E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
bronchopulmonary dysplasia |
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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 establish the efficacy of hydrocortisone given after one week of life to reduce the incidence of the combined outcome death or BPD in chronically ventilated preterm infants. |
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E.2.2 | Secondary objectives of the trial |
Secondary outcomes are short term effects on the pulmonary condition, adverse effects during hospitalization, and long-term neurodevelopmental sequelae assessed at 2 years corrected gestational age (CGA). |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Preterm infants with a gestational age < 30 wks and/or birth weight < 1250 g, ventilator dependent at 7-14 days PNA with a respiratory index (MAwP x FiO2) of ≥ 3.5. This index should be present for more than 12 h/day for at least 48 hours and must ensure normal oxygen saturation (86-94%) and pCO2 values in premature infants (5.0-7.0 kPa). |
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E.4 | Principal exclusion criteria |
Infants with chromosomal defects (e.g. trisomy 13, 18, 21) or major congenital malformations that are expected to compromise lung function (e.g. surfactant protein deficiencies, congenital diaphragmatic hernia) or result in chronic ventilation (e.g. Pierre Robin sequence), or increase the risk of death or adverse neurodevelopmental outcome (congenital cerebral malformations) will be excluded. |
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E.5 End points |
E.5.1 | Primary end point(s) |
the dichotomous variable BPD free survival at 36 weeks PMA. BPD at 36 weeks PMA will be assessed according to the NIHCHD Consensus Statement defining normal oxygen saturation as 86%-94%. The severity of the BPD will be assessed as proposed by Jobe et.al., since the severity of BPD has a high association with neurodevelopmental sequelae. In case of supplemental oxygen delivery >21% and < 30% or low flow at 36 weeks PMA, the oxygen reduction test as described by Walsh et.al. should be preformed. A positive oxygen reduction test has a high correlation with the risk on discharge home with oxygen, the length of hospital stay, and pulmonary morbidity requiring hospital readmission during the first year of life. |
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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 | 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.8.3 |
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 | No |
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
|
The last follow-up visit at 2 years CGA of the last subject. |
|
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 | 2 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 0 |
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 |