Pomalidomide for Myelofibrosis Patients
Condition: Polycythemia Vera; Thrombocythemia
MeSH terms: Polycythemia Vera; Primary Myelofibrosis; Polycythemia; Thrombocytosis
Intervention: CC-4047; Prednisone; CC-4047
Intervention description: 0.5 mg capsules daily by mouth day 1 through day 28.; 30 mg by mouth daily during cycle 1, 15 mg/day during cycle 2, and 15 mg every other day during cycle 3, and then it will be discontinued.; 3.0 mg capsules daily by mouth day 1 through 21.
Intervention MeSH terms: Thalidomide; Prednisone; Pomalidomide
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Non-Randomized
Intervention model: Parallel Assignment
Intervention model description: The first 21 participants received pomalidomide 3.0 mg/day on a 21 day schedule. Due to poor tolerance the study was suspended and amended to treat 20 participants with pomalidomide 0.5 mg/day continuously for 28 days. The study was amended to treat an additional 29 participants with pomalidomide 0.5 mg/day for 28 days plus prednisone.
Masking: None (Open Label)
Enrollment: 70.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: CC-4047; Pomalidomide; polycythemia vera; essential thrombocythemia myelofibrosis

Summary:

      The goal of this clinical research study is to learn if CC-4047 (now called pomalidomide) and
      prednisone can help to control MMM. The safety of this therapy will also be studied.
    

Eligibility criteria:

        Inclusion Criteria:

          1. Must be >/= 18 years of age at the time of voluntarily signing an Institutional Review
             Board/Independent Ethics Committee (IRB/IEC) - approved informed consent form.

          2. Must be diagnosed with myelofibrosis requiring therapy including myelofibrosis with
             myeloid metaplasia (MMM), de novo presentation (i.e. agnogenic myeloid metaplasia
             [AMMM], and developing after an antecedent history of Polycythemia vera (i.e.,
             post-polycythemic myeloid metaplasia [PPMM]), or essential Polycythemia (i.e., post
             thrombocythemic myeloid metaplasia [PTMM]).

          3. Screening total hemoglobin level < 10 g/dL or transfusion-dependent anemia defined as
             per International Working Group (IWG) criteria (transfusion dependency defined by a
             history of a least 2 units of red blood cell transfusions in the last 28 days for
             hemoglobin < 8.5 g/dL that was not associated with overt bleeding)

          4. Must have adequate organ function as demonstrated by the following </= 14 days prior
             to starting study drug: ·Alanine transaminase (ALT) (SGOT) and Aspartate
             aminotransferase (AST) (SGPT) </= 3 x upper limit of normal (ULN), [unless upon
             judgment of the treating physician, it is believed to be due to extramedullary
             hematopoiesis (EMH)] ·Total bilirubin < 3 x ULN or Direct Bilirubin < 2 x ULN ·Serum
             creatinine </= 2.5 mg/dL ·Absolute neutrophil count >/= 1,000/µL (>/=1.0 x 10^9/L)
             ·Platelet count >/= 50,000/µL (>/=50 x 10^9/L)

          5. Subjects must be willing to receive transfusion of blood products

          6. Eastern Cooperative Oncology Group (ECOG) performance status (PS) of 0, 1, or 2 at
             screening.

          7. Must be willing to adhere to the study visit schedule and other protocol requirements.

          8. No active malignancies with the exception of basal cell or squamous cell carcinoma of
             the skin, or carcinoma (in situ) of the cervix or breast

          9. All study participants must be registered into the mandatory POMALYST REMS™ program,
             and be willing and able to comply with the requirements of the POMALYST REMS™ program.

         10. Females of reproductive potential (FCBP†) must adhere to the scheduled pregnancy
             testing as required in the POMALYST REMS™ program. Able to take aspirin (81 or 325 mg)
             daily as prophylactic anticoagulation (patients intolerant to ASA may use warfarin or
             low molecular weight heparin).

        Exclusion Criteria:

          1. Known positive status for HIV, hepatitis B carrier, or active hepatitis C infection.

          2. The use of any growth factors, cytotoxic chemotherapeutic agents (e.g. hydroxyurea),
             corticosteroids, or experimental drug or therapy within 14 days of starting CC-4047
             and/or lack of recovery from all toxicity from previous therapy to grade 1 or better.

          3. Any serious medical condition or psychiatric illness that would prevent, (as judged by
             the treating physician) the subject from signing the informed consent form or any
             condition, including the presence of laboratory abnormalities, which places the
             subject at unacceptable risk if he/she were to participate in the study or confounds
             the ability to interpret data from the study.

          4. Pregnant or lactating females

          5. Prior use of CC-4047

          6. Currently enrolled on another clinical trial or receiving investigational agent