Compassionate Use of Omegaven for Pediatric Patients With Parenteral Nutrition Associated Liver Disease

Omegaven is an intravenous fat emulsion (IFE) comprised of omega-3 fatty acids derived from
fish oil. It will be used in an open label compassionate use treatment protocol, as an
alternative to soybean oil (omega-6), as the sole IFE source of parenteral nutrition in an
effort to reduce and/or reverse parenteral nutrition associated liver disease. The study will
evaluate the safety and efficacy of Omegaven use in pediatric patients with PN dependence and
PNALD.

Inclusion Criteria:

- PN dependence due to congenital or acquired gastrointestinal disease

- Predicted PN requirement for at least an additional 30 days

- Parenteral nutrition associated liver disease (PNALD), defined as two conjugated
bilirubin levels >= 2 mg/dL at least one week apart, must be obtained to demonstrate
persistence of PNALD

- Failure to respond to standard therapies which may include cycling PN, reduction in
the dose of soybean derived IFE, attempts to advance enteral feeds, ursodiol,
metronidazole, and avoidance of excessive caloric provision

- signed parent or legal guardian informed consent

Exclusion Criteria:

- Acute treatable infection (e.g. urinary tract infection, sepsis)

- Known allergy to egg or fish protein

- Contraindications to Omegaven

- Pregnancy

- Serum triglyceride level greater than 400 mg/dL at baseline

- History of severe hemolytic disorders or INR greater than 1.5 at baseline (INR cutoff
of greater than 2 for infants less than 1 week of age)