Expanded Access Use of Omegaven® in the Treatment of Parenteral Nutrition Induced Liver Injury in Children

Study Overview
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Brief Summary
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This is an expanded access study to assess the safety profile and changes in serum direct bilirubin levels in infants with PN associated cholestasis. Eligible patients will receive therapy with Omegaven on an expanded access basis by method of continuous infusion. Omegaven will be infused intravenously through either a central or peripheral catheter in conjunction with parenteral nutrition. The same standards of care provided to all patients receiving parenteral nutrition solution will be followed.

Detailed Description
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This expanded access protocol provides a mechanism for critically ill infants with parenteral nutrition-associated liver disease (PNALD) to receive Omegaven for compassionate use situations for which there are no satisfactory alternative treatments.

Official Title
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Expanded Access Use of Omegaven® in the Treatment of Parenteral Nutrition Induced Liver Injury in Children

Conditions
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Parenteral Nutrition-Associated Liver Disease

Intervention / Treatment
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* Drug: Omegaven


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Greater than 4 weeks (28 days) old and less then 5 years of age Diagnosis of PNALD as defined by serum direct bilirubin greater than 2 mg/dL on 2 consecutive occasions Expected to require intravenous nutrition for at least an additional 28 days Have been PN-dependent for at least four weeks prior to planned Omegaven initiation PN-dependent and unable to meet nutritional requirements by enteral means Have failed standard therapies to prevent progression of PNALD Hospitalized at time of Omegaven initiation Exclusion Criteria: Have a congenitally lethal condition (e.g. Trisomy 13). 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. Have been in another clinical trial within 30 days prior to enrollment or received an investigational drug within 30 days prior to enrollment or scheduled to receive an investigational drug other than Omegaven during the study period. Severe and/or unstable concomitant systemic disease such as complex congenital cardiac disease, renal failure, autoimmune disease, sepsis, inborn error of metabolism, genetic liver disease Bleeding disorder Biochemical disturbance with potential of worsening with proposed treatment, e.g. persistent hyperglycemia, hypertriglyceridemia, hypercalcemia.

Ages Eligible for Study
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Minimum Age: 4 Weeks
Maximum Age: 5 Years

Sexes Eligible for Study
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All

Study Plan
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How is the study designed?
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Arms and Interventions

| Intervention/Treatment |
| --- |
|Drug: Omegaven|Therapy with Omegaven will be provided at a dose of 1 gm/kg/day infused intravenously through either a central or peripheral catheter in conjunction with parenteral nutrition. Parenteral fat emulsion (Intralipid) will be administered only if necessary to administer adequate calories during Omegaven therapy. The same standards of care provided to all patients receiving parenteral nutrition solution will be followed. Home Use of Omegaven: The Omegaven dose for home use will be the same as that used while in the hospital: 1 gm/kg/day. As with the inpatient part of the protocol, this is a maximum dose and may be decreased at the discretion of the provider.|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Cholestasis