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
- EUCTR2009-016203-32-Outside-EU/EEA
- Lead Sponsor
- Amgen Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- A
- Sex
- All
- Target Recruitment
- 78
• Subject or subject’s legally acceptable representative has provided informed consent (and assent where required) prior to any study-specific procedure.
• Subject completed a romiplostim study for the treatment of thrombocytopenia in pediatric subjects with ITP.
Are the trial subjects under 18? yes
Number of subjects for this age range: 78
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
• Subject has or previously had any 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).
• Subject has any new active malignancy diagnosed since enrollment in the previous romiplostim ITP study.
• Subject received any alkylating agents within four weeks before the screening visit or anticipated use during the time of the proposed study.
• Other investigational medications are excluded.
• 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) (with the exception of romiplostim in a previous clinical study).
• Female subject of child bearing potential (defined as having first menses) is not willing to use highly effective contraception during treatment and for 4 weeks after the end of treatment.
• Female subject is pregnant or breast feeding, or planning to become pregnant within 4 weeks after the end of treatment.
• Subject has known sensitivity to any of the products to be administered during dosing.
• Subject previously has entered this study.
• Subject will not be available for protocol-required study visits, to the best of the subject 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 Main Objective: The primary objective of this study is to evaluate the safety of romiplostim as a long-term treatment in pediatric thrombocytopenic subjects with immune (idiopathic) thrombocytopenic purpura (ITP).;Secondary Objective: The secondary objectives of the study are:<br>• the long-term platelet response to romiplostim<br>• the possible reductions in the dose of concurrent ITP therapies while receiving romiplostim.;Primary end point(s): The primary endpoints are the subject incidence and exposure adjusted incidence of adverse events, including clinically significant changes in laboratory values and incidence of antibody formation.;Timepoint(s) of evaluation of this end point: Weekly during the study.
- Secondary Outcome Measures
Name Time Method Secondary end point(s): The secondary endpoints are:<br>• Platelet response to romiplostim defined as platelet counts = 50 x 109/L in the absence of rescue medication.<br>• The prevalence of concurrent ITP therapy use over time (corticosteroids, danazol, or azathioprine).;Timepoint(s) of evaluation of this end point: Weekly during the study, but at the point that a patient is receiving a stable dose of romiplostim, then platelet response will be evaluated every 8 weeks.