Prevention of growth retardation due to lack of bile salt-stimulated lipase in enteral nutrition MedDRA version: 14.1 Level: PT Classification code 10036590 Term: Premature baby System Organ Class: 10036585 - Pregnancy, puerperium and perinatal conditions
Conditions
Interventions
Product Name: Recombinant Human Bile Salt-stimulated Lipase
Product Code: rhBSSL
Pharmaceutical Form: Powder for oral solution
INN or Proposed INN: bucelipase alfa
Current Sponsor code: rhBSSL
Other d
Sponsors
Swedish Orphan Biovitrum AB (publ)
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Preterm infant born before Week 32 of gestation. 2. Preterm infant who is =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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 • To determine the effect of rhBSSL treatment in shortening the time of hospital stay • To determine the effect of rhBSSL treatment in improving early development • To determine the effect of rhBSSL treatment in decreasing readmittance to hospital • To determine the effect of rhBSSL treatment in increasing the levels of DHA and AA • To compare the safety and tolerability of rhBSSL treatment with that of placebo treatment ;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
| Measure | Time frame |
|---|---|
| Secondary end point(s): Change from Baseline in body weight (g) at 3 months Body weight (g) at 12 months’ corrected age Change from Baseline in total body length (mm) at 4 weeks and 3 months Body length (mm) at 12 months’ corrected age Time to readiness for discharge Time to discharge Change from Baseline in head circumference (mm) at 4 weeks and 3 months Head circumference (mm) at 12 months’ corrected age Time from Baseline to 150 mL/kg/day of enteral feeding Readmission to hospital within 1 month of discharge Levels of DHA and AA at 4 weeks Adverse events (AEs) Physical examination Bayley Scale of Infant and Toddler Development III (cognitive, language,motor) Vomiting (frequency and volume) Vital signs (heart rate, blood pressure, body temperature) Laboratory variables including amylase, bilirubin, aminotransferases, sodium and urea Levels of vitamin A and D Levels of rhBSSL antibodies;Timepoint(s) of evaluation of this end point: Baseline and weekly to discharge then 3 and 12 month follow-up | — |
Countries
Belgium, Czech Republic, France, Germany, Hungary, Italy, Poland, Russian Federation, Spain, Sweden, United Kingdom
Contacts
Public ContactTracy Roe
PPD
Outcome results
None listed