A Phase I/II Study of MGCD0103 With Azacitidine in Patients With High-Risk Myelodysplastic Syndrome (MDS) or Acute Myelogenous Leukemia

Inclusion Criteria:

Patients must have high-risk MDS (≥ 10% BM blasts) or AML

RAEB (RA with excess blasts) with ≥10% BM blasts: 10%-20% blasts in BM, <5% blasts in peripheral blood
RAEB-T (RAEB in transformation): 21%-30% blasts in BM, <5% blasts in peripheral blood, absolute monocytosis (>109/L)
AML
Disease may be relapsed/refractory or de novo. Once the MTD has been determined, all subsequent patients in the phase II portion of the study should have no prior azacitidine
ECOG performance status of 0, 1, or 2
Age ≥18 years
Laboratory requirements
Patients or their legal representative must be able to read, understand, and sign a written informed consent (approved by the institutional review board/Ethics Committee (IRB/EC)) within 14 days prior to start of treatment

Exclusion Criteria:

Patients with another active cancer (excluding basal cell carcinoma or cervical intraepithelial neoplasia (CIN / cervical in situ)). Prior history of cancer is allowed, as long as there is no active disease
Pregnant or lactating women. Women of child-bearing potential (WOCBP) must have a negative serum pregnancy test documented within 7 days prior start of study drug
WOCBP and men whose partners are WOCBP must use an acceptable method of contraception while enrolled on this study, and for a period of 3 months following study drug treatment. Patients unwilling or unable to follow this guideline will be excluded. Examples of acceptable forms of contraception include an oral contraceptive or a double barrier method, such as condom with diaphragm
Patients with uncontrolled intercurrent illness, active or uncontrolled infections, or a fever >38.5ËšC on the day of scheduled dosing
Patients with serious illnesses, medical conditions, or other medical history, including laboratory results, which, in the investigator's opinion, would be likely to interfere with a patient's participation in the study, or with the interpretation of the results
Patients who have been treated with any investigational drug within 30 days prior to study initiation (an investigational drug is one for which there is no approved indication), or who are receiving concurrent treatment with other experimental drugs or anti-cancer therapy
Known hypersensitivity to HDAC inhibitors, to any of the components of MG-0103 or Vidaza, including mannitol
Prior treatment with azacitidine during the expanded phase II portion only
Known HIV or active Hepatitis B or C
Any condition (e.g., known or suspected poor compliance, psychological instability, geographical location, etc) that, in the judgment of the investigator, may affect the patient's ability to sign the informed consent and undergo study procedures
Any condition that will put the patient at undue risk or discomfort as a result of adherence to study procedures. For example, consider requirement to take MG-0103 with an acidic drink and recommendation to avoid agents that increase gastric-pH.