E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Late onset sepsis & necrotising enterocolitis |
|
E.1.1.1 | Medical condition in easily understood language |
Late onset sepsis & necrotising enterocolitis |
|
E.1.1.2 | Therapeutic area | Body processes [G] - Immune system processes [G12] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10053598 |
E.1.2 | Term | Late onset neonatal sepsis |
E.1.2 | System Organ Class | 100000004862 |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10028884 |
E.1.2 | Term | Necrotising enterocolitis |
E.1.2 | System Organ Class | 100000004856 |
|
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 |
1. Survival without any disability at 24 months corrected age |
|
E.2.2 | Secondary objectives of the trial |
2. Survival until discharge home and at follow-up 3. Any disability at 24 months 4. Disability by grade and type (motor, language, cognitive, etc) 5. Progression from Stage II to Stage IIIA/B NEC 6. Surgery for NEC 7. Duration of parenteral nutrition 8. Brain injury (intraventricular haemorrhage (IVH) or periventricular leukomalacia (PVL) 9. Duration of any mechanical ventilation 10. Chronic lung disease 11. Time to full enteral feeds 12. Retinopathy of prematurity 13. Length of hospital stay |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Infants <29 weeks gestation within 6 hours of blood culture taken for suspected LOS or NEC 2. Signed, written informed consent by a parent or a legal guardian |
|
E.4 | Principal exclusion criteria |
Presence of major congenital malformation(s) or chromosomal abnormalities or any other conditions considered likely to be incompatible with disability-free survival |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Primary outcome will be survival without any disability at 24 months corrected gestational age.
Survival a) All infants discharged home alive b) All infants alive at follow-up
Any disability at 24 months: Any disability assessed by the following assessments at 24 months corrected age a) Bayley-III and/or b) parent report on the Ages and Stages Questionnaire and/or c) a modified Short Health Status Questionnaire documenting either - major developmental delay, including language or speech problems, or - cerebral palsy with inability to walk unassisted at or after 2 years corrected age, or - severe visual loss (cannot fixate/ legally blind, or corrected acuity <6/60 in both eyes), or - deafness, requiring a hearing aid or cochlear implants |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
24 months of age corrected for gestation |
|
E.5.2 | Secondary end point(s) |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
|
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 | No |
E.6.5 | Efficacy | No |
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 | 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 | Yes |
E.8.2.3 | Other | No |
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
| 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 | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Australia |
Canada |
New Zealand |
Singapore |
Taiwan |
|
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
|
end of trial is completion of last recruited subject follow up visit. |
|
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 | 5 |
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 |