Study on Bone Marrow Morphology in Adults Receiving Romiplostim for Treatment of Thrombocytopenia Associated With Immune Thrombocytopenia Purpura (ITP)
Condition: Thrombocytopenia; Idiopathic Thrombocytopenic Purpura
MeSH terms: Thrombocytopenia; Purpura; Purpura, Thrombocytopenic, Idiopathic; Purpura, Thrombocytopenic
Intervention: romiplostim
Intervention description: Romiplostim administered by subcutaneous injection
Phase: Phase 4
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 169.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: Idiopathic Thrombocytopenic Purpura; Idiopathic Thrombocytopenia Purpura; Immune Thrombocytopenic Purpura; Immune Thrombocytopenia; ITP

Summary:

      The purpose of this study is to evaluate changes in bone marrow morphology (structure) after
      long-term exposure to romiplostim.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Diagnosis of ITP according to American Society of Hematology (ASH) guidelines

          -  Subject must have had a bone marrow biopsy within one year prior to planned first dose
             of romiplostim (with available bone marrow tissue block or unstained histological
             slides to send to a central laboratory for interpretation) or must consent to a
             pre-treatment bone marrow biopsy within 3 weeks prior to planned first dose of
             romiplostim. Central laboratory interpretation is required prior to first dose of
             romiplostim

          -  Subject must agree to a scheduled bone marrow biopsy at Year 1, Year 2, or Year 3
             following romiplostim treatment and any unscheduled biopsies if clinically indicated

          -  Subject ≥18 years of age

          -  Baseline bone marrow reticulin grade of 0, 1, 2, or 3 according to the modified
             Bauermeister grading scheme as assessed by central laboratory interpretation

          -  Platelet count < 50 x 10^9/L

          -  Must have received at least 1 prior ITP therapy (examples of ITP therapy include
             corticosteroids, intravenous immunoglobulin [IVIG], splenectomy)

          -  Subject (or legally-acceptable representative) is willing and able to provide written
             informed consent

        Exclusion Criteria:

          -  Baseline bone marrow biopsy positive for collagen fibrosis

          -  Any known history of or currently active bone marrow stem cell disorder, hematological
             malignancy, myeloproliferative disorder, myelodysplastic syndrome

          -  Any current active malignancy

          -  Any prior exposure to cytostatic chemotherapy or radiotherapy for malignancy

          -  Subject has undergone pacemaker placement, cardiac ablation of arrhythmia, and/or any
             current treatment with Vaughan Williams Class IA - IC and Class III agents (Vaughan
             Williams, 1970)

          -  Subject has participated in any study evaluating pegylated recombinant human
             megakaryocyte growth and development factor (PEG-rHuMGDF), recombinant human
             thrombopoietin (rHuTPO), or thrombopoietin receptor agonists (ie romiplostim or
             eltrombopag)

          -  Subject has a known hypersensitivity to any recombinant E coli-derived product

          -  Subject is currently enrolled in or has not yet completed (at least 4 weeks since
             ending) other investigational device or drug trial(s) or subject is receiving other
             investigational agent(s)

          -  Other investigational procedures are excluded

          -  Subject of child-bearing potential is evidently pregnant (eg positive pregnancy test)
             or is breast feeding

          -  Subject is not using adequate contraceptive precautions

          -  Subject has any kind of disorder that compromises the ability of the subject to give
             written informed consent and does not have a legally acceptable representative and/or
             is unable to comply with study procedures.