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

Study Overview
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Brief Summary
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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.

Official Title
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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

Conditions
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Myelogenous Leukemia, Acute, Myelodysplastic Syndromes

Intervention / Treatment
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* Drug: MGCD0103


Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| No Intervention: 1<br>Elderly (≥65 years) untreated arm | |
| Experimental: 2<br>Relapsed/Refractory Arm | Drug: MGCD0103<br>* MGCD0103 administered orally three-times per week<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety and tolerability |  | 1 year (anticipated | 
| Response rate |  | 1 year (anticipated) | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Progression-free survival |  | 1 year (anticipated) | 
| Duration of objective response |  | 1 year (anticipated) | 
| Pharmacokinetics |  | 1 year (anticipated) | 
| Pharmacodynamics |  | 1 year (anticipated) | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Leukemia, MDS, Phase II, Acute Myelogenous Leukemia (AML), High Risk Myelodysplastic Syndromes (MDS)