Compassionate Use of Omegaven IV Fat Emulsion

NCT ID: NCT00816348

Last Updated: 2017-03-10

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE2
• Total Enrollment: 15 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2008-12-31
• Study Completion Date: 2014-02-28

Brief Summary

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).

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.

Conditions

Cholestasis; Total Parenteral Nutrition-induced Cholestasis

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Omegaven

All subjects will receive Omegaven

Group Type: OTHER

Omegaven

Intervention Type: DRUG

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.

Interventions

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.

Intervention Type: DRUG

Eligibility Criteria

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).

• Minimum Eligible Age: 2 Weeks
• Maximum Eligible Age: 5 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Cindy Haller

OTHER

Sponsor Role: lead

Responsible Party

Cindy Haller

Physician

Responsibility Role: SPONSOR_INVESTIGATOR

Principal Investigators

Cindy Haller, MD

Role: PRINCIPAL_INVESTIGATOR

Cohen Children's Medical Center of New York

Locations

Cohen Children's Medical Cenetr of New York at North Shore

Manhasset, New York, United States

Cohen Children's Medical Center of New York

New Hyde Park, New York, United States

Countries

United States

Other Identifiers

08-204

Identifier Type: -

Identifier Source: org_study_id