E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
preterm neonates with Respiratory Distress Syndrome (RDS) |
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E.1.1.1 | Medical condition in easily understood language |
Too early born babys with breathing problems |
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E.1.1.2 | Therapeutic area | Body processes [G] - Circulatory and Respiratory Physiological Phenomena [G09] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 16.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10038690 |
E.1.2 | Term | Respiratory distress syndrome (neonatal) |
E.1.2 | System Organ Class | 10038738 - Respiratory, thoracic and mediastinal disorders |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 16.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10028974 |
E.1.2 | Term | Neonatal respiratory distress syndrome |
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 |
The primary aim of this study is to evaluate the safety and tolerability of intratracheal administration of two different single doses of CHF 5633 in preterm neonates with RDS, in terms of adverse events (AEs) and adverse drug reactions (ADRs), haematology and biochemistry values and incidence of major neonatal morbidities and mortality. |
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E.2.2 | Secondary objectives of the trial |
To explore the effect of the drug on oxygenation and ventilatory requirements and need for rescue surfactant treatment.
To evaluate the extent of systemic exposure to SP-B analogue (CHF 5736.03) and SP-C analogue (CHF 4902.03) contained in CHF 5633.
To assess immunogenicity through the measurement of antibodies to SP-B analogue (CHF 5736.03) and SP-C analogue (CHF 4902.03) contained in CHF 5633. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Written informed consent obtained by parents/legal representative (according to local regulation) prior to any study-related procedures
2. Inborn and outborn, preterm neonates of either sex with a gestational age of 27 weeks up to 33 weeks+6
3. Clinical and radiological findings typical of RDS
4. Test treatment administration within 48 hours from birth
5. Requirement for endotracheal surfactant administration
6. Fraction of inspired oxygen (FiO2) ≥0.35 to maintain SpO2 between 90–95 %
7. Documentation of normal cranial ultrasound scan.
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E.4 | Principal exclusion criteria |
1. Use of surfactant prior to study entry and need for intratracheal administration of any other treatment (e.g. nitric oxide)
2. Known genetic or chromosomal disorders, major congenital anomalies (cardiac malformations, myelomeningocele etc)
3. Maternal drug abuse (heroin, methadone, methamphetamine, or cocaine) or significant alcohol consumption during pregnancy
4. Strong suspicion of congenital pneumonia/infection, sepsis
5. Evidence of severe birth asphyxia or a 5 minutes Apgar score ≤3
6. Presence of air leaks prior to study entry
7. Neonatal seizures prior to study entry
8. Mothers with prolonged rupture of the membranes (> 3 weeks duration)
9. Any condition that, in the opinion of the Investigator, would place the neonate at undue risk
10. Participation in another clinical trial of any placebo, drug or biological substance conducted under the provisions of a protocol.
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E.5 End points |
E.5.1 | Primary end point(s) |
Due to the nature of this study design an end point is not defined; please refer to the study objectives (E.2) |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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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 | No |
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 | Yes |
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) | Yes |
E.7.1.1 | First administration to humans | Yes |
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) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
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 | No |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 1 |
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 | 4 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 12 |
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
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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 | 2 |
E.8.9.1 | In the Member State concerned months | 8 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 8 |
E.8.9.2 | In all countries concerned by the trial days | 0 |