A Phase I/II Study of MGCD0103 (MG-0103) in Combination With Azacitidine in Patients With High-Risk Myelodysplastic Syndrome 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 >38.5˚C 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.