COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC:

Get the latest research information from NIH: Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01412359
Expanded Access Status : Approved for marketing
First Posted : August 9, 2011
Last Update Posted : April 24, 2019
University of Pittsburgh
Information provided by (Responsible Party):
Jeffrey Rudolph, University of Pittsburgh

Brief 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.

Condition or disease Intervention/treatment
Total Parenteral Nutrition-induced Cholestasis Drug: Omegaven®

Detailed Description:

In the United States, patients dependent upon parenteral nutrition (PN) receive parenteral fat emulsions composed of soybean oils. Lipids are necessary in PN dependent patients due to their high caloric value and essential fatty acid content. Phytosterols such as those contained in soybean oil based fat emulsions are thought to have a deleterious effect on biliary secretion and they have been implicated in predisposing patients to PN associated liver disease. Children requiring prolonged courses of PN are at risk for developing PN associated liver disease. The investigators hypothesize that although soybean oil based fat emulsions prevent fatty acid deficiency, they are not cleared in a manner similar to enteral chylomicrons and therefore accumulate in the liver resulting in steatotic liver injury.

Animal studies have shown that fish oil based IV fat emulsions(IFE)which are high in eicosapentaenic and docosahexaenoic acid, reduce impairment of bile flow seen in cholestasis caused by conventional fat emulsions. Omegaven® is a fish oil based IV fat emulsion. The investigators hypothesize that administering Omegaven® in place of conventional soybean fat emulsions may reverse or prevent the progression of PN associated cholestasis and thus allow the patient to be maintained on adequate PN until he/she is able to ingest adequate nutrition enterally.

Primary outcome measures to be addressed include the normalization of liver enzymes including bilirubin upon the start of Omegaven® and continued growth on Omegaven®. Safety measures will include the prevalence of essential fatty acid deficiency, hypertriglyceridemia, and bleeding disorders.

Layout table for study information
Study Type : Expanded Access
Expanded Access Type : Treatment IND/Protocol
Official Title: Compassionate Use of Omegaven® in the Treatment of Parenteral Nutrition Associated Hepatic Injury

Resource links provided by the National Library of Medicine

Intervention Details:
  • Drug: Omegaven®
    Omegaven® 10% fat emulsion 1g/kg/day to infuse via IV over a period of 12 -24 hours every day until patient no longer requires intravenous lipid emulsion, is determined ineffective, or the patient stops participating in the study for any reason.
    Other Name: Intavenous fish oil emulsion

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

Layout table for eligibility information
Ages Eligible for Study:   2 Months to 10 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01412359

Layout table for location information
United States, Pennsylvania
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States, 15224
Sponsors and Collaborators
Jeffrey Rudolph
University of Pittsburgh
Layout table for investigator information
Principal Investigator: Jeffrey A Rudolph, MD University of Pittsburgh
Layout table for additonal information
Responsible Party: Jeffrey Rudolph, Associate Professor, University of Pittsburgh Identifier: NCT01412359    
Other Study ID Numbers: PRO08080394
First Posted: August 9, 2011    Key Record Dates
Last Update Posted: April 24, 2019
Last Verified: April 2019
Keywords provided by Jeffrey Rudolph, University of Pittsburgh:
TPN Cholestasis
Additional relevant MeSH terms:
Layout table for MeSH terms
Bile Duct Diseases
Biliary Tract Diseases
Digestive System Diseases