Compassionate Use of Omegaven to Reverse Parenteral Nutrition Induced Cholestasis

Not Applicable

Completed

Conditions: Cholestasis

Interventions: Drug: Omegaven

Registration Number: NCT01173159

Lead Sponsor: Children's Hospital Medical Center, Cincinnati

Brief Summary: The purpose of this research study is to see if giving Omegaven (an intravenous fat emulsion containing fish oil) instead of the current lipid emulsion, which contains fat derived from soybeans, as part of your child's intravenous (IV) nutrition therapy may be tolerated better. It may reduce the harmful effects to the liver, may stop any further liver damage and may reverse damage already done to the liver because of the prolonged use of nutrition through your child's IV.

Detailed Description: Enrollment of subjects into this study will occur for up to 4 years. Subjects will receive Omegaven at a dose of up to 1 g/kg body weight/day until they no longer require total parenteral nutrition or until their conjugated/direct bilirubin has normalized and their enteral lipid intake is sufficient to discontinue intravenous lipids.

Recruitment & Eligibility

Status: COMPLETED

Sex: All

Target Recruitment: 10

Inclusion Criteria

Males and females ages one month of age to 18 years of age
Patients with intestinal failure on TPN
Patients who have a conjugated/direct bilirubin of ≥3 mg/dl for more than weeks and in whom other causes of cholestasis have been excluded with reasonable certainty utilizing biochemical, serologic, microbiologic, and radiographic techniques. Liver biopsy is not required to rule out other disorders, but may be utilized at the clinician's discretion
Patients in whom reduction of IV soy-based lipid to an average <1.2g/kg body weight/day has failed to reduce the conjugated/direct bilirubin within ≥ 30 days of implementation
Willing to use birth control during study participation for females of child- bearing potential, as determined by investigator.
Signed informed consent for use of Omegaven® obtained

Exclusion Criteria

Any of the contraindications to use of Omegaven®
- Impaired lipid metabolism (triglycerides >1000 mg/dL) while on
  
  1g/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
- Pregnancy (positive pregnancy test) prior to enrollment in the study for females of child-bearing potential
- Females of child-bearing potential who are unwilling to use birth control during study participation
Parental decision to forego the use of Omegaven®
Known fish or egg allergy
Pregnancy
Causes of liver disease other than Parenteral Nutrition Associated Cholestasis

Study & Design

Study Type: INTERVENTIONAL

Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Omegaven	Omegaven	Subjects will receive Omegaven at a dose of up to 1 g/kg body weight/day until they no longer require Total Parenteral Nutrition or until their conjugated/direct bilirubin has normalized and their enteral lipid intake is sufficient to discontinue intravenous lipids.

Primary Outcome Measures

Name	Time	Method
Number of Participants With a Change in Conjugated/Direct Bilirubin	Completion of Therapy (time frame from 1-14 weeks)	Change in conjugated/direct bilirubin level to below 1 mg/dl.

Secondary Outcome Measures

Name	Time	Method
Number of Participants With a Change in Unconjugated/Total Bilirubin	Completion of Therapy (time frame from 1-14 weeks)	Change in unconjugated/total bilirubin level to below 1.1 mg/dL. General outcomes of participants are reported due to the enrollment of very few patients so that we have too small a cohort with which to assess achievement of our stipulated outcomes.
Number of Participants With a Change in Aspartate Transaminase (AST)	Completion of Therapy (time frame from 1-14 weeks)	Change in aspartate transaminase (AST) 57 units/L. General outcomes of participants are reported due to the enrollment of very few patients so that we have too small a cohort with which to assess achievement of our stipulated outcomes.
Number of Participants With a Change in Liver Enzyme (ALT)	Completion of Therapy (time frame from 1-14 weeks)	Change in liver enzyme ALT to below 59 unit/L. General outcomes of participants are reported due to the enrollment of very few patients so that we have too small a cohort with which to assess achievement of our stipulated outcomes.
Number of Participants With a Change in Liver Enzyme Alkaline Phosphatase	Completion of Therapy (time frame from 1-14 weeks)	Change in liver enzyme alkaline phosphatase to below 345 unit/L. General outcomes of participants are reported due to the enrollment of very few patients so that we have too small a cohort with which to assess achievement of our stipulated outcomes.
Number of Participants With a Change in Liver Enzyme Gamma-glutamyltransferase (GGT)	Completion of Therapy (time frame from 1-14 weeks)	Change in Liver Enzyme Gamma-glutamyltransferase (GGT) to below 15 unit/L. General outcomes of participants are reported due to the enrollment of very few patients so that we have too small a cohort with which to assess achievement of our stipulated outcomes.
Number of Participants With a Change in Triglycerides	Completion of Therapy (time frame from 1-14 weeks)	Change in Triglycerides to below 119 mg/dL. General outcomes of participants are reported due to the enrollment of very few patients so that we have too small a cohort with which to assess achievement of our stipulated outcomes.