other CNS involvement by leukemia
Uncontrolled or poorly controlled hypertension (systolic BP ≥ 160 mmHg or diastolic BP ≥ 100 mmHg) Note: an isolated reading that is not sustained will be permitted.
Evidence of NYHA Class III or IV cardiac disease, or presence of unstable life-threatening arrhythmia, or history of myocardial infarction during the past 6 months
Active bacterial, fungal, or viral infection requiring systemic treatment
Known infection with HIV
History or major surgery within 4 weeks before the first dose of study treatment, or not recovered from prior surgery
Exposure to any other investigational agent at any time within 4 weeks before the first dose of study treatment
Exposure to any other anti-leukemic therapy (except hydroxyurea, see Section 5.5.1) within 2 weeks before the first dose of study treatment
Pregnant or lactating female
Unwilling or unable to comply with the requirements of the study protocol