A phase 3 clinical study investigating the effects of rhBSSL (an enzyme to help digestion of fat) added to pasteurized breast milk or formula in babies born prematurely.

Phase 1

Conditions: Prevention of growth retardation due to lack of bile salt-stimulated lipase in enteral nutrition
MedDRA version: 14.1Level: PTClassification code 10036590Term: Premature babySystem Organ Class: 10036585 - Pregnancy, puerperium and perinatal conditions
Therapeutic area: Body processes [G] - Digestive System and Oral Physiological Phenomena [G10]

Registration Number: EUCTR2010-023909-35-GB

Lead Sponsor: Swedish Orphan Biovitrum AB (publ)

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 432

Inclusion Criteria

1. Preterm infant born before Week 32 of gestation.
2. Preterm infant who is <33 weeks postmenstrual age at the time of randomization.
3. Preterm infant who is AGA or SGA at birth.
4. Preterm infant who is receiving food enterally (bottle or gavage tube) at a level of at least 100 mL/kg/day at randomization.
5. Preterm infant whose enteral feeding consists of only infant formula or only PBM at the time of inclusion, and who are expected to remain on only infant formula for 4 weeks, or only PBM for at least 2 weeks following treatment initiation.
6. Preterm infant who is expected not to receive any fresh breast milk for 4 weeks following treatment initiation.
7. Informed consent is obtained from the patient’s legally acceptable representative. Every effort will be made to ensure informed consent is obtained from both parents. Where this is not possible, the local regulations will be followed in the definition of legally acceptable representative.

Are the trial subjects under 18? yes
Number of subjects for this age range: 432
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Expected stay at the hospital is less than 4 weeks from the first dose of study drug.
2. Currently receiving mechanical ventilation via endotracheal tube (continuous positive airway pressure [CPAP] or high-flow nasal cannula are not criteria for exclusion).
3. Require =30% oxygen (if on CPAP or in head box) or >0.5 L/min oxygen.
4. Evidence of severe brain disease or damage, including grade III or IV peri- or intraventricular hemorrhage, meningitis or hydrocephalus, grade III or IV intracranial hemorrhage, or periventricular leukomalacia.
5. Presence of major dysmorphology or congenital abnormalities that are likely to affect growth and/or development.
6. Current clinical evidence of hemodynamically significant persistent ductus arteriosus.
7. Clinical evidence of sepsis (including low or high white blood cell count and/or low platelet count and bacteriologically proven evidence of systemic infection). This should be based on the investigator’s opinion and available local laboratory reference ranges. Patients should not have received antibiotics in preceding 48 hours except where these are being administered for prophylaxis as per routine unit protocols (eg, antifungal prophylaxis with fluconazole).
8. Evidence of congenital infection (eg, cytomegalovirus).
9. Previous or current diagnosis of necrotizing enterocolitis (Bell’s stage 2 or greater).
10. Prior or current treatment with corticosteroids, except hydrocortisone.
11. Presence of any condition that in the opinion of the investigator makes the patient unsuitable for inclusion.
12. Enrolled in another concurrent clinical intervention study.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: The primary objective of this study is to demonstrate that rhBSSL improves growth in preterm infants as compared with placebo when administered in infant formula or PBM.;Secondary Objective: The secondary objectives of this study are<br>• To determine the effect of rhBSSL treatment in shortening the time of hospital stay<br>• To determine the effect of rhBSSL treatment in improving early development <br>• To determine the effect of rhBSSL treatment in decreasing readmittance to hospital<br>• To determine the effect of rhBSSL treatment in increasing the levels of DHA and AA<br>• To compare the safety and tolerability of rhBSSL treatment with that of placebo treatment<br>;Primary end point(s): The primary efficacy variable is growth velocity in grams per kilogram per day during 4 weeks of treatment.;Timepoint(s) of evaluation of this end point: Baseline and weekly to discharge then 3 and 12 month follow-up

Secondary Outcome Measures

Name	Time	Method

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