Compassionate Use of Omegaven IV Fat Emulsion

This is a compassionate use protocol to use intravenous fish oil infusion, Omegaven®, to infants and children with parenteral nutrition-associated liver disease to enable reversal of elevated serum liver enzymes and direct bilirubin (cholestasis).

Study Overview

• Status: Terminated
• Conditions: Cholestasis; Total Parenteral Nutrition-induced Cholestasis
• Intervention / Treatment: Drug: Omegaven

Detailed Description

Intravenous lipids are necessary in PN (parenteral nutrition) dependent patients due to their high caloric value and essential fatty acid content. However, parenteral fat emulsions composed of soybean oils (omega 6 fatty acids) have been implicated in predisposing patients to PN associated liver disease.

It is hypothesized that by administering Omegaven®, comprised of fish oil (omega 3 fatty acids) in place of conventional soybean fat emulsion, the progression of PN-associated cholestasis may be prevented or reversed.

• Study Type: Interventional
• Enrollment (Actual): 15
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • New York
    • Manhasset, New York, United States, 11030
      • Cohen Children's Medical Cenetr of New York at North Shore
    • New Hyde Park, New York, United States, 11040
      • Cohen Children's Medical Center of New York

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 weeks to 5 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Two consecutive direct bilirubin levels of 2 mg/dl or more in a parenteral nutrition dependent infant or child (unable to meet nutritional needs solely by enteral nutrition)
• Other causes of liver disease have been excluded. A liver biopsy is not necessary for treatment.
• The patient must have utilized standard therapies to prevent the progression of the liver disease including reduction/removal of copper and manganese from daily PN, trial of enteral feeding if possible, and the use of ursodiol (i.e., Actigall®).

Exclusion Criteria:

• Documented causes of chronic liver disease other than parenteral nutrition associated liver disease
• Proven severe advanced liver disease including cirrhosis on biopsy, varices, ascites.
• An allergy to any seafood product, egg protein, and/or previous allergy to Omegaven®
• Active coagulopathy characterized by ongoing bleeding or by a requirement for clotting factor replacement (e.g. fresh frozen plasma or cryoprecipitate) to maintain homeostasis
• Impaired lipid metabolism or severe hyperlipidemia with or without pancreatitis
• Unstable diabetes mellitus or hyperglycemia
• Stroke, embolism, collapse and shock, recent MI
• Cholestasis due to any reason other than parenteral associated liver disease
• Active new infection at time of initiation of Omegaven®
• Hemodynamic instability
• The patient may not be enrolled in any other clinical trial involving an investigational agent (unless approved by the designated physicians on the multidisciplinary team).

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Other: Omegaven; All subjects will receive Omegaven

Drug: Omegaven; Omegaven® will be initiated at a dose of 0.5 gram/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 either a central or peripheral catheter alone or in conjunction with parenteral nutrition. Omegaven® will continue until weaned from PN. Monotherapy with Omegaven® can continue as an additional source of calories after the dextrose/protein portion of PN is discontinued. Omegaven may be restarted within seven days of discontinuing therapy. After seven days, and meeting inclusion criteria, Omegaven can resume at the initial dose of 0.5 grams/kg/day, advancing to 1 gm/kg/day.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Level of Bilirubin	measurement of bilirubin level weekly. Available data reported.	week 2, 3,4,and 8

Collaborators and Investigators

• Sponsor: Cindy Haller
• Investigators: Principal Investigator: Cindy Haller, MD, Cohen Children's Medical Center of New York

Study record dates

Study Major Dates

• Study Start: December 1, 2008
• Primary Completion (Actual): February 1, 2014
• Study Completion (Actual): February 1, 2014

Study Registration Dates

• First Submitted: December 31, 2008
• First Submitted That Met QC Criteria: December 31, 2008
• First Posted (Estimate): January 1, 2009

Study Record Updates

• Last Update Posted (Actual): March 10, 2017
• Last Update Submitted That Met QC Criteria: January 30, 2017
• Last Verified: January 1, 2017

More Information

Terms related to this study

• Keywords: Cholestasis; Parenteral Nutrition Associated Liver Disease
• Additional Relevant MeSH Terms: Digestive System Diseases; Biliary Tract Diseases; Bile Duct Diseases; Cholestasis
• Other Study ID Numbers: 08-204