A Phase I/II, Prospective, Open-Label Study to Determine the Safety and Efficacy of CC-4047 in Patients With Primary, Post Polycythemia Vera, or Post Essential Thrombocythemia Myelofibrosis®

Inclusion Criteria

DISEASE CHARACTERISTICS:

- Diagnosis of primary and post essential thrombocythemia (ET) or post polycythemia
vera (PV) myelofibrosis requiring therapy

- De novo presentation (i.e., agnogenic myeloid metaplasia AND post ET or post PV
myelofibrosis)

- Developed after an antecedent history of PV (i.e., post polycythemic myeloid
metaplasia) or essential polycythemia (i.e., post thrombocythemic myeloid
metaplasia)

- Total hemoglobin < 10 g/dL OR transfusion dependent anemia (defined by a history of ≥
2 units of RBC transfusions within the past 28 days for hemoglobin < 8.5 g/dL that
was not associated with overt bleeding) OR marked splenomegaly (e.g., ≥ 10 cm below
costal margin)

PATIENT CHARACTERISTICS:

- ECOG performance status 0-2

- ANC ≥ 500/μL

- Platelet count ≥ 20,000/μL

- AST and ALT ≤ 3 times upper limit of normal (ULN) (≤ 5 times ULN if attributed to
hepatic extramedullary hematopoiesis)

- Total bilirubin ≤ 3 times ULN OR direct bilirubin ≤ 2 times ULN

- Serum creatinine ≤ 2.0 mg/dL

- Not pregnant or nursing

- Negative pregnancy test

- Fertile patients must use effective double-method contraception for ≥ 28 days before,
during, and for ≥ 28 days after completion of study treatment

- Agrees to abstain from donating blood, semen, or sperm during and for ≥ 28 days after
completion of study treatment

- Willing to undergo transfusion of blood products (if applicable)

- Able to complete questionnaire(s) alone or with assistance

- No known HIV positivity, hepatitis B carrier, or active hepatitis C infection

- No serious medical condition, psychiatric illness, or any other condition, including
the presence of laboratory abnormalities, that (as judged by the treating physician)
would preclude giving informed consent or participating in the study or confound the
ability to interpret data from the study

- No other active malignancies, except basal cell or squamous cell carcinoma of the
skin or carcinoma in situ of the cervix or breast

- No active deep vein thrombosis or pulmonary embolism that has not been
therapeutically anticoagulated

PRIOR CONCURRENT THERAPY:

- Recovered from all prior therapy

- No prior CC-4047

- More than 28 days since prior growth factors, cytotoxic chemotherapeutic agents
(e.g., hydroxyurea or anagrelide), corticosteroids, or experimental drugs or
therapies

- No other concurrent experimental drugs or therapies or cytotoxic chemotherapeutic
agents (e.g., hydroxyurea or anagrelide) for myelofibrosis

- No concurrent growth factors (including erythropoietin) for myelofibrosis, except
G-CSF or pegfilgrastim

- No concurrent chronic use (i.e., > 2 weeks) of more than physiologic doses of
corticosteroids (dose equivalent to > 10 mg/day of prednisone)