Intravenous Fat Emulsion Comprised of Fish Oil in the Treatment of Parenteral Nutrition Induced Liver Injury in Infants

Study Overview
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Brief Summary
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To provide a mechanism for critically ill infants with parenteral nutrition (PN) associated cholestasis to receive Omegaven for compassionate use situations for which there are no satisfactory alternative treatments.

Official Title
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Compassionate Use of an Intravenous Fat Emulsion Comprised of Fish Oil in the Treatment of Parenteral Nutrition Induced Liver Injury in Infants

Conditions
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Total Parenteral Nutrition-induced Cholestasis

Intervention / Treatment
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* Drug: Omegaven
* Drug: Omegaven
* Drug: Omegaven


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Critically ill infants with either a known anatomic short gut (greater than 50% of the bowel removed) or known severe dysmotility of the gut reflecting non-functional gut similar to anatomic short gut will be offered Omegaven® when their direct bilirubin reaches 2 mg/dL. Infants who do not meet the above criteria for anatomic or dysfunctional short gut will be allowed to receive Omegaven® when their direct bilirubin reaches 4 mg/dL. The qualifying measurements of 2 mg/dL or 4 mg/dL direct bilirubin must be consecutive and obtained at least 24 hours apart. Be expected to require intravenous nutrition for at least an additional 28 days Patient must have documented failure of or ineligibility for the following therapies to prevent progression of PNALD: Reduction of Intralipid® to 1 g/kg/day Limiting trace minerals including copper and manganese Initiation and use of Ursodiol Cycling of parenteral nutrition Advancement of enteral feedings Parental informed consent must be signed. Exclusion Criteria: Have a congenitally lethal condition (e.g. Trisomy 13). Have clinically severe bleeding not able to be managed with routine measures. Have evidence of a viral hepatitis or primary liver disease as the primary etiology of their cholestasis. Have other health problems such that survival is extremely unlikely even if the infant's cholestasis improves. Has culture positive sepsis

Ages Eligible for Study
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Minimum Age: 14 Days
Maximum Age: 2 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: 1<br>Drug: Omegaven | Drug: Omegaven<br>* Therapy with Omegaven will be provided at a dose of 1 gm/kg/day (by continuous infusion). Omegaven will be infused intravenously through either a central or peripheral catheter in conjunction with parenteral nutrition.<br>* Other names: omega-3 enriched fat emulsion;Drug: Omegaven<br>* Treatment will be given for as long as the child needs any TPN AND has a conjugated bilirubin greater than 2 mg/dL for a maximum of 2 years.<br>Drug: Omegaven<br>* If the bilirubin is less than 2 mg/dL but the child still requires TPN, then the Omegaven will be continued until the infant no longer requires TPN.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Death associated with liver disease | Those patients with death only associated with liver diseasepre-treatment period and the expected rate of growth. | Maximum 2 years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Liver transplantation |  | Maximum 2 years | 
| Growth over time as determined by weight in grams. |  | Maximum 2 years | 
| Growth over time as measured by length in cm. |  | Maximum 2 years | 
| Growth over time as measured by head circumference in cm. |  | Maximum 2 years | 
| Development of essential fatty acid (EFA) deficiency as measured by triene:tetraene ratio in those with prolonged NPO status. |  | Maximum 2 years | 
| Hyperlipidemia as measured by triglyceride level |  | Maximum 2 years | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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omegaven, cholestasis, parenteral nutrition associated liver disease