Compassionate Use of Omegaven in Children

Study Overview
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Brief Summary
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This is a single-assignment study to evaluate whether Omegaven (IV fish oil) is effective at treating liver disease in children on long-term IV nutrition.

Detailed Description
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Children up to 18 years of age, on parenteral nutrition, with a direct bilirubin level of 2 mg/dL or greater will be eligible to receive Omegaven at a maximum dose of 1 g/kg/day. Up to 200 children will be eligible for enrollment. Direct bilirubin levels and other labs will be monitored as well as growth parameters.

Official Title
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Fish Oil Lipid Emulsion for the Treatment of Parenteral-Nutrition-Associated Liver Disease in Infants

Conditions
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Total Parenteral Nutrition-induced Cholestasis, Cholestasis, Short Bowel Syndrome, Gastroschisis, Intestinal Atresia, Infant, Premature, Diseases, Cholestasis of Parenteral Nutrition

Intervention / Treatment
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* Drug: Omegaven


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Live in or temporarily relocate to Oklahoma Age less than 18 years, both sexes, all races Have a direct bilirubin level of ≥2 mg/dL for two consecutive weeks after at least 14 days of parenteral nutrition Received parenteral lipids at a maximum dose of 1.7 g/kg/day (24 g/kg over the two weeks prior) Are not currently enrolled in another lipid emulsion study Exclusion Criteria: Known food allergy to fish Known metabolic disorder of lipid metabolism Active coagulopathies (active bleeding or requiring blood product treatment in the prior 48 hours) Medical condition likely to result in death in the next 30 days

Ages Eligible for Study
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Maximum Age: 18 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 |
| --- | --- |
| Experimental: Omegaven<br>Children will receive Omegaven at a maximum of 1 g/kg/day upon enrollment in this arm. | Drug: Omegaven<br>* Once the direct bilirubin is 2 mg/dL or more x 2 weeks, Intralipid will be switched to Omegaven at 1 g/kg/day. The bilirubin level will be monitored to determine when resolution of cholestasis (DB <2 mg/dL) occurs.<br>* Other names: Omega-3-fatty acid lipid emulsion;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Resolution of Cholestasis for Subjects Who Received Omegaven | To determine if Omegaven results in the resolution of cholestasis (DB <2 for 2 consecutive weeks) | Within the first 3 months of sole Omegaven use | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety Issues for Infants Who Received Omegaven | To determine if Omegaven results in safety issues such as increased bleeding, essential fatty acid deficiency, elevated liver function tests, increased liver and/or intestinal transplant rates, or death | Within the first year of use | 
| Essential Fatty Acid Deficiency in Infants Who Received Omegaven | To determine if Omegaven can resolve essential fatty acid deficiency | Within the first month of use | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Fat Emulsions, Intravenous, Parenteral-Nutrition-Associated Liver Disease, Cholestasis, Omegaven, Fish Oil