Compassionate Use of Omegaven® in the Treatment of Parenteral Nutrition Associated Hepatic Injury

• Status: Approved for marketing
• Trial end date: 1969-12-31
• Target enrollment: 0
• Participant gender: All

Summary

The overall purpose of this study is to determine if replacing standard soybean oil based fat emulsions with Omegaven®, a fish oil based fat emulsion, can reverse or prevent the progression of parenteral nutrition associated cholestasis. It is a compassionate use protocol for patients who already have significant cholestasis related to parenteral nutrition.

• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Jeffrey Rudolph; University of Pittsburgh
• Collaborators: Children's Hospital of Pittsburgh; University of Pittsburgh
• Treatments: Fat Emulsions, Intravenous

Criteria

Inclusion Criteria:

• Patients must be 2 months -10 years of age.

• Patients who are PN dependent (unable to meet nutritional needs solely by enteral nutrition) and are expected to require PN for at least another 30 days

• Patients considered eligible for study participation must have parenteral nutrition associated liver disease (PNALD) as defined by a direct bilirubin of >2.0mg/dL. Other causes of liver disease should be excluded. A liver biopsy is not necessary for treatment.

• 2 consecutive direct bilirubin test results >2.0mg/dL

• The patient must have failed standard therapies to prevent the progression of liver disease such as surgical treatment, cyclic TPN, avoiding overfeeding, reduction/removal of copper and manganese from TPN, advancement of enteral feeding, and use of ursodiol (Actigall).

• Subjects who are currently under treatment for PNALD with Compassionate Use Omegaven and have a direct Bilirubin of< 2, but who remain TPN dependent and require continued therapy with Omegaven.

Exclusion Criteria:

• Other known causes of chronic liver disease (hepatitis C, cystic fibrosis, biliary atresia and alpha 1 anti-trypsin deficiency)

• Enrollment in any other clinical trial involving an investigational agent (unless approved by the designated physicians on the multidisciplinary team)

• The parent, guardian, or child is unwilling to provide consent or assent

• Allergy to any fish product, egg protein, and/or previous allergy to Omegaven

• Active coagulopathies characterized by on-going bleeding or by a requirement for clotting factor replacement such as fresh frozen plasma or cryoprecipitate to maintain homeostasis

• Impaired lipid metabolism or severe hyperlipidemia with or without pancreatitis

• Unstable diabetes mellitus

• Stroke/embolism

• Collapse and shock

• Undefined coma status

• Active infection at time of initiation of Omegaven® up until such time as child is afebrile with stable vital signs and one negative 48 hour culture.