E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Thrombocytopenia in pediatric subjects with ITP---------Sujetos Pediátricos Trombocitopénicos con Púrpura Trombocitopénica Inmune (Idiopática) (PTI) |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 12.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10043558 |
E.1.2 | Term | Thrombocytopenia purpura |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
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). |
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E.2.2 | Secondary objectives of the trial |
This study will also evaluate the long-term platelet response to romiplostim as well as the possible reductions in the dose of concurrent ITP therapies while receiving romiplostim. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Ethical - Subject or subject's legally acceptable representative has provided informed consent. Demographic - Subject is less than 18 years old at time of screening. Disease-Related - Subject completed a romiplostim study for the treatment of thrombocytopenia in pediatric subjects with ITP. |
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E.4 | Principal exclusion criteria |
Disease Related - 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. Medications - Subject received any alkylating agents within four weeks before the screening visit or anticipated use during the time of the proposed study. General - 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). - Subject (male or female) is not willing to use highly effective contraception during treatment if and when sexually active and for 4 weeks (women) or 14 weeks (men) after the end of treatment. - Subject is pregnant or breast feeding, or planning to become pregnant within 4 weeks after the end of treatment. - Male subject with a pregnant partner who is not willing to use a condom if and when sexually active during treatment and for 14 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 (this will depend on the type of 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. |
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E.5 End points |
E.5.1 | 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. |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | Information not present in EudraCT |
E.7.1.2 | Bioequivalence study | Information not present in EudraCT |
E.7.1.3 | Other | Information not present in EudraCT |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | Yes |
E.8.1.7.1 | Other trial design description |
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E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
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E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
| |
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
E.8.9.1 | In the Member State concerned months | 11 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 11 |
E.8.9.2 | In all countries concerned by the trial days | 0 |