Phase 3
N=21
Standard Lipid Therapy vs IVFE Minimization for Prevention of PNALD
Cholestasis
Bottom Line
View on ClinicalTrials.gov: NCT02357576 ↗Enrolled (actual)
21
Serious AEs
42.9%
Results posted
Jan 2020
Primary outcome: Primary: Rate of Rise of Direct Bilirubin as a Function of Time — 1.033001353; 1.05457485; 1.025504411; 1.049318179 mg/dL/day
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Intralipid 20% I.V. Fat Emulsion (Drug)
- Age
- Pediatric
- Sex
- All
- Sponsor
- University of Michigan
- Primary completion
- Oct 2017
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Rate of Rise of Direct Bilirubin as a Function of Time |
1.033001353; 1.05457485; 1.025504411; 1.049318179; 0.999691048; 1.031125269 | — |
| SECONDARY Prevalence of Parenteral Nutrition-associated Cholestasis (PNAC) (Direct Bilirubin ≥2 mg/dL) |
2; 3 | 0.617 |
| SECONDARY Prevalence of Severe Parenteral Nutrition-associated Cholestasis (PNAC) (Direct Bilirubin ≥4 mg/dL in Subjects on Parenteral Nutrition for at Least 2 Weeks) |
0; 1 | 0.450 |
| SECONDARY The Time to Development of PNAC |
19.50; 39.00 | 0.2716 |
| SECONDARY The Time to Development of Severe PNAC |
42 | — |
| SECONDARY Peak Total Bilirubin Level |
1.7; 2.9 | — |
| SECONDARY Peak Direct Bilirubin Level |
0.8; 1.7 | — |
| SECONDARY The Prevalence of Essential Fatty Acid Deficiency (EFAD) |
2; 0 | — |
| SECONDARY Adequacy of Growth as Evaluated by Z-scores for Weight |
-1.36; -0.93; -2.28; -1.85; -2.32; -1.3 | — |
| SECONDARY Adequacy of Growth as Evaluated by Z-scores for Height |
-1.06; -0.87; -1.365; -1.22; -1.53; -1.55 | — |
| SECONDARY Adequacy of Growth as Evaluated by Z-scores for Head Circumference |
-0.99; -0.94; -1.18; -1.3; -1.36; -0.82 | — |
| SECONDARY Adverse Events, as Defined by Any Episode of Sepsis and Catheter-related Blood Stream Infections |
2; 3; 1 | — |
| SECONDARY Bayley Scales for Infant and Toddler Development (BSID-III) at One Year |
9; 10; 18; 16; 15; 20 | — |
| SECONDARY Bayley Scales for Infant and Toddler Development (BSID-III) at Two Years |
7.5; 10.5; 17; 23; 17.5; 22 | — |
| SECONDARY MacArthur-Bates Communicative Development Inventories (CDI) |
25; 57.5; 20; 50; 16.25; 88.75 | — |
| SECONDARY Brief Infant Toddler Social Emotional Assessment (BITSEA) Part 1 of 2 |
26; 26; 25.5; 26 | — |
| SECONDARY Gross Motor Function Classification System (GMFCS) |
5; 2; 0; 0; 0; 0 | — |
| SECONDARY Behavioral Assessment System for Children-Third Edition (BASC3) Part 1 of 2 |
43; 55.5; 47; 48; 48.5; 53 | — |
| SECONDARY Behavioral Assessment System for Children-Third Edition (BASC3) Part 2 of 2 |
5; 1; 1; 1; 6; 1 | — |
| SECONDARY Brief Infant Toddler Social Emotional Assessment (BITSEA) Part 2 of 2 |
5; 2; 1; 0; 5; 2 | — |
Summary
Parenteral nutrition-associated cholestasis (PNAC) and liver disease (PNALD) are associated with significant morbidity and mortality in neonates and is felt to be exacerbated by soybean-based lipid emulsions. Much research is currently being directed at identifying ways to reduce this risk. Reduction of the dose of soybean-based lipid given as a component of parenteral nutrition is one possible strategy. In this study we will compare standard dosing of soybean-based lipid (up to 3/kg/day) with a minimized dose (1 g/kg/day) and evaluate for the development of cholestasis and adequate growth between the two groups. Longterm followup will include an assessment of neurodevelopmental outcomes at 12 and 24 months of age.
Funding source - FDA OOPD
Eligibility Criteria
Inclusion Criteria
- neonates and infants who are at least 28 weeks corrected gestational age at the time of enrollment who are parenteral nutrition (PN) naive
- current direct bilirubin 4cm or with liver herniated outside of the abdominal cavity
- necrotizing enterocolitis requiring surgical intervention
- volvulus
- intestinal atresia with >50% bowel loss
Exclusion Criteria
- weight =2 mg/dL at time of enrollment
- congenital or acquired anomaly which will require major cardiovascular surgery
- major congenital or chromosomal anomaly
- hypoxic ischemic encephalopathy
- congenital defect of the brain
- major seizure disorder
Data sourced from ClinicalTrials.gov (NCT02357576). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.