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Compassionate Use of Omegaven® for the Treatment of Intestinal Failure Associated Liver Disease in Children

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: NCT02328768
Recruitment Status : Completed
First Posted : December 31, 2014
Last Update Posted : October 15, 2018
Children's Hospital and Medical Center, Omaha, Nebraska
University of Nebraska
Information provided by (Responsible Party):
Brian Jones, MD, University of Nebraska

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 liver disease. It is a compassionate use protocol for patients who already have significant liver disease related to parenteral nutrition.

Condition or disease Intervention/treatment Phase
Total Parenteral Nutrition-Induced Cholestasis Drug: Omegaven Phase 2

Detailed Description:
Parenteral Nutrition (PN) is a potentially life-saving intervention for children with short bowel syndrome and intestinal failure. Many common neonatal surgical diseases including Necrotizing enterocolitis (NEC), intestinal atresias, and gastroschisis can cause intestinal failure. The recovery from these illnesses often involves prolonged periods of parenteral nutrition. Intestinal Failure Associated Liver Disease (IFALD) is the most prevalent complication of long term parenteral nutrition in children, affecting up to 2/3 of children with short bowel syndrome. Parenteral lipids are an important source of calories in children, and provide essential fatty acids. Development of IFALD is a multifactorial process. Phytosterols contained in soybean based lipid emulsions have been shown to predispose animals to IFALD. Previous studies in children have shown that dosing the soybean based parenteral lipid emulsion at doses greater than 1g/kg/day may contribute to the development of IFALD. It is currently our practice to limit the lipid dose in children at risk of development of IFALD to 1g/kg/day. Despite this, some patients will still develop biochemical evidence of cholestasis and IFALD. Previous studies in humans have shown that children with IFALD who were administered the intravenous fish oil lipid emulsion Omegaven® reduced their serum direct bilirubin levels. This may be due to a reduction in the amount of arachidonic acide derived inflammatory mediators. 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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 6 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Compassionate Use of an Intravenous Fish Oil Lipid Emulsion (Omegaven®) for the Treatment of Intestinal Failure Associated Liver Disease in Children
Actual Study Start Date : April 3, 2015
Actual Primary Completion Date : November 10, 2017
Actual Study Completion Date : September 5, 2018

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Liver Diseases
Drug Information available for: Fish oil

Arm Intervention/treatment
Experimental: Omegaven®
Omegaven® 10% fat emulsion 1g/kg/day to infuse via IV over a period of 8-24 hours every day until the patient no longer requires intravenous lipid emulsion, is determined ineffective, or the patient stops participating in the study for any reason.
Drug: Omegaven
Other Name: Intravenous fish oil emulsion

Primary Outcome Measures :
  1. 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 ]
    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.

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Parenteral nutrition (PN) dependent (unable to meet nutritional needs solely by enteral nutrition) and expected to require PN for at least another 30 days.
  • Parenteral nutrition associated liver disease (PNALD) as defined as a direct bilirubin ≥ 2mg/dL or by histology and/or currently on Omegaven through another protocol.
  • Other causes of liver disease have been excluded. A liver biopsy is not necessary for treatment.
  • Exhaustion of standard therapies to prevent the progression of the liver disease including surgical treatment, cyclic PN, avoiding overfeeding, reduction/removal of copper and manganese form PN, advancement of enteral feeding, and the use of ursodiol.

Exclusion Criteria:

  • Pregnancy
  • Other causes of liver disease
  • Enrollment in any other clinical trial involving an investigational agent (unless approved by the designated physicians on the multidisciplinary team)
  • The parent/guardian or subject is unwilling to provide consent and assent

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): NCT02328768

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United States, Nebraska
Children's Hospital & Medical Center
Omaha, Nebraska, United States, 68114
Sponsors and Collaborators
Brian Jones, MD
Children's Hospital and Medical Center, Omaha, Nebraska
University of Nebraska
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Principal Investigator: Brian A Jones, MD University of Nebraska Medical Center/Children's Hospital & Medical Center
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Responsible Party: Brian Jones, MD, Assistant Professor of Surgery, University of Nebraska Identifier: NCT02328768    
Other Study ID Numbers: O-2014-BJ
First Posted: December 31, 2014    Key Record Dates
Last Update Posted: October 15, 2018
Last Verified: October 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Additional relevant MeSH terms:
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Liver Diseases
Digestive System Diseases
Bile Duct Diseases
Biliary Tract Diseases