E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Neonates or infants/toddlers at risk of thrombosis |
|
MedDRA Classification |
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 |
Pharmacodynamic assessment. to determine the dose of clopidogrel to achieve a mean 30-50% inhibition of ADP-induced platelet aggregation in neonates or infants/toddlers at risk for thrombosis.
|
|
E.2.2 | Secondary objectives of the trial |
assess pharmacokinetics and safety of clopidogrel when administered to neonates and infants/toddlers at the doses tested for demonstration of an appropriate inhibitory effect on ADP-induced platelet aggregation. Additionally, in patients whose total body weight is more than 3 kg (or above 5 kg, as per investigator judgment), the level of inhibition of TRAP-induced platelet aggregation will be determined. |
|
E.2.3 | Trial contains a sub-study | Information not present in EudraCT |
E.3 | Principal inclusion criteria |
Patients less than or equal to 30 days old at the time of randomization should be included as a substantial part of the overall study population. Therefore, not only infants/toddlers aged up to 24 months but also neonates will be potentially eligible for this trial. All patients must have either a Blalock-Taussig shunt or any systemic to pulmonary artery shunt or have potential therapeutic benefit from clopidogrel because of a related pathological condition requiring antiplatelet therapy (such as but not limited to Kawasaki disease, vascular stent, Glenn shunt or Fontan physiology). |
|
E.4 | Principal exclusion criteria |
1. Ongoing bleeding or increased risk of bleeding, bleeding disorders (e.g. hemophilia, von Willebrand disease) or previous intracranial or life-threatening hemorrhage 2. Allergy to 2 or more classes of drug 3. Current or planned treatment with anticoagulants 4. Weight less than 2 kg; gestational age less than 35 weeks 5. Unable to receive study drug enterically 6. Previous platelet transfusion within the last 7 days 7. Concurrent use of another experimental drug 8. Thrombocytopenia (< 80,000 platelets per mm3 – this threshold is required for technical reason in order to allow pharmacodynamic assessment to be run appropriately) 9. Severe hepatic or renal failure (i.e., more than 2.5 times the upper limit for age of hepatic enzymes or creatininemia). |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Primary efficacy criteria will be percent inhibition of maximum extent and rate of aggregation of 5 µM ADP-induced platelet aggregation. In patients above 3kg body weight (or above 5 kg, as per investigator judgment), the percent inhibition of maximum extent and rate of aggregation of 5 µM TRAP-induced platelet aggregation will also be determined. |
|
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 | Yes |
E.6.10 | Pharmacogenetic | Information not present in EudraCT |
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) | Information not present in EudraCT |
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) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | Information not present in EudraCT |
E.7.4 | Therapeutic use (Phase IV) | Information not present in EudraCT |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
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 | Yes |
E.8.2.3 | Other | No |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.5 | The trial involves multiple Member States | Yes |
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 |
|
E.8.7 | Trial has a data monitoring committee | Information not present in EudraCT |
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 | |
E.8.9.1 | In the Member State concerned months | 8 |
E.8.9.1 | In the Member State concerned days | |
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 | 8 |