A Phase I Study of AC220 in Patients With Relapsed/Refractory Acute Myeloid Leukemia Regardless of FLT3 Status

Inclusion Criteria

• •1. Males and females age ≥ 18 years;
• •2. Histopathologically documented primary or secondary AML, as defined by WHO criteria (Jaffe et al, 2001), confirmed by pathology review at treating institution, meeting at least one of the following:
• •1. Refractory to at least 1 cycle of induction chemotherapy, or
• •2. Relapsed after at least 1 cycle of induction chemotherapy, or
• •3. Patient is not, according to the clinical judgment of the Principal Investigator, a candidate for induction chemotherapy due to age, comorbidity, or other factors;
• •3. Patients for whom no standard therapies are anticipated to result in a durable remission, or who have failed potentially curative therapy, or who refuse standard therapy or patients for whom there is no known therapy of documented treatment benefit;
• •4. Eastern Cooperative Oncology Group (ECOG) performance status of 0-3;
• •5. In the absence of rapidly progressing disease, the interval from prior treatment to time of AC220 administration should be at least 2 weeks for cytotoxic agents (other than hydroxyurea, per Section 8.8), or at least 5 half-lives for noncytotoxic agents;
• •6. Persistent chronic clinically significant toxicities from prior chemotherapy or surgery must be less than Grade 2;
• •7. Serum creatinine ≤ 2.0 mg/dL;
• •8. Total serum bilirubin ≤ 1.5 × ULN unless considered due to Gilbert's syndrome or leukemic organ involvement;
• •9. Serum AST or ALT ≤ 3.0 × ULN unless considered due to leukemic organ involvement;
• •10. Females of childbearing potential must have a negative pregnancy test (urine β-hCG);
• •11. Females of childbearing potential and sexually mature males must agree to use a medically accepted method of contraception throughout the study;
• •12. Written informed consent must be provided.