E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Neonates or infants (age less than or equal to 92 days at the time of randomization) with systemic to pulmonary arterial shunt for palliation of cyanotic congenital heart disease. |
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E.1.1.1 | Medical condition in easily understood language |
Neonates or infants (age less than or equal to 92 days at the time of randomization) with systemic to pulmonary arterial shunt for palliation of cyanotic congenital heart disease. |
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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 | 16.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10019273 |
E.1.2 | Term | Heart disease congenital |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic 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 |
The primary objective of the study is to evaluate the efficacy of Clopidogrel 0.2 mg/kg/day for the reduction of all cause mortality and shunt related morbidity in neonates or infants with cyanotic congenital heart disease palliated with a systemic-to-pulmonary artery shunt (e.g. modified Blalock Taussig Shunt [BTS]).
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E.2.2 | Secondary objectives of the trial |
The secondary objective was to assess the safety of Clopidogrel in the study population.
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E.2.3 | Trial contains a sub-study | Yes |
E.2.3.1 | Full title, date and version of each sub-study and their related objectives |
Sub-study n°1
Full title : Pharmacokinetic Substudy
Date : 2006/08/11
Version : 2.0
Sub-study n°1 objectives : The description of SR26334 plasma concentrations in the targeted population and the possible relationship with covariates such as age, weight, and gender.
Sub-study n°2
Full title : Pharmacodynamic Substudy
Date : 2006/08/11
Version : 2.0
Sub-study n°2 objectives : The description of the platelet aggregation inhibition in this population. |
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E.3 | Principal inclusion criteria |
•Cyanotic congenital heart disease treated by any palliative systemic-to-pulmonary artery shunt.
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E.4 | Principal exclusion criteria |
•Active bleeding or increase risk of bleeding,
•Allergy to 2 or more classes of drug,
•Unable to receive drug orally or enterically,
•Current clinically significant or persistent thrombocytopenia, neutropenia, severe hepatic or renal failure.
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E.5 End points |
E.5.1 | Primary end point(s) |
•Number of Participants Reaching Primary Endpoint Criteria (First Occurrence of Death / Shunt Thrombosis / Cardiac Procedure < 120 Days Considered of Thrombotic Nature)
The primary endpoint was the first occurence of any of the following events: Death (including heart transplant); Shunt thrombosis requiring intervention; Hospitalization for bi-directional Glenn procedure or any cardiac related intervention prior to 120 days of age following an event or a shunt narrowing considered to be of thrombotic nature by the blinded adjudication committee.
Only the first event was counted.
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
•Number of Participants Reaching Primary Endpoint Criteria (First Occurrence of Death / Shunt Thrombosis / Cardiac Procedure < 120 Days Considered of Thrombotic Nature) [ Time Frame: Median follow-up of 5.8 months (up to a maximum of 12 months after randomization) ]
The primary endpoint was the first occurence of any of the following events: Death (including heart transplant); Shunt thrombosis requiring intervention; Hospitalization for bi-directional Glenn procedure or any cardiac related intervention prior to 120 days of age following an event or a shunt narrowing considered to be of thrombotic nature by the blinded adjudication committee.
Only the first event was counted.
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E.5.2 | Secondary end point(s) |
•Number of Participants With Bleeding Events [ Time Frame: From randomization up to 28 days after treatment discontinuation or final follow-up visit, whichever comes first ]
Bleeding events spanning from signature of the Informed Consent Form up to the last visit were collected as for any Adverse Event.
The 'on-treatment' period was defined as the period from randomization up until 28 days after treatment discontinuation or final follow-up visit, whichever came first, and participants who experienced bleeding events during that period were counted.
•Number of Participants According to Bleeding Type/Etiology [ Time Frame: From randomization up to 28 days after treatment discontinuation or final follow-up visit, whichever comes first ]
For all reported bleeding events, the type and the etiology of the bleeding event were collected. Participants who experienced bleeding events during the 'on-treatment period' were counted by bleeding type and etiology. Participants who had multiple bleedings could be counted several times.
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
•Number of Participants With Bleeding Events [ Time Frame: From randomization up to 28 days after treatment discontinuation or final follow-up visit, whichever comes first ]
•Number of Participants According to Bleeding Type/Etiology [ Time Frame: From randomization up to 28 days after treatment discontinuation or final follow-up visit, whichever comes first ]
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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 | 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 | 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.8.2.4 | Number of treatment arms in the trial | 2 |
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 |
China |
Norway |
Argentina |
Brazil |
Egypt |
Hong Kong |
India |
Korea, Republic of |
Malaysia |
Thailand |
Israel |
Mexico |
Russian Federation |
Singapore |
South Africa |
Taiwan |
United States |
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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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E.8.9 Initial estimate of the duration of the trial |
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 | 6 |