Status:
COMPLETED
Long-term Study of Romiplostim in Thrombocytopenic Pediatric Patients With Immune Thrombocytopenia (ITP)
Lead Sponsor:
Amgen
Conditions:
Immune Thrombocytopenia
Eligibility:
All Genders
1-18 years
Phase:
PHASE3
Brief Summary
This is a phase 3b single arm, open label, multicenter study describing the percentage of time pediatric participants with ITP have a platelet response while receiving romiplostim, defined as a platel...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
December 10 2014
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
August 8 2019
Estimated Enrollment :
203 Patients enrolled
Trial Details
Trial ID
NCT02279173
Start Date
December 10 2014
End Date
August 8 2019
Last Update
June 23 2022
Active Locations (78)
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1
Research Site
Roseville, California, United States, 95661
2
Research Site
Atlanta, Georgia, United States, 30322
3
Research Site
Chicago, Illinois, United States, 60611
4
Research Site
Peoria, Illinois, United States, 61615