Efficacy and Safety Study of Romiplostim in Paediatrics with Immune Thrombocytopenia

• Conditions: Immune Thrombocytopenia (ITP) in Paediatric Subjects; MedDRA version: 17.1Level: LLTClassification code 10043558Term: Thrombocytopenia purpuraSystem Organ Class: 100000004851; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2011-005019-96-Outside-EU/EEA
• Lead Sponsor: Amgen Inc

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2014-09-24
• Last Update Posted: 6 January 2015

Recruitment & Eligibility

• Status: A
• Sex: All
• Target Recruitment: 200

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 < 18 years at the time of providing informed consent
• Subject must be refractory to a prior ITP therapy, having relapsed after at least 1 prior ITP therapy, or be ineligible for other ITP therapies
• Examples of prior therapy include but are not limited to:
corticosteroids, IVIG, anti-D immunoglobulin, and platelet transfusions.
Subjects who have failed a splenectomy are eligible for study participation
• Subject has a documented platelet count = 30 x10 (to the)9/L or is experiencing bleeding that is uncontrolled with conventional therapies
• Subject's legally acceptable representative (or subject, if applicable) has provided informed consent before any study-specific procedure; and subject has proved assent, where required by the IRB/IEC
• Adequate hematologic, renal, and liver function during the screening period:
-Hemoglobin > 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
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified