Back to resultsCompassionate Use of Omegaven in the Treatment of Parenteral Nutrition Induced Hepatic Injury
Primary Purpose
Liver Disease, Impaired Liver Function, Parenteral Nutrition Associated Liver Disease
Status
Completed
Phase
Not Applicable
Locations
United States
Study Type
Interventional
Intervention
Omegaven
Sponsored by
About this trial
This is an interventional supportive care trial for Liver Disease focused on measuring TPN, Liver Function, parenteral nutritional fat emulsions
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
Sites / Locations
- Children's Medical Center of Dallas
Arms of the Study
Arm 1
Arm Type
Other
Arm Label
Compassionate use
Arm Description
This lipid preparation is only being used for compassionate use.
Outcomes
Primary Outcome Measures
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.
Secondary Outcome Measures
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.
Full Information
NCT ID
NCT02010034
First Posted
August 19, 2013
Last Updated
February 5, 2019
Sponsor
Children's Medical Center Dallas
1. Study Identification
Unique Protocol Identification Number
NCT02010034
Brief Title
Compassionate Use of Omegaven in the Treatment of Parenteral Nutrition Induced Hepatic Injury
Official Title
Compassionate Use of Omegaven in the Treatment of Parenteral Nutrition Induced Hepatic Injury
Study Type
Interventional
2. Study Status
Record Verification Date
February 2019
Overall Recruitment Status
Completed
Study Start Date
August 2013 (undefined)
Primary Completion Date
February 5, 2019 (Actual)
Study Completion Date
February 5, 2019 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Children's Medical Center Dallas
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
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.
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Liver Disease, Impaired Liver Function, Parenteral Nutrition Associated Liver Disease
Keywords
TPN, Liver Function, parenteral nutritional fat emulsions
7. Study Design
Primary Purpose
Supportive Care
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
15 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Compassionate use
Arm Type
Other
Arm Description
This lipid preparation is only being used for compassionate use.
Intervention Type
Drug
Intervention Name(s)
Omegaven
Intervention Description
Omegaven will be used in place of soy oil Intralipid
Primary Outcome Measure Information:
Title
Bilirubin in mg/dL
Description
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.
Time Frame
12 weeks
Secondary Outcome Measure Information:
Title
Number of participants with adverse events
Description
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.
Time Frame
12 weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
2 Months
Maximum Age & Unit of Time
10 Years
Accepts Healthy Volunteers
No
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
Nandini Channabasappa, MD
Organizational Affiliation
Childrens Health
Official's Role
Principal Investigator
Facility Information:
Facility Name
Children's Medical Center of Dallas
City
Dallas
State/Province
Texas
ZIP/Postal Code
75235
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Compassionate Use of Omegaven in the Treatment of Parenteral Nutrition Induced Hepatic Injury
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