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

• Status: Completed
• Trial end date: 2019-08-08
• Target enrollment: 0
• Participant gender: All

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 platelet count ≥ 50 x 10^9/L in the absence of ITP rescue medications for the past 4 weeks.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Amgen

Criteria

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