MGCD0103 in Elderly Patients With Previously Untreated AML/High Risk MDS or Adults With Relapsed/Refractory Disease

A Phase II Study of MGCD0103 (MG-0103) in Patients With Acute Myelogenous Leukemia/High Risk MDS Who Are Elderly and Have Previously Untreated Disease or Who Are Adult and Have Relapsed/Refractory Disease

In this study, MGCD0103, a new anticancer drug under investigation, is given three times per week to elderly patients with previously untreated acute myelogenous leukemia/high risk myelodysplastic syndrome or adults with relapsed/refractory disease.

Inclusion Criteria:

• Pathologic confirmation of AML or high risk MDS.
• Elderly cohort (70 years of age or more): no prior chemotherapy treatment for AML/high risk MDS and not currently candidates for intensive chemotherapy.
• Relapsed/refractory cohort (18 years of age or more): relapsed or refractory to prior treatment.
• ECOG performance status of 0 or 1.
• Total bilirubin < 1.5 x upper limit of normal (ULN).
• AST/SGOT and ALT/SGPT < 2.5 x ULN.
• Serum creatinine < 1.5 x ULN.
• Patients must read, understand, and sign a written informed consent form (ICF).
• Women of childbearing potential and male patients' female partners must use an acceptable method of contraception while on study, and for 3 months after study drug treatment.

Exclusion Criteria:

• Pregnant or lactating women.
• Patients with uncontrolled, intercurrent illness, active or uncontrolled infections, or a fever > 38.5C (not due to tumor fever) on the day of scheduled dosing.
• Patients with serious illnesses or medical conditions, including laboratory results, which, in the investigator's opinion, would interfere with a patient's participation, or with the interpretation of the results.
• Patients treated with an investigational drug within 30 days prior to study initiation.
• Known hypersensitivity to HDAC inhibitors or to any of the components of MG-0103.
• Known HIV or active hepatitis B or C.
• Any condition that may affect the patient's ability to sign the ICF and undergo study procedures.
• Any conditions that will put the patient at undue risk or discomfort as a result of adherence to study procedures.