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Clinical Trial VICCNCPED1225


PHIII Randomized, Double Blind, Placebo Controlled Study to Determine the Safety and Efficacy of Romiplostim in Thrombocytopenic Pediatric Subjects with Immune Thrombocytopenia (ITP)

Principal Investigator(s)

Richard Ho


  • Protocol No. VICCNCPED1225
  • Open Date: 05/24/2012
  • Staging: Phase III
  • Age Group: Children
  • Scope: National
  • Objective: To evaluate the efficacy of romiplostim in the treatment of thrombocytopenia in pediatric subjects with ITP as measured by durable platelet response
  • Disease Sites: None Specified
  • Therapies: Correlative
  • Drugs: None Specified
  • Participating Institutions: Vanderbilt University
  • National Clinical Trial ID: NCT01444417
  • Secondary Protocol No: 20080279


None Provided.


Ages Eligible for Study:1 Year to 17 Years
Genders Eligible for Study:Both
Accepts Healthy Volunteers:No


Inclusion Criteria:
• Diagnosis of primary ITP according to the ASH Guidelines at least 6 months prior to screening, regardless of splenectomy status
• Subject must be refractory to a prior ITP therapy, having relapsed after at least 1 prior ITP therapy, or ineligible for other ITP therapies; prior therapy includes first-line therapies
• Age ≥ 1 year and < 18 years at the time of providing informed consent
• The mean of 2 platelet counts taken during the screening period must be ≤ 30 x 10^9/L with neither count > 35 x 10^9/L
• A serum creatinine concentration ≤ 1.5 times the laboratory normal range (for each age category) during the screening period
• Adequate liver function; serum bilirubin ≤ 1.5 times the laboratory normal range during the screening period;AST and ALT ≤ 3.0 times the laboratory normal range during the screening period
• Hemoglobin > 10.0 g/dL during the screening period
• Subject and/or subject's legally acceptable representative has provided informed consent prior to any study-specific procedure; subject has provided assent, where required
Exclusion Criteria:
• Known 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 in the study
• Known active or prior malignancy except adequately treated basal cell carcinoma
• Known history of congenital thrombocytopenia
• Known history of hepatitis B, hepatitis C, or HIV
• Known history of H. pylori by urea breath test or stool antigen test within 6 months of enrollment or successfully treated with no evidence of infection
• Known history of systemic lupus erythematosus, evans syndrome, or autoimmune neutropenia
• Known history of antiphospholipid antibody syndrome or positive for lupus anticoagulant
• Known history of disseminated intravascular coagulation, hemolytic uremic syndrome, or thrombotic thrombocytopenic purpura
• Previous history of venous thromboembolism or thrombotic events
• Previous use of romiplostim, PEG-rHuMGDF, Eltrombopag, rHuTPO or any platelet producing agent
• Rituximab (for any indication) or 6-MP within 14 weeks before the screening visit, or anticipated use during the time of the proposed study
• Splenectomy within 4 weeks of the screening visit
• All hematopoietic growth factors including IL-11 (oprelvekin) within 4 weeks before 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
• Known hypersensitivity to any recombinant E coli-derived product (eg, Infergen, Neupogen, Somatropin, and Actimmune)
• Other criteria may apply