CEP-701 for PH-negative Myelofibrosis
Condition: Leukemia; Myelofibrosis
MeSH terms: Primary Myelofibrosis
Intervention: CEP-701
Intervention description: 80 mg orally twice a day for 30 days
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 27.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: CEP-701; Leukemia; Myelofibrosis; MF; JAK2; Tyrosine Kinase Inhibitor; Lestaurtinib; Chronic Idiopathic Myelofibrosis; CIMF

Summary:

      The goal of this clinical research study is to find out if CEP-701 can help control
      myelofibrosis (MF). The safety of CEP-701 will also be studied.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Patients with Chronic Idiopathic Myelofibrosis (CIMF) requiring therapy, including
             those 1) previously treated by CIMF-directed therapy and relapsed, intolerant, or
             refractory to therapy; or 2) if newly diagnosed then with intermediate or high risk
             according to Lille scoring system (adverse prognostic factors are: Hb < 10 g/dl, WBC <
             4 or > 30 x 10^9/L; risk group: 0 = low, 1 = intermediate, 2 = high), or with
             symptomatic spleen that is >/= 10cm below costal margin. However, patients with
             asymptomatic intermediate risk disease are not eligible.

          -  JAK2 mutation positive test

          -  Age of at least 18 years

          -  Eastern Cooperative Oncology Group (ECOG) performance status 0-2

          -  Adequate liver and renal function: total bilirubin </=2.0 mg/dL, alanine
             aminotransferase (ALT or SGPT) </=2.0 x institutional upper limit of normal (ULN), and
             creatinine </=2.0 mg/dl

          -  Patients must be at least 2 weeks from prior chemotherapy, biological therapy,
             radiation therapy, major surgery, or other investigational anticancer therapy that is
             considered MF-directed, and have recovered from prior toxicities to Grade 0-1.
             Concurrent therapy with supportive care medications (hydroxyurea, anagrelide) is
             allowed during the study.

          -  All men of reproductive potential and women of child-bearing potential (WOCBP) must
             agree to practice effective contraception (iud, birth control pill, latex condoms,
             diaphragm) during the entire study period and for one month after the study ends,
             unless documentation of infertility exists. Should a woman become pregnant or suspect
             she is pregnant while participating in this study, she should inform her treating
             physician immediately. WOCBP are women who are not menopausal for 12 months or no
             previous surgical sterilization."

          -  Ability to understand and willingness to sign the informed consent form

          -  Not willing to undergo, not a candidate for, or not having a donor for, a bone marrow
             transplant

        Exclusion Criteria:

          -  Pregnant or nursing women, due to the unknown effects of therapy on the developing
             fetus or newborn infant.

          -  Patients diagnosed with another malignancy - unless following curative intent therapy
             the patient has been disease free for at least 3 years. Patients with early stage
             squamous cell carcinoma of the skin, basal cell carcinoma of the skin, or cervical
             intraepithelial neoplasia (CIN) are eligible for this study

          -  Any condition, including serious medical condition, laboratory abnormality, or
             psychiatric illness, which places the subject at unacceptable risk as judged by the
             Principal Investigator, if he/she was to participate in the study

          -  Known positive for Human immunodeficiency virus (HIV) or infectious hepatitis, type A,
             B or C

          -  Presence of any gastrointestinal condition or concomitant medication use (e.g.
             coumadin) that would render a patient at high risk for gastrointestinal bleeding as
             judged by treating physician

          -  History of any upper or lower gastrointestinal bleeding in the 6 months prior to
             enrollment

          -  Elevated international normalized ratio (INR) or Partial thromboplastin time (PTT)