- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02328768
Compassionate Use of Omegaven® for the Treatment of Intestinal Failure Associated Liver Disease in Children
January 4, 2024 updated by: University of Nebraska
Compassionate Use of an Intravenous Fish Oil Lipid Emulsion (Omegaven®) for the Treatment of Intestinal Failure Associated Liver Disease in Children
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 liver disease.
It is a compassionate use protocol for patients who already have significant liver disease related to parenteral nutrition.
Study Overview
Status
Approved for marketing
Intervention / Treatment
Detailed Description
Parenteral Nutrition (PN) is a potentially life-saving intervention for children with short bowel syndrome and intestinal failure.
Many common neonatal surgical diseases including Necrotizing enterocolitis (NEC), intestinal atresias, and gastroschisis can cause intestinal failure.
The recovery from these illnesses often involves prolonged periods of parenteral nutrition.
Intestinal Failure Associated Liver Disease (IFALD) is the most prevalent complication of long term parenteral nutrition in children, affecting up to 2/3 of children with short bowel syndrome.
Parenteral lipids are an important source of calories in children, and provide essential fatty acids.
Development of IFALD is a multifactorial process.
Phytosterols contained in soybean based lipid emulsions have been shown to predispose animals to IFALD.
Previous studies in children have shown that dosing the soybean based parenteral lipid emulsion at doses greater than 1g/kg/day may contribute to the development of IFALD.
It is currently our practice to limit the lipid dose in children at risk of development of IFALD to 1g/kg/day.
Despite this, some patients will still develop biochemical evidence of cholestasis and IFALD.
Previous studies in humans have shown that children with IFALD who were administered the intravenous fish oil lipid emulsion Omegaven® reduced their serum direct bilirubin levels.
This may be due to a reduction in the amount of arachidonic acide derived inflammatory mediators.
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.
Study Type
Expanded Access
Expanded Access Type
- Individual Patients
- Treatment IND/Protocol
Phase
- Phase 2
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Nebraska
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Omaha, Nebraska, United States, 68114
- Children's Hospital & Medical Center
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
No older than 16 years (Child, Adult)
Accepts Healthy Volunteers
No
Description
Inclusion Criteria:
- Parenteral nutrition (PN) dependent (unable to meet nutritional needs solely by enteral nutrition) and expected to require PN for at least another 30 days.
- Parenteral nutrition associated liver disease (PNALD) as defined as a direct bilirubin ≥ 2mg/dL or by histology and/or currently on Omegaven through another protocol.
- Other causes of liver disease have been excluded. A liver biopsy is not necessary for treatment.
- Exhaustion of standard therapies to prevent the progression of the liver disease including surgical treatment, cyclic PN, avoiding overfeeding, reduction/removal of copper and manganese form PN, advancement of enteral feeding, and the use of ursodiol.
Exclusion Criteria:
- Pregnancy
- Other causes of liver disease
- Enrollment in any other clinical trial involving an investigational agent (unless approved by the designated physicians on the multidisciplinary team)
- The parent/guardian or subject is unwilling to provide consent and assent
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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Experimental: Omegaven®
Omegaven® 10% fat emulsion 1g/kg/day to infuse via IV over a period of 8-24 hours every day until the patient no longer requires intravenous lipid emulsion, is determined ineffective, or the patient stops participating in the study for any reason.
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Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Primary efficacy outcome will be normalized direct bilirubin, defined as three consecutive direct bilirubin results < 2mg/dL or a direct bilirubin < 2mg/dL and weaned from total parenteral nutrition (TPN).
Time Frame: 1 year
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Bilirubin levels will be monitored according to the standard of care for TPN dependent patients, usually weekly, for the duration of their participation in the study protocol.
Duration of participation cannot be determined in advance.
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1 year
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Collaborators
Investigators
- Principal Investigator: Brian A Jones, MD, University of Nebraska Medical Center/Children's Hospital & Medical Center
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
April 3, 2015
Primary Completion
November 10, 2017
Study Completion
September 5, 2018
Study Registration Dates
First Submitted
December 19, 2014
First Submitted That Met QC Criteria
December 23, 2014
First Posted (Estimated)
December 31, 2014
Study Record Updates
Last Update Posted (Actual)
January 9, 2024
Last Update Submitted That Met QC Criteria
January 4, 2024
Last Verified
January 1, 2024
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- 0018-15-FB
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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