Compassionate Use of Omegaven IV Fat Emulsion

Study Overview
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Brief Summary
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The purpose of the study is to provide use of Omegaven in children with life threatening Total Parenteral Nutrition (TPN) associated cholestasis when all other medical and surgical treatments/therapies have been either ineffective or not feasible in the treatment of this serious condition. This is a compassionate use protocol.

Official Title
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Compassionate Use of Omegaven IV Fat Emulsion in Infants With Life Threatening Parenteral Nutrition-associated Liver Disease (PNALD).

Conditions
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Total Parenteral Nutrition-induced Cholestasis

Intervention / Treatment
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* Drug: Omegaven IV lipid emulsion


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Diagnosis of life threatening PNALD Dependent on Parenteral Nutrition for caloric needs Expected to require PN for at least another 30 days Other causes of liver disease have been excluded GI/Liver service is involved in patient care Standard therapies for the treatment of PNALD must have been utilized prior to initiating omegaven. Inpatient at Morgan Stanley Children's Hospital of NewYork Presbyterian Hospital Must be at least 2 months of age Exclusion Criteria: Not inpatient Younger than 2 months of age Expected to be weaned off of parenteral nutrition within 30 days have other documented causes of liver disease have signs of proven severe advanced liver disease Allergy to seafood, egg protein and/or previous allergy to Omegaven active coagulopathy, impaired lipid metabolism or severe hyperlipidemia with or without pancreatitis, stoke, embolism, hemodynamic collapse or shock, recent MI, cholestasis due to any reason other than PNALD, active new infection at time of initiation of Omegaven, hemodynamic instability or if unable to tolerate the necessary laboratory monitoring must not be enrolled in another clinical trial involving an investigational agent (unless approved by the designated physicians). Parent or legal guardian must be willing to provide consent.

Ages Eligible for Study
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Minimum Age: 2 Months
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>Omegaven IV lipid emulsion administration for infants with life threatening parenteral nutrition associated liver disease | Drug: Omegaven IV lipid emulsion<br>* For infants meeting inclusion criteria and whose guardians consent to participation, Omegaven will be initiated at the dose of 0.5gram/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 a central or peripheral catheter alone or in conjunction with parenteral nutrition.<br>* Other names: Omega-3 Fatty Acids;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Reduction of direct serum bilirubin level (mg/dL) | For effectiveness endpoint, the allowable minimum time interval between serial direct bilirubin measurements that will determine reversal of cholestasis is 2 consecutive measurements of direct bilirubin < or = to 2mg/dL obtained at least 2 weeks apart. | Up to 6 months from the completion of the study. | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Essential fatty acid profile | Previous studies have found no association between Omegaven administration and essential fatty acid deficiency. Due to theoretical concern for developing fatty acid deficiency with low dose Omegaven administration, these levels will be followed during Omegaven therapy. | Up to 30 days from therapy completion | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Total Parenteral Nutrition-induced Cholestasis, Omegaven, liver disease, Cholestasis, Fat emulsions