E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Immune Thrombocytopenia (ITP) in Paediatric Subjects |
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E.1.1.1 | Medical condition in easily understood language |
Bleeding disorder - ITP is a condition that may cause easy bruising or bleeding due to an abnormally low number of platelets in the blood. |
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E.1.1.2 | Therapeutic area | Diseases [C] - Blood and lymphatic diseases [C15] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 17.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10043558 |
E.1.2 | Term | Thrombocytopenia purpura |
E.1.2 | System Organ Class | 100000004851 |
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E.1.3 | Condition being studied is a rare disease | No |
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 |
The secondary objectives of the study are:
• the long-term platelet response to romiplostim
• 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 |
• 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. |
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E.4 | Principal exclusion criteria |
• 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. |
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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.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
The secondary endpoints are:
• Platelet response to romiplostim defined as platelet counts ≥ 50 x 109/L in the absence of rescue medication.
• The prevalence of concurrent ITP therapy use over time (corticosteroids, danazol, or azathioprine). |
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E.5.2.1 | 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. |
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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 | Yes |
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 | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
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 | No |
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 | No |
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 |
Will this trial be conducted at a single site globally?
| No |
E.8.4 | Will this trial be conducted at multiple sites globally? | Yes |
E.8.6 Trial involving sites outside the EEA |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | Specify the countries outside of the EEA in which trial sites are planned |
Australia |
Canada |
United States |
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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
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All subjects will be required to complete an EOS visit 1 week after the last dose of romiplostim. The end of the study is defined as the final subject’s EOS visit. |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.2 | In all countries concerned by the trial years | 7 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |