Compassionate Use of Omegaven IV Fat Emulsion

Study Overview
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Brief Summary
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This is a compassionate use protocol to use intravenous fish oil infusion, Omegaven®, to infants and children with parenteral nutrition-associated liver disease to enable reversal of elevated serum liver enzymes and direct bilirubin (cholestasis).

Detailed Description
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Intravenous lipids are necessary in PN (parenteral nutrition) dependent patients due to their high caloric value and essential fatty acid content. However, parenteral fat emulsions composed of soybean oils (omega 6 fatty acids) have been implicated in predisposing patients to PN associated liver disease. It is hypothesized that by administering Omegaven®, comprised of fish oil (omega 3 fatty acids) in place of conventional soybean fat emulsion, the progression of PN-associated cholestasis may be prevented or reversed.

Official Title
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Compassionate Use of Omegaven IV Fat Emulsion

Conditions
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Cholestasis, Total Parenteral Nutrition-induced Cholestasis

Intervention / Treatment
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* Drug: Omegaven


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Two consecutive direct bilirubin levels of 2 mg/dl or more in a parenteral nutrition dependent infant or child (unable to meet nutritional needs solely by enteral nutrition) Other causes of liver disease have been excluded. A liver biopsy is not necessary for treatment. The patient must have utilized standard therapies to prevent the progression of the liver disease including reduction/removal of copper and manganese from daily PN, trial of enteral feeding if possible, and the use of ursodiol (i.e., Actigall®). Exclusion Criteria: Documented causes of chronic liver disease other than parenteral nutrition associated liver disease Proven severe advanced liver disease including cirrhosis on biopsy, varices, ascites. An allergy to any seafood product, egg protein, and/or previous allergy to Omegaven® Active coagulopathy characterized by ongoing bleeding or by a requirement for clotting factor replacement (e.g. fresh frozen plasma or cryoprecipitate) to maintain homeostasis Impaired lipid metabolism or severe hyperlipidemia with or without pancreatitis Unstable diabetes mellitus or hyperglycemia Stroke, embolism, collapse and shock, recent MI Cholestasis due to any reason other than parenteral associated liver disease Active new infection at time of initiation of Omegaven® Hemodynamic instability The patient may not be enrolled in any other clinical trial involving an investigational agent (unless approved by the designated physicians on the multidisciplinary team).

Ages Eligible for Study
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Minimum Age: 2 Weeks
Maximum Age: 5 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Other: Omegaven<br>All subjects will receive Omegaven | Drug: Omegaven<br>* Omegaven® will be initiated at a dose of 0.5 gram/kg/day and is infused over 24 hours for 1-2 days, and then advanced to 1 gram/kg/day. Omegaven® will be infused intravenously through either a central or peripheral catheter alone or in conjunction with parenteral nutrition. Omegaven® will continue until weaned from PN. Monotherapy with Omegaven® can continue as an additional source of calories after the dextrose/protein portion of PN is discontinued. Omegaven may be restarted within seven days of discontinuing therapy. After seven days, and meeting inclusion criteria, Omegaven can resume at the initial dose of 0.5 grams/kg/day, advancing to 1 gm/kg/day.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Level of Bilirubin | measurement of bilirubin level weekly. Available data reported. | week 2, 3,4,and 8 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Cholestasis, Parenteral Nutrition Associated Liver Disease