Omegaven as Alternative Parenteral Fat Nutrition

Study Overview
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Brief Summary
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To provide Omegaven® as a compassionate treatment for critically ill infants with parenteral nutrition associated cholestasis

Detailed Description
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An intermediate population, Investigational New Drug study, to provide an alternate intravenous fat nutrition, Omegaven®, to help improve liver function while providing adequate nutrition for critically ill infants with parenteral nutrition associated cholestasis

Official Title
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Omegaven as Alternative Parenteral Fat Nutrition

Conditions
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Cholestasis, 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: Greater than 14 days old Has parenteral nutrition associated cholestasis defined as at least 2 consecutive direct bilirubin >2 mg/dL with anatomical or functional short gut (OR >4 mg/dL if intact intestine) obtained at least 1 week apart with a ratio of direct: total bilirubin > 0.4 Patient is parenteral nutrition dependent (unable to meet nutritional needs solely by enteral nutrition) and are expected to require parenteral nutrition for at least 3 more weeks Patient has not responded to other therapeutic approaches for parenteral nutrition-associated liver disease such as : cycling of parenteral nutrition, avoiding overfeeding, reduction/removal of copper and manganese from parenteral nutrition, advancement of enteral feeding, and use of Ursodiol Patient has been on at least 2 weeks of SMOFLIPIDs with no improvement or worsening of direct bilirubin levels Signed patient informed consent The patient is expected to have a reasonable possibility of survival No other known etiology of cholestasis other than parenteral nutrition-associated liver disease at the time of Omegaven® initiation Exclusion Criteria: Known causes of cholestasis other than parenteral nutrition-associated liver disease including but not limited to Hepatitis C, Cystic fibrosis, biliary atresia, and alpha 1 antitrypsin deficiency are present, prior to Omegaven® initiation Known fish or egg allergy Any of the contraindications to use of Omegaven®: Active new infection at the time of initiation of Omegaven® Hemodynamic instability Recent use of medications with associated risk of bleeding, including NSAIDs Active coagulopathy or bleeding Platelet counts persistently under 30,000 despite transfusions Unstable hyperglycemia Impaired lipid metabolism (triglycerides >1000 mg/dL) while on 1 g/kg/day or less of Intralipid History of severe hemorrhagic disorders (ie. hemophilia, Von Willebrand disease, etc.) Unstable diabetes mellitus Collapse and shock Stroke/ Embolism Cardiac infarction within the last 3 months Undefined coma status

Ages Eligible for Study
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Minimum Age: 14 Days

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: Drug - Omegaven®<br>Therapy with Omegaven® will be initiated at the goal dose of 0.5-1/kg/day. The default is over 24 hours, but shorter intervals may be considered if a program of total parenteral nutrition cycling is recommended. Omegaven® will be infused intravenously through either a central or peripheral catheter. | Drug: Omegaven<br>* Omegaven® fat emulsion will be used as a compassionate use treatment for critically ill infants with parenteral nutrition associated liver injury<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participant With Decrease in Direct Bilirubin Level | Number of participant with decrease in direct bilirubin levels within 30 days of treatment | First month of treatment | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participant With Resolution of Direct Hyperbilirubinemia | Number of participant with Direct bilirubin level of <2mg/dL at discontinuation of treatment | 2 months | 
| Number of Participant With Preservation of Length | Monitoring of weekly length while the patient is receiving Omegaven® treatment | Weekly measurement during treatment. Evaluation every 30 days from treatment initiation until discontinuation of treatment, patient hospital discharge, whichever comes first, or up to 6 months following initiation of treatment | 
| Number of Participant With Preservation of Head Circumference | Monitoring of weekly head circumference while the patient is receiving Omegaven® treatment | Weekly measurement during treatment. Evaluation every 30 days from treatment initiation until discontinuation of treatment, patient hospital discharge, whichever comes first, or up to 6 months following initiation of treatment | 
| Number of Participant With Preservation of Normal Weight Gain | Monitoring of daily weight gain while the patient is on Omegaven® | Weekly measurement during treatment. Evaluation every 30 days from treatment initiation until discontinuation of treatment, patient hospital discharge, whichever comes first, or up to 6 months following initiation of treatment | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Omegaven, parenteral nutrition, Liver disease, Cholestasis