E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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E.1.1.1 | Medical condition in easily understood language |
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E.1.1.2 | Therapeutic area | Diseases [C] - Cardiovascular Diseases [C14] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10066899 |
E.1.2 | Term | Venous thromboembolism |
E.1.2 | System Organ Class | 10047065 - Vascular disorders |
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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 |
This study is exploratory in nature and will investigate safety and tolerability of an oral liquid formulation of dabigatran etexilate in pediatric patients 1 to < 12 years old treated for primary VTE. The study will also provide preliminary pharmacokinetic and pharmacodynamic data for this age group. |
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E.2.2 | Secondary objectives of the trial |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1) Males or females 1 to < 12 years of age
2) Objective diagnosis of VTE
3) End of planned treatment course with low molecular weight heparin (LMWH) or oral anticoagulant (OAC) for VTE with INR and aPTT both within normal limits.
4) Written informed consent provided by the patient’s parent (or legal guardian) and assent provided by the patient (if applicable) at the time of ICF signature. |
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E.4 | Principal exclusion criteria |
1) conditions associated with increased risk of bleeding
2) severe renal dysfunction or requirement for dialysis
3) active infective endocarditis
4) hepatic disease
5) anemia or thrombocytopenia
6) use of prohibited or restricted drug within previous week
7) pregnant females or females not using medically accepted contraceptive method
8) received investigational drug within past 30 days
9) unreliable patients or patients who have any condition that would not allow safe participation in study |
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E.5 End points |
E.5.1 | Primary end point(s) |
1) Incidence of all bleeding events,
2) Incidence of all adverse events.
3) Pharmacodynamic parameters: central and local measurement of TT
4) Pharmacokinetic parameters: plasma concentrations of total and free dabigatran, BIBR 1048 BS, BIBR 951BS, and BIBR 1087 SE, |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Endpoints 1) & 2) measured during screening, treatment period and 30 day follow-up.
Endpoints 3) & 4) measured during screening (baseline) and treatment period. |
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E.5.2 | Secondary end point(s) |
1) Change in laboratory and clinical parameters such as liver enzymes, ECG, and physical examination,
2) Occurrences of clinical outcomes including recurrent thrombosis, post thrombotic syndrome (PTS), pulmonary emboli (PE), and total and venous thrombotic event (VTE) related mortality,
3) Global assessment of tolerability to study medication (including patient taste assessment),
4) Pharmacodynamic parameters: local measurement of aPTT |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Endpoints 1) to 3) measured during screening, treatment period and 30 day follow-up.
Endpoint 4) measured during screening (baseline) and treatment period. |
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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 | No |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | Yes |
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) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | Information not present in EudraCT |
E.8.1.2 | Open | Information not present in EudraCT |
E.8.1.3 | Single blind | Information not present in EudraCT |
E.8.1.4 | Double blind | Information not present in EudraCT |
E.8.1.5 | Parallel group | Information not present in EudraCT |
E.8.1.6 | Cross over | Information not present in EudraCT |
E.8.1.7 | Other | Information not present in EudraCT |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
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 | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 1 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 12 |
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 | No |
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 |
Austria |
Belgium |
Canada |
Czech Republic |
France |
Hungary |
Italy |
Latvia |
Lithuania |
Netherlands |
Slovakia |
Spain |
Switzerland |
Thailand |
Ukraine |
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E.8.7 | Trial has a data monitoring committee | Yes |
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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Sixteen patients who have completed the 30 day post treatment followup period. |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 1 |
E.8.9.1 | In the Member State concerned months | 0 |
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 | 7 |
E.8.9.2 | In all countries concerned by the trial days | 0 |