Compassionate Use of Omegaven® in the Treatment of Parenteral Nutrition Associated Hepatic Injury
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.
|Study Design:||Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Compassionate Use of Omegaven® in the Treatment of Parenteral Nutrition Associated Hepatic Injury|
- Primary efficacy outcome will be normalized direct bilirubin, defined as three consecutive direct bilirubin results < 2mg/dL or a direct bilirubin < 2mg/dL and weaned from total parenteral nutrition (TPN). [ Time Frame: 1 year ] [ Designated as safety issue: No ]Bilirubin levels will be monitored according to the standard of care for TPN dependent patients, usually weekly, for the duration of their participation in the study protocol. Duration of participation cannot be determined in advance.
|Study Start Date:||June 2009|
|Estimated Study Completion Date:||July 2012|
|Estimated Primary Completion Date:||July 2012 (Final data collection date for primary outcome measure)|
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.
|United States, Pennsylvania|
|Children's Hospital of Pittsburgh of UPMC|
|Pittsburgh, Pennsylvania, United States, 15224|
|Principal Investigator:||R. Cartland Burns, MD||Clinical Director, Division of Pediatric General and Thoracic Surgery, Children's Hospital of Pittsburgh of UPMC|