Safety and Efficacy of Obatoclax Mesylate (GX15-070MS) for the Treatment of Hematological Malignancies

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 oncogneic 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.

Detailed Description
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This is a multi-center, open-label, Phase I study of obatoclax administered every 2-week or weekly cycles, or as a Prolonged Infusion every 2 to 3 weeks to patients with Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome (MDS), Chronic Myeloid Leukemia (CML)in Myeloid Blast Phase, Myelofibrosis, Previously-Treated Chronic Lymphocytic Leukemia (CLL), or Aplastic Anemia. Due to the PK/PD sampling schedule Cycle 1 will require overnight hospitalization. For the following cycles treatment may be administered on an outpatient basis but is at the discretion of the investigator. No investigational or commercial agents or therapies other than those described within the protocol may be administered with the intent to treat the patient's malignancy. Supportive care measures including those directed at controlling symptoms resulting from hematological malignancies are allowed.

Official Title
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A Phase I Study of Obatoclax Mesylate (GX15-070MS) in Patients With Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome (MDS), Chronic Myeloid Leukemia (CML)in Myeloid Blast Phase, Myelofibrosis, Previously-Treated Chronic Lymphocytic Leukemia (CLL), or Aplastic Anemia

Conditions
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Hematological Malignancies

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: Histologically or cytologically confirmation of AML, MDS, CML in blast phase, myelofibrosis, CLL, or aplastic anemia There are no limitations on additional, allowable type and amount of prior therapy as long as acute toxicities have resolved to the allowable grade. Must have normal organ functions Must be willing to submit to blood sampling for the planned PK and PD analyses. Must have the ability to understand and willingness to sign a written informed consent form Exclusion Criteria: No other agents or therapies administered for 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
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

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

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Determine the recommended Phase II dose of GX15-070MS; Characterize the DLTs of GX15-070MS; Determine the PK/PD response to GX15-070MS |  |  | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Describe any clinical responses of patient with hematological malignancies. |  |  |