Evaluating the Safety and Efficacy of Romiplostim (AMG 531) in Thrombocytopenic Subjects With Immune Thrombocytopenic Purpura (ITP)
Condition: Idiopathic Thrombocytopenic Purpura
MeSH terms: Purpura; Purpura, Thrombocytopenic; Purpura, Thrombocytopenic, Idiopathic
Intervention: Romiplostim; Placebo
Intervention description: Administered by subcutaneous injection; Administered by subcutaneous injection
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Sequential Assignment
Intervention model description: Part A was a sequential cohort dose escalation study. Part B was a randomized, placebo-controlled parallel group study.
Masking: Double (Participant, Investigator)
Enrollment: 45.0
Sex: All
Minimum age: 18 Years
Maximum age: 65 Years
Healthy volunteers: No
Keywords: Immune Thrombocytopenic Purpura; Idiopathic Thrombocytopenic Purpura; Thrombocytopenic; Thrombocytopenia; ITP

Summary:

      The primary objective of this study is to evaluate the safety and tolerability of romiplostim
      in thrombocytopenic patients with ITP.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Diagnosis of ITP according to American Society of Hematology (ASH) guidelines at least
             3 months before enrollment

          -  Have completed at least 1 prior treatment for ITP

          -  Two (including day -2) of the 3 platelet counts taken during the screening and
             pre-treatment periods must have fulfilled the following:

               -  less than 30 x 10^9/L for those subjects not receiving any ITP therapy,

               -  less than 50 x 10^9/L for those subjects receiving any ITP therapy

          -  Eastern Cooperative Oncology Group performance status of 0 to 2

          -  Serum creatinine concentration ≤ 2 mg/dL (≤ 176.8 µmol/L)

          -  Adequate liver function, as evidenced by a serum bilirubin ≤ 1.5 times the laboratory
             normal range

          -  Hemoglobin greater than 10.0 g/dL

          -  Written informed consent

        Exclusion Criteria:

          -  Considered a substantial risk for adverse outcomes because of a clinically important
             trend (as determined by the investigator) detected in the platelet counts during the
             screening period

          -  Any known history of bone marrow stem cell disorder

          -  Any active malignancy. If prior history of cancer other than basal cell carcinoma or
             cervical carcinoma in situ, no treatment or active disease within 5 years before
             randomization

          -  Documented diagnosis of arterial thrombosis (ie, stroke, transient ischemic attack, or
             myocardial infarction) in the previous year; history of venous thrombosis (ie, deep
             vein thrombosis, pulmonary embolism) and receiving anticoagulation therapy

          -  Unstable or uncontrolled disease or condition related to or impacting cardiac function
             (e.g., unstable angina, congestive heart failure [New York Heart Association (NYHA)
             greater than class II], uncontrolled hypertension [diastolic greater than 100 mmHg] or
             cardiac arrhythmia)

          -  Have 3 or more of the following predisposing factors for thromboembolic events:
             diabetes; smoker using oral contraceptives; hypercholesteremia (> 240 mg/dL);
             treatment for hypertension

          -  Known positive test for human immunodeficiency virus (HIV) infection or hepatitis C
             virus

          -  Received any treatment for ITP (except for a constant dose schedule of
             corticosteroids) within 4 weeks before the screening visit

          -  Received intravenous (IV) immunoglobulin (Ig) or WinRho within 2 weeks before the
             screening visit

          -  Received hematopoietic growth factors, including interleukin (IL)-11 (Neumega®) within
             4 weeks before the screening visit

          -  Past or present participation in any study evaluating polyethylene glycol recombinant
             human magakaryopoiesis differentiating factor (PEG-rHuMGDF), recombinant human
             thrombopoietin (rHuTPO), or related platelet product

          -  Received any alkylating agents within 8 weeks before the screening visit or
             anticipated use during the time of the proposed study

          -  Received any monoclonal antibody (eg, rituximab) within 16 weeks before the screening
             visit or anticipated use during the time of the proposed study

          -  Less than 4 weeks since receipt of any therapeutic drug or device that is not FDA
             approved for any indication before the screening period

          -  Less than 2 months since major surgery (including laparoscopic splenectomy)

          -  Pregnant or breast feeding

          -  Subjects of reproductive potential who are not using adequate contraceptive
             precautions, in the judgment of the investigator