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Compassionate Use of Omegaven® in the Treatment of Parenteral Nutrition Associated Hepatic Injury

Primary Purpose

Total Parenteral Nutrition-induced Cholestasis

Status

Approved for marketing

Phase

Locations

United States

Study Type

Expanded Access

Intervention

Omegaven®

About this trial

This is an expanded access trial for Total Parenteral Nutrition-induced Cholestasis focused on measuring TPN Cholestasis

Eligibility Criteria

2 Months - 10 Years (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Patients must be 2 months -10 years of age.
Patients who are PN dependent (unable to meet nutritional needs solely by enteral nutrition) and are expected to require PN for at least another 30 days
Patients considered eligible for study participation must have parenteral nutrition associated liver disease (PNALD) as defined by a direct bilirubin of >2.0mg/dL. Other causes of liver disease should be excluded. A liver biopsy is not necessary for treatment.
2 consecutive direct bilirubin test results >2.0mg/dL
The patient must have failed standard therapies to prevent the progression of liver disease such as surgical treatment, cyclic TPN, avoiding overfeeding, reduction/removal of copper and manganese from TPN, advancement of enteral feeding, and use of ursodiol (Actigall).
Subjects who are currently under treatment for PNALD with Compassionate Use Omegaven and have a direct Bilirubin of< 2, but who remain TPN dependent and require continued therapy with Omegaven.

Exclusion Criteria:

Other known causes of chronic liver disease (hepatitis C, cystic fibrosis, biliary atresia and alpha 1 anti-trypsin deficiency)
Enrollment in any other clinical trial involving an investigational agent (unless approved by the designated physicians on the multidisciplinary team)
The parent, guardian, or child is unwilling to provide consent or assent
Allergy to any fish product, egg protein, and/or previous allergy to Omegaven
Active coagulopathies characterized by on-going bleeding or by a requirement for clotting factor replacement such as fresh frozen plasma or cryoprecipitate to maintain homeostasis
Impaired lipid metabolism or severe hyperlipidemia with or without pancreatitis
Unstable diabetes mellitus
Stroke/embolism
Collapse and shock
Undefined coma status
Active infection at time of initiation of Omegaven® up until such time as child is afebrile with stable vital signs and one negative 48 hour culture.

Sites / Locations

Children's Hospital of Pittsburgh of UPMC

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

NCT ID

NCT01412359

First Posted

August 2, 2011

Last Updated

April 19, 2019

Sponsor

Jeffrey Rudolph

Collaborators

University of Pittsburgh

1. Study Identification

Unique Protocol Identification Number

NCT01412359

Brief Title

Compassionate Use of Omegaven® in the Treatment of Parenteral Nutrition Associated Hepatic Injury

Official Title

Compassionate Use of Omegaven® in the Treatment of Parenteral Nutrition Associated Hepatic Injury

Study Type

Expanded Access

2. Study Status

Record Verification Date

April 2019

Overall Recruitment Status

Approved for marketing

Study Start Date

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Primary Completion Date

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Study Completion Date

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3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor-Investigator

Name of the Sponsor

Jeffrey Rudolph

Collaborators

University of Pittsburgh

4. Oversight

5. Study Description

Brief Summary

The overall purpose of this study is to determine if replacing standard soybean oil based fat emulsions with Omegaven®, a fish oil based fat emulsion, can reverse or prevent the progression of parenteral nutrition associated cholestasis. It is a compassionate use protocol for patients who already have significant cholestasis related to parenteral nutrition.

