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

Prior treatment

• 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

Other illness

• 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

Other

• 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
• Pregnant or lactating female
• Unwilling or unable to comply with the requirements of the study protocol

• 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)