Omegaven® in the Treatment of Parenteral Nutrition (PN) Induced Liver Injury

Phase 2

Completed

Brief Summary: The purpose of this study is to determine whether the omega-3 fatty acid emulsion (Omegaven®), when used in place of the conventional soy-based fat emulsion (Intralipid), is effective in treating parenteral nutrition associated liver disease (PNALD) in children.

The study hypothesis is that Omegaven® can be safely provided to children who are dependent on parenteral nutrition and have PNALD, and can reverse or prevent progression of PNALD until the child can take adequate nutrition by mouth.

Detailed Description: Rationale for Omegaven® Treatment

Unlike conventional intravenous fat emulsions, Omegaven® is comprised solely of fish oils containing primarily omega-3 fatty acids. Animal studies have shown that IV lipid emulsions such as fish oil that are high in eicosapentaenic and docosahexaenoic acid reduce impairment of bile flow as seen in cholestasis caused by conventional fat emulsions(1,2). By administering Omegaven® in place of conventional phytosterol/soybean fat emulsions, the cholestasis may be reversed and patients will be able to be maintained on adequate PN until they are able to ingest adequate nutrition enterally.

References

1. Chen W. Effects of fat emulsions with different fatty acid composition on plasma and hepatic lipids in rats receiving total parenteral nutrition. Clinical Nutrition 1996;15:24.

2. Yeh S. Effects of fish oil and safflower oil emulsions on diet-induced hepatic steatosis in rats receiving total parenteral nutrition. Clinical Nutrition 1996;15:80.

Recruitment & Eligibility

Inclusion Criteria

Children 0-18 years of age
Patients will be PN-dependent and expected to continue PN for at least 30 days
Patients considered eligible for study participation must have PN-associated liver diseases . Other causes of liver disease (i.e., biliary atresia, galactosemia, alpha-1 antitrypsin deficiency) will be excluded. A liver biopsy is not necessary for treatment
Direct bilirubin > 2.0 mg/dl
Signed patient informed consent
Signed patient assent where applicable.

Exclusion Criteria

Pregnancy
Other causes of chronic liver disease (cystic fibrosis, biliary atresia, alpha-1 antitrypsin deficiency)
Signs of advanced liver disease including cirrhosis on biopsy, varices, ascites
The patient is allergic to eggs/shellfish
The patient has a severe hemorrhagic disorder
The patient is enrolled in any other clinical trial involving an investigational agent (unless approved by the designated physicians on the multidisciplinary team)
The parent or guardian or child unwilling to provide consent or assent.

Arm && Interventions

Group	Intervention	Description
Single Arm	Omegaven®	Single Omegaven® Intervention Arm

Primary Outcome Measures

Name	Time	Method
Number of Participants With Normalization of Direct Bilirubin	Month 9	Normalization of direct bilirubin is defined as less than 0.4mg/dL by 9 months. This relates to the efficacy of parenteral administration of fish oil derived fat emulsion (Omegaven®) to reverse established parenteral nutrition associated liver disease.

Secondary Outcome Measures

Name	Time	Method
Number of Participants With Normal Essential Fatty Acid (EFA) Profiles.	Months 1 through 9	EFA is defined at a triene tetraene ratio of greater than 0.2 (so normal is \> 0.2). Collected monthly from month 1-9.
Number of Participants With Unexpected Bleeding or Coagulopathies	Month 6
Number of Participants With Triglyceride Levels > 400 mg/dL	baseline data and then weekly and monthly evaluations, for an average of 9 months	Number of participants with triglyceride levels \> 400 mg/dL

Locations (1): Levine Children's Hospital at Carolinas HealthCare System
🇺🇸
Charlotte, North Carolina, United States

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