Safety and Efficacy of Obatoclax Mesylate (GX15-070MS) in the Treatment of Myelofibrosis With Myeloid Metaplasia

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 oncogenic 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 II study of single-agent obatoclax administered in 2-week cycles as a 24-hour infusion every 2 weeks at a fixed dose of 60 mg to patients with myelofibrosis. Infusions may be administered on an outpatient basis. No investigational or commercial agents or therapies other than those described in 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 myelofibrosis (blood products, growth factor, hydroxyurea, etc.) are allowed.

Official Title
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A Multi-Center, Open-Label, Phase II Study of Single-Agent GX15-070MS Administered as a 24-Hour Infusion Every 2 Weeks to Patients With Myelofibrosis With Myeloid Metaplasia (MF)

Conditions
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Myelofibrosis

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 confirmed myelofibrosis with myeloid metaplasia. No limitations on allowable type and amount of prior therapy. Patients must have normal organ function. Must be willing to submit to blood sampling for planned PK and PD analyzes. Must have ability to understand and willingness to sign a written informed consent form. Exclusion Criteria: No other agents or therapies administered with the intent to treat malignancy. Patients with prior exposure to obatoclax. 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: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: GX15-070MS<br>Obatoclax mesylate 60mg | Drug: Obatoclax mesylate (GX15-070MS)<br>* 60 mg q2wks<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Determine the steady-state pharmacokinetic parameters and pharmacodynamic response. |  | 1 Year | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Peripheral blood counts |  | 4 Weeks to 1 Year | 
| Bone marrow aspirates and biopsies |  | 8 weeks to 1 year | 
| Transfusion and growth factor requirements |  | 8 weeks to 1 year |