Efficacy and Safety Study of Romiplostim in Paediatrics with Immune Thrombocytopenia
- Conditions
- Immune Thrombocytopenia (ITP) in Paediatric SubjectsMedDRA version: 17.0Level: LLTClassification code 10043558Term: Thrombocytopenia purpuraSystem Organ Class: 100000004851Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
- Registration Number
- EUCTR2010-018426-39-Outside-EU/EEA
- Lead Sponsor
- Amgen Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- A
- Sex
- All
- Target Recruitment
- 60
• 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 109/L with neither count > 35 x 109/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
Are the trial subjects under 18? yes
Number of subjects for this age range: 60
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
•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)
•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 any other investigational procedures performed while enrolled in this clinical study
•Subject is pregnant or breast feeding, or planning to become pregnant within
1 month after the end of treatment
•Female subject of child bearing potential is not willing to use, in combination with her partner, 2 forms highly effective contraception during treatment and for 1 month after the end of treatment
•Subject has known sensitivity to any of the products to be administered during dosing
•Subject has previously enrolled into a prior Amgen romiplostim study
•Subject will not be available for protocol-required study visits, 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
- Primary Outcome Measures
Name Time Method Primary end point(s): The primary endpoint is the incidence of durable platelet response. A subject with durable platelet response is defined as achieving at least 6 weekly platelet counts of = 50 x 109/L during weeks 18 through 25 of treatment.;Timepoint(s) of evaluation of this end point: From week 18;Main Objective: The primary objective is to evaluate the efficacy of romiplostim in the treatment of thrombocytopenia in pediatric subjects with immune thrombocytopenia (ITP) as<br>measured by durable platelet response.;Secondary Objective: The secondary objectives are:<br>• To evaluate overall and weekly platelet response<br>• To evaluate the use of rescue ITP medications<br>• To evaluate a combined bleeding event and rescue medication use endpoint (composite bleeding episodes)<br>• To evaluate the overall safety of romiplostim
- Secondary Outcome Measures
Name Time Method Timepoint(s) of evaluation of this end point: From week 0;Secondary end point(s): • Subject incidence of overall platelet response. Overall responders are defined as subjects who achieve a platelet count = 50 x 109/L at a minimum of 4 times during weeks 2 to 25 of the treatment period<br>• Number of weekly platelet counts = 50 x 109/L during weeks 2 to 25 of the<br>treatment period<br>• Subject incidence of rescue ITP medications used<br>• The number of composite bleeding episodes. The composite bleeding episode is defined as clinically significant bleeding events or the use of a rescue medication to prevent a clinical significant bleeding event during weeks 2 to 25 of the<br>treatment period. A clinically significant event is defined as a CTCAE version 3.0 grade = 2 bleeding event<br>• Incidence of adverse events, including thromboembolic events and hematologic malignancies, clinically significant changes in laboratory values and the incidence of antibody formation