Detailed Description

In the United States, patients dependent upon parenteral nutrition (PN) receive parenteral fat emulsions composed of soybean oils. Lipids are necessary in PN dependent patients due to their high caloric value and essential fatty acid content. Phytosterols such as those contained in soybean oil based fat emulsions are thought to have a deleterious effect on biliary secretion and they have been implicated in predisposing patients to PN associated liver disease. Children requiring prolonged courses of PN are at risk for developing PN associated liver disease. The investigators hypothesize that although soybean oil based fat emulsions prevent fatty acid deficiency, they are not cleared in a manner similar to enteral chylomicrons and therefore accumulate in the liver resulting in steatotic liver injury. Animal studies have shown that fish oil based IV fat emulsions(IFE)which are high in eicosapentaenic and docosahexaenoic acid, reduce impairment of bile flow seen in cholestasis caused by conventional fat emulsions. Omegaven® is a fish oil based IV fat emulsion. The investigators hypothesize that administering Omegaven® in place of conventional soybean fat emulsions may reverse or prevent the progression of PN associated cholestasis and thus allow the patient to be maintained on adequate PN until he/she is able to ingest adequate nutrition enterally. Primary outcome measures to be addressed include the normalization of liver enzymes including bilirubin upon the start of Omegaven® and continued growth on Omegaven®. Safety measures will include the prevalence of essential fatty acid deficiency, hypertriglyceridemia, and bleeding disorders.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Total Parenteral Nutrition-induced Cholestasis

Keywords

TPN Cholestasis

7. Study Design

8. Arms, Groups, and Interventions

Intervention Type

Drug

Intervention Name(s)

Omegaven®

Other Intervention Name(s)

Intavenous fish oil emulsion

Intervention Description

Omegaven® 10% fat emulsion 1g/kg/day to infuse via IV over a period of 12 -24 hours every day until patient no longer requires intravenous lipid emulsion, is determined ineffective, or the patient stops participating in the study for any reason.

10. Eligibility

Sex

All

Minimum Age & Unit of Time

2 Months

Maximum Age & Unit of Time

10 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Patients must be 2 months -10 years of age. Patients who are PN dependent (unable to meet nutritional needs solely by enteral nutrition) and are expected to require PN for at least another 30 days Patients considered eligible for study participation must have parenteral nutrition associated liver disease (PNALD) as defined by a direct bilirubin of >2.0mg/dL. Other causes of liver disease should be excluded. A liver biopsy is not necessary for treatment. 2 consecutive direct bilirubin test results >2.0mg/dL The patient must have failed standard therapies to prevent the progression of liver disease such as surgical treatment, cyclic TPN, avoiding overfeeding, reduction/removal of copper and manganese from TPN, advancement of enteral feeding, and use of ursodiol (Actigall). Subjects who are currently under treatment for PNALD with Compassionate Use Omegaven and have a direct Bilirubin of< 2, but who remain TPN dependent and require continued therapy with Omegaven. Exclusion Criteria: Other known causes of chronic liver disease (hepatitis C, cystic fibrosis, biliary atresia and alpha 1 anti-trypsin deficiency) Enrollment in any other clinical trial involving an investigational agent (unless approved by the designated physicians on the multidisciplinary team) The parent, guardian, or child is unwilling to provide consent or assent Allergy to any fish product, egg protein, and/or previous allergy to Omegaven Active coagulopathies characterized by on-going bleeding or by a requirement for clotting factor replacement such as fresh frozen plasma or cryoprecipitate to maintain homeostasis Impaired lipid metabolism or severe hyperlipidemia with or without pancreatitis Unstable diabetes mellitus Stroke/embolism Collapse and shock Undefined coma status Active infection at time of initiation of Omegaven® up until such time as child is afebrile with stable vital signs and one negative 48 hour culture.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Jeffrey A Rudolph, MD

Organizational Affiliation

University of Pittsburgh

Official's Role

Principal Investigator

Facility Information:

Facility Name

Children's Hospital of Pittsburgh of UPMC

City

Pittsburgh

State/Province

Pennsylvania

ZIP/Postal Code

15224

Country

United States

12. IPD Sharing Statement

Learn more about this trial

Compassionate Use of Omegaven® in the Treatment of Parenteral Nutrition Associated Hepatic Injury

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