E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
prevention of chronic lung disease in pre-term infants ( gestational age < 29 weeks) with respiratory distress. |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 7.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10054933 |
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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 assess the safety and efficacy of inhaled nitric oxide to reduce the risk of chronic lung disease in pre-term infants with respiratory distress and to assess the long term effects of the therapy on the development of these children over 7 years of clinical follow up. |
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E.2.2 | Secondary objectives of the trial |
-Number of days of assisted ventilation. -Length of hospitalization. -Total number of days in-hospital from 36 weeks GA to one year and two years corrected age. - Number of days in-hospital for respiratory illness from 36 weeks GA to one year and two years corrected age.
GA- gestational age |
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E.2.3 | Trial contains a sub-study | Information not present in EudraCT |
E.3 | Principal inclusion criteria |
-Inborn preterm infants 24+0 weeks–28+6 weeks gestational age (defined by first ultrasound at 16-18 weeks or if not available based on the last menstrual period) who requires the use of surfactant within 12 hours of birth, (either prophylactically or for signs of developing respiratory distress), or who requires the use of CPAP (FiO2 ≥0.30 and mean airway pressure ≥4cm H2O) within 12 hours of birth in order to maintain an SpO2 ≥85%.
-Informed consent of the parent or legal guardian.
CPAP- Continuous Positive Airway Pressure SpO2- Oxygen Saturation by pulse oximeter |
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E.4 | Principal exclusion criteria |
- Outborn infants. - Infants ≥ 29 weeks gestational age. - Infants with birth weight <500 grams. - Infants requiring FiO2 >0.5 to maintain SpO2 >85%, on a sufficient mean airway pressure (e.g. > 8 cm H2O on CMV ) in order to achieve adequate chest inflation (8-9 ribs on Chest X-ray) two hours after the proper administration of exogenous surfactant. - Any suspected congenital heart disease other than patent ductus arteriosus or atrial septal defect.
- Any infant with suspected lung hypoplasia associated with congenital diaphragmatic hernia
- Any infant with severe bleeding or coagulation abnormalities at high-risk of diathesis, e.g., platelet <50,000/mm3, fibrinogen <0.5 g/L, other clotting factors <10%.
- Any infant in whom a decision has been made not to provide full treatment, e.g., chromosomal abnormalities, severe multiple abnormalities, severe birth asphyxia, etc. - Use of another investigational drug or device before or during the active study period
CMV- Controlled Mechanical Ventillation
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary efficacy variable for this study will be a binary endpoint determined by assessment at 36 week post-menstrual age (PMA). An infant who is alive without CLD at 36 PCA will be counted as a ‘success’. An infant who has died, or who has CLD at 36 weeks PCA will be counted as a ‘failure’.
CLD is defined as the need for supplemental oxygen at 36 weeks post-menstrual age (based on a physiologic assessment). |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | Information not present in EudraCT |
E.6.2 | Prophylaxis | Information not present in EudraCT |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Information not present in EudraCT |
E.6.5 | Efficacy | Information not present in EudraCT |
E.6.6 | Pharmacokinetic | Information not present in EudraCT |
E.6.7 | Pharmacodynamic | Information not present in EudraCT |
E.6.8 | Bioequivalence | Information not present in EudraCT |
E.6.9 | Dose response | Information not present in EudraCT |
E.6.10 | Pharmacogenetic | Information not present in EudraCT |
E.6.11 | Pharmacogenomic | Information not present in EudraCT |
E.6.12 | Pharmacoeconomic | Information not present in EudraCT |
E.6.13 | Others | Information not present in EudraCT |
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) | Information not present in EudraCT |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
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 | Information not present in EudraCT |
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.8.2.3.1 | Comparator description |
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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 | 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 | 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
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Study to include 7 years of clinical follow up in order to assess the long term effects on the development of the infant |
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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 | 9 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 9 |
E.8.9.2 | In all countries concerned by the trial months | 0 |