E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Survival of extremely Low Birth Weight (ELBW) infants has improved in recent decades but Bronchopulmonary Dysplasia (BPD) remains a major health care problem. BPD is a chronic lung disease that occurs in premature infants requiring mechanical ventilation and oxygen therapy, but also develops in preterm neonates who require little or no ventilatory or oxygen support |
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E.1.1.1 | Medical condition in easily understood language |
Prevention of chronic lung disease that mostly occurs in very preterm infants |
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E.1.1.2 | Therapeutic area | Diseases [C] - Respiratory Tract Diseases [C08] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10006475 |
E.1.2 | Term | Bronchopulmonary dysplasia |
E.1.2 | System Organ Class | 10038738 - Respiratory, thoracic and mediastinal 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 |
To determine if the early (within 12 hours of life) prophylactic use of inhaled corticosteroids (Budesonide) in very preterm infants (gestational age 23 0/7-27 6/7 weeks) requiring any form of positive pressure support (mechanical or nasal ventilation or continuous positive airway pressure (CPAP)) increases survival without bronchopulmonary dysplasia (BPD) at 36 weeks gestational age. |
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E.2.2 | Secondary objectives of the trial |
To determine whether early inhalation of corticosteroids for the prevention of BPD alters the incidence of infants born prematurely with neurodevelopmental impairment at a corrected age of 18 to 22 months.
To determine whether inhalation of corticosteroids is associated with adverse treatment effects alters mortality at 36 weeks gestational age, BPD incidence at 36 weeks gestational age, and the duration of positive pressure respiratory support or supplemental oxygen.
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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 |
1. BPD definitions substudy
Objective: To assess the prognostic impact of three different BPD definitions on pulmonary mortality and neurodevelopment Version: final (02.09.2008)
2. Genetic background of BPD substudy
Objective: To determine whether certain candidate genes are associated with the development of BPD Version: final (02.09.2008)
3. Pharmacokinetic / Pharmacodynamic / Pharamcogenetic substudy
Objective: To perform PK / PD analyses and to determine whether certain individual genetic aspects account for individualised drug metabolism Version: final (02.09.2008)
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E.3 | Principal inclusion criteria |
A gestational age of 23 0/7-27 6/7 weeks, a postnatal age < 12 hours, the requirement for any form of positive pressure support (mechanical or nasal ventilation or CPAP) and parental consent for participation. |
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E.4 | Principal exclusion criteria |
A clinical decision not to administer therapies (infant not considered viable), dysmorphic features or congenital malformations that adversely affect life expectancy or neurodevelopment and known or suspected congenital heart disease (not including a persistent ductus arteriosus and/or an atrial septum defect). The clinical assessment of dysmorphic
features, congenital malformations, suspected congenital heart disease and the decision to exclude an infant for the mentioned reasons will be left to the discretion of the attending physician.
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E.5 End points |
E.5.1 | Primary end point(s) |
The parameter for the confirmatory analysis of efficacy will be the incidence of primary endpoints. A primary endpoint is defined as the occurrence of any of the following events during the first 36 weeks gestational age: (1) Death for any reason until the end of week 36 gestational age; (2) BPD according to the physiological definition, diagnosed at 36 weeks +/- 1 day gestational age. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
(1) Death for any reason until the end of week 36 gestational age; (2) BPD according to the physiological definition, diagnosed at 36 weeks +/- 1 day gestational age. |
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E.5.2 | Secondary end point(s) |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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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 | Yes |
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 |
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.8.4.1 | Number of sites anticipated in Member State concerned | 5 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 30 |
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 |
Belgium |
Czech Republic |
Finland |
France |
Germany |
Greece |
Israel |
Italy |
Netherlands |
United Kingdom |
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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.8.9.1 | In the Member State concerned years | 5 |
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 | 5 |
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 |