Safety and Efficacy of Obatoclax Mesylate (GX15-070MS)for the Treatment of Myelodysplastic Syndromes (MDS)

Study Overview
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Brief Summary
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Defects in the apoptotic process can lead to the onset of cancer by allowing cells to grow unchecked when an oncogeneic signal is present. Obatoclax is designed to restore apoptosis through inhibition of the Bcl-2 family of proteins, thereby reinstating the natural process of cell death that is often inhibited in cancer cells. This is a multi-center, open-label, Phase II study of obatoclax administered in 2-week cycles to patients with previously-untreated Myelodysplastic Syndromes with anemia and/or thrombocytopenia. Treatment may be administered on an outpatient basis. No investigational or commercial agents or therapies other than those described herein may be administered with the intent to treat the patient's malignancy. Supportive care measures including those directed at controlling symptoms resulting from Myelodysplastic Syndromes are allowed

Official Title
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A Phase II Study of Obatoclax Mesylate (GX15-070MS) in Patients With Previously-Untreated Myelodysplastic Syndromes (MDS) With Anemia and/or Thrombocytopenia

Conditions
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Myelodysplastic Syndromes

Intervention / Treatment
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* Drug: Obatoclax mesylate (GX15-070MS)


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Pathological confirmation of Myelodysplastic Syndromes (MDS) Patients must have had no prior systemic therapy Must have normal organ functions Must have the ability to understand and willingness to sign a written informed consent form Exclusion Criteria: Must not be a result of prior chemotherapy and/or radiotherapy for another malignancy No other agents or therapies administered in the intent to treat Uncontrolled, intercurrent illness Pregnant women and women who are breast feeding

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
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Obatoclax Mesylate<br>Obatoclax Mesylate 30mg | Drug: Obatoclax mesylate (GX15-070MS)<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| International Working Group (IWG) Response Criteria for MDS | Determine the response rate according to bone marrow blast count less than or equal to 10% | 52 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Peripheral blood counts; Bone marrow aspirates and biopsies; Transfusions and growth factor requirements | hemoglobin level less than 10 g/dL and or platelets less than 50 x 10 9/L. Eastern Cooperative Onocology Group (ECOG) performance status: 0 fully active-2 ambulatory 50% of the time; and total bilirubin less than or equal to 2 mg/dL; normal limits of SGOT/SGPT and creatinine according to laboratory standards | 52 weeks |