E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
High risk of bronchopulmonary dysplasia in very preterm neonates |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 12.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10006475 |
E.1.2 | Term | Bronchopulmonary dysplasia |
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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 whether inhalation of budesonide within 12 hours of life improves survival and precludes bronchopulmonary dysplasia (BPD) at 36 weeks gestational age (GA) in infants born between 23 and 27 weeks GA. |
|
E.2.2 | Secondary objectives of the trial |
To determine whether prophylactic inhalation of budesonide affects neurodevelopment at a corrected age of 18-22 months in preterm infants; to determine whether inhalation of corticosteroids is associated with adverse treatment effects, alters mortality at 36 weeks GA, BPD incidence at 36 weeks GA, and the duration of positive pressure respiratory support or supplemental oxygen. The severity of BPD will be determined according to three different definitions in an exploratory analysis. Data on the pharmacokinetics and pharmacogenetics of inhaled corticosteroids will be collected and a substudy on the genetic predisposition to BPD will be performed in conjunction with the controlled clinical study. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Entry criteria involve a gestational age of 23 0/7- 27 6/7 weeks, a postnatal age < 12 hours, the necessity for any form of positive pressure support (mechanical or nasal ventilation or CPAP), singleton or second born in case of multiple pregnancy and parental consent for participation. |
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E.4 | Principal exclusion criteria |
Exclusion criteria involve 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). |
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary outcome is defined as a combination of BPD or death at 36 weeks gestational age. The primary outcome comprises two highly patient-relevant components (causing mortality or BPD). The biological rationale suggests that both outcomes tend towards the same direction. The individual components of the primary outcome will also be considered as secondary outcomes, and the results will be presented together with the primary composite outcome. |
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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 | Yes |
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 | Yes |
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.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
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 | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
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
|
One formal interim analysis for efficacy is planned. The results will be reported directly to the members of the independent DMC. In the event that the null hypothesis of equal proportions of primary endpoints in the two groups is rejected, the DMC will decide about whether or not to advise the SC to discontinue the study. |
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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 | 3 |
E.8.9.1 | In the Member State concerned months | 2 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 3 |
E.8.9.2 | In all countries concerned by the trial months | 2 |
E.8.9.2 | In all countries concerned by the trial days | 0 |