E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Prevention of Staphylococcal Sepsis in Very Low Birth Weight (VLBW) Neonates |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10040049 |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10056430 |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
The primary objectives of the study are to confirm the safety of Pagibaximab Solution for Infusion in VLBW infants and to determine the efficacy of the drug in reducing the incidence of staphylococcal sepsis by study day 35 (in relation to placebo) in the modified intent-to-treat population (defined as all subjects who receive at least one dose of Pagibaximab Solution for Infusion or placebo). Staphylococcal sepsis will be diagnosed by new onset of two or more clinical signs of sepsis and either one positive peripheral blood culture (non-catheter) for S. aureus or two positive peripheral blood cultures for CoNS as defined in the protocol |
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E.2.2 | Secondary objectives of the trial |
 Reduction in the initial secondary endpoint defined by: a combination of the primary endpoint plus staphylococcal infections that do not meet the primary endpoint but do meet the criteria of one normally sterile body fluid (non-blood) culture that is positive for the S. aureus or two normally sterile body fluid cultures that are positive for CoNS obtained within 24 hours of each other. One or both of these positive cultures for CoNS may be from samples obtained from non-blood sterile sites for this endpoint.  Reduction in staphylococcal sepsis in the subgroup of infants weighing 600-900 grams at birth  Reduction in sepsis caused by CoNS or by S. aureus alone  Reduction in number of subjects with sepsis caused by any organism  Reduction in the number of subjects with positive blood cultures due to organisms other than staphylococci  Reduction in all-cause mortality |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
In-patient at a Neonatal Intensive Care Unit (NICU) - Informed consent obtained from the legally authorized representative(s) - Less than 48 hours after birth at the time of initiation of first infusion - Birth weight of 600 grams to 1200 grams - Estimated gestation age ≤33 weeks as determined by the neonatologist - Existing intravenous access at the time of the first infusion - Infants from multiple births up to triplets may be included, but each infant must meet the inclusion/exclusion criteria and each infant will be individually randomized to a treatment group |
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E.4 | Principal exclusion criteria |
- Proven staphylococcal sepsis as per this protocol prior to randomization - Concomitant infection or other medical condition, whose participation, in the opinion of the Investigator and/or medical advisor, may create an unacceptable additional risk - Currently receiving, recently received, or planned to receive other agents (e.g., investigational or IVIG) that could interfere with the conduct, results or interpretation of this study - Severe congenital or chromosomal anomaly that would limit life expectancy or require corrective measures during the period of this study - Uncontrolled seizures |
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E.5 End points |
E.5.1 | Primary end point(s) |
Incidence of staphylococcal sepsis between study days 0-35, as defined by: At least two clinical signs of sepsis, AND EITHER - One positive peripheral blood culture (non-catheter) for S. aureus, OR - Two positive peripheral blood cultures for CoNS obtained within 24 hours |
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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 | Yes |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | Yes |
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 | Yes |
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.8.3 |
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 | 8 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 20 |
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 | Information not present in EudraCT |
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 |
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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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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 | |
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 | 6 |