E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated | Respiratory Distress Syndome in Premature Babies | |
E.1.1.1 | Medical condition in easily understood language | Premature baby immature lung disease. | |
E.1.1.2 | Therapeutic area | Diseases [C] - Respiratory Tract Diseases [C08] |
MedDRA Classification |
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 evaluate in a randomised controlled trial the efficacy of surfactant delivery via a minimally invasive technique in preterm infants 25-28 weeks gestation with RDS treated with CPAP. | |
E.2.2 | Secondary objectives of the trial | To evaluate that early surfactant administration via a minimallyinvasive technique to preterm infants on CPAP will result in a lesser duration of mechanical respiratory support, and a higher incidence of survival without bronchopulmonary dysplasia. | |
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria | 1. Requiring CPAP or nasal IPPV because of respiratory distress. 2. CPAP pressure of 5-8 cm H2O and FiO2 ≥0.30. 3. Less than 6 hours of age. 4. Agreement of the Treating Physician in charge of the infant's care. 5. Signed parental consent. | |
E.4 | Principal exclusion criteria | 1. Previously intubated, or in imminent need of intubation because of respiratory distress, apnoea or persistent acidosis. 2. Congenital anomaly or condition that might adversely affect breathing. 3. Identifiable alternative cause for respiratory distress (e.g. congenital pneumonia or pulmonary hypoplasia). 4. Lack of availability of an OPTIMIST treatment team. | |
E.5 End points |
E.5.1 | Primary end point(s) | Incidence of composite outcome of death or physiological BPD | |
E.5.1.1 | Timepoint(s) of evaluation of this end point | |
E.5.2 | Secondary end point(s) | • Physiological BPD • Clinical BPD (requirement for oxygen or any form of positive pressure support at 36 weeks corrected gestation). • Mild/moderate/severe BPD • Death • Death or BPD (clinical definition) • Intraventricular haemorrhage (IVH) (all grades) • IVH grades III and IV • Periventricular leukomalacia • Retinopathy of prematurity > stage II • Major morbidity (any of IVH grade III or IV, periventricular leukomalacia, ROP >stage II, physiological BPD) • Death or major morbidity • NEC (Modified Bell stage 2 or greater) • NEC or spontaneous intestinal perforation requiring surgery • Requirement for intubation in the first 72 hours of life • Requirement for intubation at any time • Need for additional surfactant therapy • Overall number of surfactant doses (including that given by MIST) • Duration of intubation (all episodes) • Duration of CPAP/NIPPV (all episodes) • Duration of intubation and CPAP • Duration of high flow nasal cannula (HFNC), minimum flow rate 2 L/min • Duration of respiratory support • Duration of oxygen therapy • Requirement for oxygen at home • Length of stay in intensive care • Length of hospital stay • Total hospital billings • Calculated cost of hospitalisation • Pneumothorax requiring drainage • Pulmonary haemorrhage • Patent ductus arteriosus (PDA) requiring anti-prostaglandin therapy • PDA requiring ligation • Late onset sepsis (positive bacterial or fungal culture from a normally sterile site) • Time to regain birth weight • Incidence of successful surfactant administration via MIST • Number of catheterisation attempts • Duration of bradycardia and hypoxaemia • Requirement for, and duration of, positive pressure ventilation by mask • Incidence of apparent discomfort | |
E.5.2.1 | Timepoint(s) of evaluation of this end point | Sischarge of baby from the hospital. | |
E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | Yes |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | No |
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) | No |
E.7.4 | Therapeutic use (Phase IV) | Yes |
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 | Yes |
E.8.1.7.1 | Other trial design description | A sham procedure will be performed. | |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | Yes |
E.8.2.3.1 | Comparator description | A sham procedure will be performed. | |
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 | 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 | No |
E.8.7 | Trial has a data monitoring committee | No |
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 | |
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 6 |