Obatoclax for Systemic Mastocytosis
Condition: Leukemia; Systemic Mastocytosis
MeSH terms: Mastocytosis; Mastocytosis, Systemic
Intervention: Obatoclax Mesylate
Intervention description: 30 mg given by vein over 3 hours on Days 1-3 of each 14-day study cycle.
Intervention MeSH terms: Obatoclax
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Non-Randomized
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 3.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: Leukemia; Systemic Mastocytosis; SM; Obatoclax mesylate; GX15-070MS

Summary:

      The goal of this clinical research study is to learn if obatoclax mesylate can help to
      control systemic mastocytosis. The safety of this drug will also be tested.
    

Eligibility criteria:

        Inclusion Criteria:

          1. Patients with Systemic Mastocytosis (SM); including mast cell leukemia.

          2. Age equal to or greater than 18 years

          3. Minimum of two weeks since any major surgery or completion of radiation

          4. Eastern Cooperative Oncology Group (ECOG) performance status equal to or less than 2

          5. Adequate liver function as shown by serum bilirubin equal to or less than 1.5 * upper
             limit of normal (ULN), and serum alanine aminotransferase (ALT) equal to or less than
             3 * ULN.

          6. Signed informed consent

        Exclusion Criteria:

          1. Patients with low blood cell counts (Grade 3 or 4), unless it is known that the low
             blood cell count is due to systemic mastocytosis.

          2. Treatment with any conventional (specifically, interferon or cladribine) or
             investigational medicine for SM within the preceding 4 weeks

          3. Chronic treatment with systemic steroids (unless limited to 10 mg prednisone
             equivalent per day or less) or another immunosuppressive agent

          4. Other malignancies within the past 3 years except for adequately treated carcinoma of
             the cervix or basal or squamous cell carcinomas of the skin. Patient may have
             SM-associated clonal hematologic disease that does not require therapy, as judged by
             the treating physician and approved by the principal investigator).

          5. Other concurrent severe and/or uncontrolled medical disease which could compromise
             participation in the study as judged by the Principal Investigator (i.e., severely
             impaired lung function, uncontrolled diabetes, uncontrolled hypertension, severe
             infection, severe malnutrition, unstable angina, or congestive heart failure - New
             York Heart Association Class III or IV, ventricular arrhythmias, active ischemic heart
             disease, myocardial infarction within six months, chronic liver or renal disease,
             active upper GI tract ulceration)

          6. A known history of human immunodeficiency virus (HIV) seropositivity

          7. Women who are pregnant or breast feeding,or women/men able to conceive and unwilling
             to practice an effective method of birth control. Women of childbearing potential must
             have a negative urine or serum pregnancy test within 48 hours prior to administration
             of obatoclax. Women of childbearing potential is defined as women who have not
             undergone a hysterectomy or bilateral oophorectomy, has not been naturally
             postmenopausal for at least 24 consecutive months.

          8. Patients with a known hypersensitivity to obatoclax mesylate or to its excipients (PEG
             300 and Tween 20)

          9. Patients unwilling to or unable to comply with the protocol