E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Enzyme replacement therapy in pre-term infants. Reduced fat uptake capabilities due to prematurity of pre-term infants |
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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 |
The primary objective of this study is to compare the fat absorption (coefficient of fat absorption) in preterm infants following treatment with rhBSSL to that with placebo when administered in pasteurized breast milk. |
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E.2.2 | Secondary objectives of the trial |
• Compare the length and body weight in preterm infants following treatment with rhBSSL to that in placebo when administered in pasteurized breast milk. • Study the safety of rhBSSL when administered in pasteurized breast milk.
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Preterm infants born before week 32 of gestation and who are less or equal than 32 weeks of gestation (extrapolated age) at the time of enrollment 2. Preterm infants appropriate for gestational age (each site should use its own growth curves or procedures and keep a copy of those used in the investigator’s file. The same growth curve should be used for all patients at one site) 3. Preterm infants receiving pasteurized breast milk 4. Preterm infants, enterally fed via nasal tube or bottle |
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E.4 | Principal exclusion criteria |
1. Infants receiving parenteral nutrition (except glucose) 2. Infants receiving milk fortifiers other than Eoprotin® (eg, Enfamil, Nutriprem) a. Otherwise eligible infants who are receiving milk fortifiers other than Eoprotin® may be enrolled if the use of fortifiers is discontinued 2 days before the first dose 3. Infants requiring mechanical ventilation 4. Infants small for their gestational age (SGA) 5. Infants requiring ≥30% O2 6. Infants receiving phototherapy (babies who have completed phototherapy and otherwise qualify for the study may be admitted) 7. Infants with severe brain disease including grade III or IV periventricular or intra ventricular hemorrhage, meningitis or hydrocephalus, intracranial hemorrhage of grade III or IV, periventricular leukomalacia 8. Major dysmorphology or congenital abnormalities that can affect growth and development 9. Infants with hemodynamically significant persistent ductus arteriosus (PDA) 10. Clinical evidence of sepsis (including low or high white cell count and/or low platelet count, and bacteriologically proven evidence of systemic infection) 11. Documented congenital infection (eg CMV) 12. Presence of necrotizing enterocolitis 13. Haemorrhagic pulmonary events 14. Prior or concomitant treatment with corticosteroids, except hydrocortisone 15. Any condition which in the opinion of the investigator makes the patient unsuitable for inclusion 16. Enrollment in another concurrent clinical study within 2 days of the screening visit through the completion of the follow-up visit
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E.5 End points |
E.5.1 | Primary end point(s) |
Coefficient of fat absorption (CFA) measured in stool collected for a 72-hour period during the final 3 days of each treatment period |
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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 | No |
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 | 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) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | Information not present in EudraCT |
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 | No |
E.8.1.6 | Cross over | Yes |
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
| 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 | 5 |
E.8.5 | The trial involves multiple Member States | No |
E.8.5.1 | Number of sites anticipated in the EEA | 5 |
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
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It is provided in the protocol |
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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 | 1 |
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 | 1 |
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 |