Immune Thrombocytopenia (ITP) in Paediatric Subjects MedDRA version: 20.0 Level: LLT Classification code 10043558 Term: Thrombocytopenia purpura System Organ Class: 100000004851
Conditions
Interventions
Trade Name: Nplate
Product Name: Nplate
Product Code: AMG 531
Pharmaceutical Form: Powder for solution for injection
INN or Proposed INN: Nplate
CAS Number: 267639-76-9
Current Sponsor code: AMG 531
O
Sponsors
Amgen Inc
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • Diagnosis of primary ITP according to The American Society of Hematology (ASH) Guidelines (Neunert et al, 2011) at least 6 months before screening, regardless of splenectomy status • Age = 1 year and 10.0 g/dL - Serum creatinine = 1.5 times the upper limit of normal - Total serum bilirubin = 1.5 times the upper limit of normal - AST and ALT = 3.0 times the upper limit of normal Are the trial subjects under 18? yes Number of subjects for this age range: 200 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range 0 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range 0
Exclusion criteria
Exclusion criteria: 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) • Prior bone marrow transplant or peripheral blood progenitor cell transplant • Known active or prior malignancy except non-melanoma skin cancers within the last 5 years • Known history of myelodysplastic syndrome • Known history of bleeding diathesis • Known history of congenital thrombocytopenia • Known history of hepatitis B, hepatitis C or human immunodeficiency virus • Known history of systemic lupus erythematosus, Evans syndrome, or autoimmune neutropenia • Known history of antiphospholipid antibody syndrome or known positive for lupus anticoagulant • Known history of disseminated intravascular coagulation, hemolytic uremic syndrome, or thrombotic thrombocytopenic purpura • History of venous thromboembolism or thrombotic events • Previous use of romiplostim. Previous use of eltrombopag within 4 weeks of enrollment. • Previous use of 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 another investigational device or drug study(s), or receiving other investigational agent(s) • Subject will have investigational procedures performed while enrolled in this clinical study • Female subject of child bearing potential (defined as having first menses) is 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®) • Subject has previously enrolled into this study • Subject will not be available for protocol-required study visits or procedures, to the best of the subject’s and investigator’s knowledge • Subject has any kind of disorder that, in the opinion of the investigator, may compromise the ability of the subject to give written informed consent and/or to comply with all required study procedures
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective is to describe the percentage of time that pediatric subjects with immune thrombocytopenia (ITP) have a platelet response in the first 6 months from the start of treatment with romiplostim. ;Secondary Objective: The secondary objectives of the study are: • To describe the percentage of time that pediatric subjects with ITP have a platelet response over the study duration • To describe the percentage of time that pediatric subjects with ITP have an increase in platelet count = 20 x 10 (to the ) 9/L above baseline over the study duration • To describe the use of rescue ITP medications • To describe the incidence of antibody formation • To describe the safety of romiplostim as a long-term treatment in pediatric thrombocytopenic subjects with ITP ;Primary end point(s): The percentage of time with a platelet count of = 50 x 10 (to the) 9/L starting from week 2 in the first 6 months of the treatment period without rescue medication use within the past 4 weeks In addition, for protocol supplement: • Evaluation of bone marrow changes after Year 1 and Year 2 for the following: - Incidence of collagen as evidenced by trichrome staining (using the modified Bauermeister grading scale) after romiplostim exposure - Incidence of bone marrow reticulin increases in severity = 2 grades (ie, grade 0 to 2-4, 1 to 3-4, 2 to 4), compared to baseline, or an increase to grade 3 or grade 4 as evidenced by reticulin silver staining using the modified Bauermeister grading scale after romiplostim exposure - Incidence of bone marrow abnormalities (eg, myelodysplastic syndrome, monosomy 7) as evidenced by cytogenetics and fluorescence in situ hybridization;Timepoint(s) of evaluation of this end point: From week 2 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): The percentage of time with a platelet count of = 50 x 10 (to the) 9/L starting from week 2 until the end of the treatment period without rescue medication use within the past 4 weeks • The percentage of time with an increase in platelet count = 20 x 10 (to the) 9/L above baseline starting from week 2 until the end of the treatment period without rescue medication use in the past 4 weeks. • Subject incidence of rescue ITP medications used • The incidence of anti-romiplostim neutralizing antibodies and cross reactive antibodies to TPO at any time during the study • The incidence of adverse events, including clinically significant changes in laboratory values In addition, for protocol supplement: • The incidence of increased reticulin as evidenced by silver staining at Year 1 or Year 2, after exposure to romiplostim;Timepoint(s) of evaluation of this end point: From week 2 | — |
Countries
Australia, Belgium, Brazil, Canada, Czech Republic, France, Hungary, Israel, Mexico, Peru, Poland, Russian Federation, South Africa, Spain, Switzerland, Turkey, United Kingdom, United States
Contacts
Public ContactIHQ Medical Information - Clinical
Amgen (EUROPE) GmbH
Outcome results
None listed