Comparison of rhBSSL With Placebo When Added to Infant Formula or Pasteurized Breast Milk During 4 Weeks of Treatment in Preterm Infants

• Register: ClinicalTrials.gov
• Last refreshed on: 10 August 2015
• Main ID: NCT01413581
• Date of registration: 08/06/2011
• Prospective Registration: No
• Primary sponsor: Swedish Orphan Biovitrum
• Public title: Comparison of rhBSSL With Placebo When Added to Infant Formula or Pasteurized Breast Milk During 4 Weeks of Treatment in Preterm Infants LAIF
• Scientific title: A Prospective, Randomized, Double-Blind, Phase 3 Study Comparing rhBSSL and Placebo Added to Infant Formula or Pasteurized Breast Milk During 4 Weeks of Treatment in Preterm Infants Born Before Week 32 of Gestational Age
• Date of first enrolment: May 2011
• Target sample size: 415
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT01413581
• Study type: Interventional
• Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator), Primary Purpose: Treatment
• Phase: Phase 3

Countries of recruitment

Belgium, Czech Republic, France, Germany, Hungary, Italy, Poland, Russian Federation, Spain, Sweden, United Kingdom

Contacts

• Name: Kristina Timdahl, MD
• Affiliation: Swedish Orphan Biovitrum

Key inclusion & exclusion criteria

Inclusion Criteria:

- Preterm infants born before week 32 of gestation.

- Preterm infant who is less than 33 weeks postmenstrual age at the time of
randomization.

- Preterm infant who is appropriate for gestational age (AGA) or small for gestational
age (SGA) at birth.

- Preterm infant who is receiving food enterally (bottle or gavage tube) at a level of
at least 100 ml/kg/day at randomization.

- Preterm infant whose enteral feeding consists of only infant formula or only
pasteurized breast milk at the time of inclusion, and who are expected to remain on
only infant formula for 4 weeks, or only pasteurized breast milk for at least 2 weeks
following treatment initiation.

- Preterm infant who is expected not to receive any fresh breast milk for 4 weeks
following treatment initiation.

- Informed consent is obtained.

Exclusion Criteria:

- Expected stay in the hospital is less than 4 weeks from the first dose of study drug.

- Criteria to ensure no disease or treatment affecting growth or development, e.g.
brain disease, necrotizing enterocolitis.

- Enrolled in another concurrent clinical intervention study.



Age minimum: N/A
Age maximum: 10 Weeks
Gender: Both

Health Condition(s) or Problem(s) studied

Prevention of Growth Restriction

Intervention(s)

Drug: rhBSSL (recombinant human bile-salt-stimulated lipase)

Drug: Placebo

Primary Outcome(s)

Growth velocity in grams per kilogram per day during 4 weeks of treatment. [Time Frame: Baseline and Day 29]

Secondary Outcome(s)

Arachidonic acid (% of total fatty acids) in the serum phosphatidylcholine fraction at 4 weeks [Time Frame: Day 29]

Change from baseline in body weight (g) at 3 months [Time Frame: Baseline and Month 3]

Arachidonic acid (% of total fatty acids) in the serum triglyceride fraction at 4 weeks [Time Frame: Day 29]

Bayley Scale of Infant and Toddler Development, third edition: Cognitive domain composite score at 24 months' corrected age [Time Frame: 24 months' corrected age]

Bayley Scale of Infant and Toddler Development, third edition: Language domain composite score at 24 months' corrected age [Time Frame: 24 months' corrected age]

Change from baseline in head circumference (mm) at 4 weeks. [Time Frame: Baseline and Day 29]

Docosahexaenoic acid (% of total fatty acids) in the serum triglyceride fraction at 4 weeks [Time Frame: Day 29]

Number of days lost from work related to the child's condition from the time of initial hospital discharge to home up to 24 months' corrected age [Time Frame: Date of initial hospital discharge to home to date of 24 months' corrected age]

Number of outpatient visits from the time of initial hospital discharge to home up to 24 months' corrected age [Time Frame: Date of initial hospital discharge to home to date of 24 months' corrected age]

Bayley Scale of Infant and Toddler Development, third edition: Motor domain composite score at 12 months' corrected age [Time Frame: 12 months' corrected age]

