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 | 19.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10066899 |
E.1.2 | Term | Venous thromboembolism |
E.1.2 | System Organ Class | 100000004866 |
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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 Phase IIa study will provide single dose pharmacokinetic and pharmacodynamic data
and assess tolerability and safety of dabigatran etexilate in successive groups of pediatric
patients aged 2 to <12 years followed by 1 to <2 years using an oral liquid formulation. |
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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) Male or female children 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.
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) Weight less than 9 kg
2) Conditions associated with an increased risk of bleeding
3) Renal dysfunction or requirement for dialysis
4) Active infective endocarditis
5) Hepatic disease
6) pregnant females or females not using medically accepted contraceptive method
7) Anemia or thrombocytopenia
8) use of prohibited or restricted drug within previous week
9) received investigational drug within past 30 days |
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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 measurement of aPTT, ECT, and Factor IIa (FIIa) inhibition
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 followup.
Endpoints 3) & 4) measured during screening (baseline) and treatment period. |
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E.5.2 | Secondary end point(s) |
1) Changes in laboratory and clinical parameters such as liver enzymes and physical
examination
2) Global assessment of tolerability to study medication (including patient taste
assessment) |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Endpoint 1) is measured during screening, treatment period and 30 day followup.
Endpoint 2) is measured during 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 | No |
E.8.1.2 | Open | Yes |
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 |
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 |
Canada |
Russian Federation |
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.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 1 |
E.8.9.2 | In all countries concerned by the trial days | 0 |