Long-term Study of Romiplostim in Thrombocytopenic Pediatric Patients With Immune Thrombocytopenia (ITP)

This is an interventional treatment trial for Immune Thrombocytopenia focused on measuring Pediatric, Immune Thrombocytopenia, Amgen Read More

Inclusion Criteria:

• Diagnosis of primary ITP according to The American Society of Hematology (ASH) Guidelines at least 6 months before screening, regardless of splenectomy status
• Age ≥ 1 year and < 18 years of age
• Refractory to prior ITP therapy, relapsed after at prior ITP therapy, or be ineligible for other therapies. Examples of prior therapy include: corticosteroids, intravenous Immunoglobulin (IVIG), anti-D immunoglobulin, platelet transfusions.
• Platelet count ≤ 30 x10^9/L or is experiencing uncontrolled bleeding
• Has provided informed consent before any study-specific procedure;

• Adequate hematologic, renal, and liver function during screening:

  • Hemoglobin > 10.0 g/dL
  • Serum creatinine ≤ 1.5 x the upper limit of normal (ULN)
  • Total serum bilirubin ≤ 1.5 x the ULN
  • Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 3.0 x the ULN
• For the EU, Switzerland and Turkey protocol supplement, subject must agree to a scheduled bone marrow biopsy and aspirate at Year 1 or Year 2 following romiplostim treatment and any unscheduled biopsies if clinically indicated
• For the EU, Switzerland and Turkey protocol supplement, a reticulin grade of 0, 1, 2, or 3 according to the modified Bauermeister grading scale, as assessed by central laboratory from a bone marrow biopsy performed within 1 year prior to planned first dose of romiplostim or consent to a pre-treatment bone marrow biopsy and aspirate prior to planned first dose of romiplostim

Exclusion Criteria:

• History of a bone marrow stem cell disorder (Any abnormal bone marrow findings other than those typical of ITP must be approved by Amgen before a subject may be enrolled)
• Prior bone marrow transplant or peripheral blood progenitor cell transplant
• Active or prior malignancy except non-melanoma skin cancers within the last 5 years
• History of myelodysplastic syndrome
• History of bleeding diathesis
• History of congenital thrombocytopenia
• History of Hepatitis B, Hepatitis C or human immunodeficiency virus (HIV)
• History of systemic lupus erythematosus, Evans syndrome, or autoimmune neutropenia
• History of antiphospholipid antibody syndrome or known positive for lupus anticoagulant
• History of disseminated intravascular coagulation, hemolytic uremic syndrome, or thrombotic thrombocytopenic purpura
• History of venous thromboembolism or thrombotic events
• Previous use of romiplostim or previous use of eltrombopag within 4 weeks of enrollment
• Previous use of pegylated recombinant human megakaryocyte growth and development factor (PEG-rHuMGDF), recombinant human thrombopoietin (rHuTPO) or any other platelet producing agent
• Rituximab (for any indication) or 6-mercaptopurine within 8 weeks of enrollment, or anticipated use at any time during the study
• Splenectomy within 4 weeks of the screening visit
• Alkylating agents within 8 weeks before the screening visit or anticipated use during the time of the proposed study
• Vaccinations known to decrease platelet counts within 8 weeks before the screening visit
• Currently enrolled in another investigational device or drug study, or less than 30 days since ending investigational study
• Will have investigational procedures while enrolled on study
• Female subject of child bearing potential (defined as having first menses) not willing to use, in combination with her partner highly effective methods of birth control during treatment and for 1 month after the end of treatment
• Subject is pregnant or breast feeding, or might become pregnant within 1 month after the end of treatment
• Subject has known hypersensitivity to any recombinant Escherichia coli derived product (eg, Infergen®, Neupogen®, somatropin, and Actimmune®)
• Has previously enrolled into this study
• Will not be available for protocol-required study visits or procedures, to the best of the subject's and investigator's knowledge
• Any kind of disorder that, may compromise the subject to give written informed consent and/or to comply with all required study procedures