Compassionate Use of Omegaven in the Treatment of Parenteral Nutrition Induced Hepatic Injury

This study is being done to determine if an investigational study drug called Omegaven can help to improve liver disease thought to be caused by Total Parenteral Nutrition (TPN). TPN is intravenous feeding (IV feeding tube) that provides patients the nutrients when they are unable to drink fluid or eat food by mouth.

Study Overview

• Status: Completed
• Conditions: Liver Disease; Parenteral Nutrition Associated Liver Disease; Impaired Liver Function
• Intervention / Treatment: Drug: Omegaven

Detailed Description

PAtients with direct bilirubin >2gm/dL and short bowel syndrome are eligible for enrollment. Omegaven at 1gm/kg/day will be administered via central venous line instead of standard lipid preparations.

• Study Type: Interventional
• Enrollment (Anticipated): 15
• Phase: Not Applicable

Contacts and Locations

Study Locations

• United States
  • Texas
    • Dallas, Texas, United States, 75235
      • Children's Medical Center of Dallas

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 months to 10 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. Patients must be 2 months -10 years of age
2. 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
3. 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
4. 2 consecutive direct bilirubin test results >2.0mg/dl
5. 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)
6. 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:

1. Other known causes of chronic liver disease (hepatitis C, cystic fibrosis, biliary atresia and alpha 1 anti-trypsin deficiency)
2. Enrollment in any other clinical trial involving an investigational agent (unless approved by the designated physicians on the multidisciplinary team)
3. The parent, guardian, or child is unwilling to provide consent or assent
4. Allergy to any fish product, egg protein, and/or previous allergy to Omegaven
5. 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
6. Impaired lipid metabolism or severe hyperlipidemia with or without pancreatitis
7. Unstable diabetes mellitus
8. Stroke/embolism
9. Collapse and shock
10. Undefined coma status
11. 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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Supportive Care
• 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: Compassionate use; This lipid preparation is only being used for compassionate use.	Drug: Omegaven; Omegaven will be used in place of soy oil Intralipid

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Bilirubin in mg/dL	PN associated liver disease can be reversed or its progression halted by using a parenteral fat emulsion prepared from fish oil as measured by normalization of serum levels of hepatic enzymes and bilirubin 1. after reaching bilirubin levels >2mg/dl, subjects receiving Omegaven will reach a bilirubin level < 2mg/dL faster that patients receiving conventional fat emulsions.	12 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of participants with adverse events	a. After starting Omegaven on PN, the rate of fatty acid deficiencies and imbalances will be as low as before Omegaven. Also, the rate of triglyceride events >400mg/dL with Omegaven will be similar to that seen with PN administration with soy oil Intralipid. B. PN containing Omegaven will be safe for patients with respect to the risk of unexpected bleeding, coagulopathies, and other adverse events.	12 weeks

Collaborators and Investigators

• Sponsor: Children's Medical Center Dallas
• Investigators: Principal Investigator: Nandini Channabasappa, MD, Childrens Health

Study record dates

Study Major Dates

• Study Start: August 1, 2013
• Primary Completion (Actual): February 5, 2019
• Study Completion (Actual): February 5, 2019

Study Registration Dates

• First Submitted: August 19, 2013
• First Submitted That Met QC Criteria: December 11, 2013
• First Posted (Estimate): December 12, 2013

Study Record Updates

• Last Update Posted (Actual): February 6, 2019
• Last Update Submitted That Met QC Criteria: February 5, 2019
• Last Verified: February 1, 2019

More Information

Terms related to this study

• Keywords: TPN; Liver Function; parenteral nutritional fat emulsions
• Additional Relevant MeSH Terms: Digestive System Diseases; Liver Diseases
• Other Study ID Numbers: STU 112010-159