Bayley Scale of Infant and Toddler Development, third edition: Motor domain composite score at 24 months' corrected age [Time Frame: 24 months' corrected age]

Bayley Scale of Infant and Toddler Development, third edition: Language domain composite score at 12 months' corrected age [Time Frame: 12 months' corrected age]

Head circumference (mm) at 12 months' corrected age. [Time Frame: 12 months´ corrected age]

Re-admission to hospital within 1 month of discharge [Time Frame: Date of discharge and date of re-admission]

Number of patients with antibodies to rhBSSL at 12 months' corrected age [Time Frame: 12 months' corrected age]

Bayley Scale of Infant and Toddler Development, third edition: Adaptive behavior domain composite score at 24 months' corrected age [Time Frame: 24 months' corrected age]

Body weight (g) at 12 months' corrected age [Time Frame: 12 months´ corrected age]

Body weight (g) at 24 months' corrected age [Time Frame: 24 months´ corrected age]

Bayley Scale of Infant and Toddler Development, third edition: Cognitive domain composite score 12 months' corrected age [Time Frame: 12 months' corrected age]

Number of inpatient visits from the time of initial hospital discharge to home up to 24 months' corrected age [Time Frame: Date of initial hospital discharge to home to date of 24 months' corrected age]

Change from baseline in head circumference (mm) at 3 months. [Time Frame: Baseline and Month 3]

Growth restriction [Time Frame: Day 29]

Change from baseline in total body length (mm) at 3 months [Time Frame: Baseline and Month 3]

Level of Vitamin A (nmol/L) at 4 weeks [Time Frame: Day 29]

Level of Vitamin D (nmol/L) at 4 weeks [Time Frame: Day 29]

Neurodevelopment Disability Composite [Time Frame: 24 months' corrected age]

Number of days of hospitalization from the time of initial hospital discharge to home up to 24 months' corrected age [Time Frame: Date of initial hospital discharge to home to date of 24 months' corrected age]

Number of patients with antibodies to rhBSSL at Month 3 [Time Frame: Month 3]

Number of patients with at least one treatment emergent Adverse Event [Time Frame: Baseline and Day 29]

Number of patients with at least one Serious Adverse Drug Reaction [Time Frame: 12 months' corrected age and 24 months' corrected age]

Number of patients with at least one treatment emergent Serious Adverse Event [Time Frame: Baseline and Day 29]

Presence of chronic medical conditions/diagnoses at 24 months' corrected age [Time Frame: 24 months' corrected age]

Time from baseline to 150 mL/kg/day of enteral feeding [Time Frame: Date of first dose and date 150 mL/kg/day of enteral feeding achieved (or exceeded)]

Total body length (mm) at 12 months' corrected age [Time Frame: 12 months' corrected age]

Time to discharge [Time Frame: Baseline and date of discharge]

Time to readiness for discharge [Time Frame: Baseline and date of readiness for discharge]

Change from baseline in total body length (mm) at 4 weeks [Time Frame: Baseline and Day 29]

Child Behavior Checklist total problem score at 24 months' corrected age [Time Frame: 24 months' corrected age visit]

Docosahexaenoic acid (% of total fatty acids) in the serum phosphatidylcholine fraction at 4 weeks [Time Frame: Day 29]

Head circumference (mm) at 24 months' corrected age. [Time Frame: 24 months´ corrected age]

Number of patients with antibodies to rhBSSL at 4 weeks [Time Frame: Day 29]

Number of patients with at least one treatment emergent Serious Adverse Event [Time Frame: Day 29 and Month 3]

Bayley Scale of Infant and Toddler Development, third edition: Social-emotional domain composite score at 24 months' corrected age [Time Frame: 24 months' corrected age]

Number of days in intensive care unit from the time of initial hospital discharge to home up to 24 months' corrected age [Time Frame: Date of initial hospital discharge to home to date of 24 months' corrected age]

Number of patients with at least one treatment emergent Adverse Event [Time Frame: Day 29 and Month 3]

Total body height (cm) at 24 months' corrected age [Time Frame: 24 months' corrected age]

Secondary ID(s)

BVT.BSSL-030

Source(s) of Monetary Support

Please refer to primary and secondary sponsors