Clinical trial NCT01178281 (phase 3, status: completed)
Conditions: primary myelofibrosis, mpn-associated myelofibrosis
ICD codes: D75.81, C94.41, C94.42, C94.40, D75.81, C94.41, C94.42, C94.40
Drugs: pomalidomide 0.5 mg, placebo, pomalidomide
SMILES: NC1=CC=CC2=C1C(=O)N(C1CCC(=O)NC1=O)C2=O, CN1C(=O)C=C(N2CCC[C@@H](N)C2)N(CC2=C(C=CC=C2)C#N)C1=O, [H][C@@]12C[C@@H](C)[C@](O)(C(=O)CO)[C@@]1(C)C[C@H](O)[C@@]1(F)[C@@]2([H])CCC2=CC(=O)C=C[C@]12C
Eligibility criteria:

        Inclusion Criteria:

          -  Age ≥ 18 years

          -  Myeloproliferative-neoplasm (MPN)-associated myelofibrosis

          -  RBC-transfusion-dependence (global study):

               -  Average RBC-transfusion frequency ≥ 2 units/28 days over at least the 84 days
                  immediately prior to randomization. There must be no interval > 42 days without ≥
                  1 RBC-transfusion.

               -  Only RBC-transfusions given when the hemoglobin ≤ 90 g/L³ are scored in

        determining eligibility.

          -  RBC-transfusions due to bleeding are not scored in determining eligibility.

          -  RBC-transfusions due to chemotherapy-induced anemia are not scored in determining
             eligibility.

               -  Severe anemia (China-specific extension):

          -  ≥ 2 hemoglobin concentrations ≤ 80 g/L for ≥ 84 days immediately before the day of
             enrollment.

          -  No RBC-transfusion within 6 months prior to enrollment.

               -  Hemoglobin ≤ 130 g/L at randomization (global study); ≤ 80 g/L at enrollment in
                  the China-specific extension.

               -  Bone marrow biopsy within 6 months (global study only).

               -  Inappropriate to receive blood cell or bone marrow allotransplant, erythropoietin
                  and androgenic steroids

               -  Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2.

               -  Agree to follow pregnancy precautions as required by the protocol.

               -  Agree to receive counseling related to teratogenic and other risks of
                  pomalidomide.

               -  Agree not to donate blood or semen.

        Exclusion Criteria:

          -  Prior blood cell or bone marrow allotransplant.

          -  Use of drugs to treat MPN-associated myelofibrosis ≤ 30 days before starting study
             drug.

          -  Treatment with erythropoietin or androgenic steroids ≤ 84 days before starting study
             drug.

          -  Anemia due to reasons other than MPN-associated myelofibrosis.

          -  Pregnant or lactating females.

          -  More than 10% blasts by bone marrow examination or more than 10% blasts in blood in
             consecutive measurements spanning at least 8 weeks

          -  Prior history of malignancies,other than the disease being studied, unless the subject
             has been free of the malignancy for ≥ 5 years with the following exceptions:

               -  Carcinoma in situ of the cervix

               -  Carcinoma in situ of the breast

               -  Incidental histologic finding of prostate cancer (T 1a or T 1b using TNM [tumor,
                  nodes, metastasis] clinical staging system)

          -  Human immunodeficiency virus (HIV) infection, active hepatitis B virus (HBV) or
             hepatitis C virus (HCV) infections.

          -  Prior treatment with pomalidomide.

          -  Allergic reaction or rash after treatment with thalidomide or lenalidomide

          -  Any of the following laboratory abnormalities:

               -  Neutrophils < 0.5x10^9 /L

               -  Platelets < 25 x 10^9 /L

               -  Estimated glomerular filtration rate (kidney function) < 30 mL/min/1.73 m²

               -  Aspartate aminotransferase (AST) and alanine transaminase (ALT) > 3.0 x upper
                  limit of normal (ULN)

               -  Total bilirubin ≥ 4 x ULN;

          -  Uncontrolled hyperthyroidism or hypothyroidism.

          -  Deep venous thrombosis (DVT) or pulmonary embolus (PE) < 6 months before starting
             study drug

          -  Clinically-important heart disease within the past 6 months