Back to resultsCompassionate Use of Omegaven IV Fat Emulsion
Primary Purpose
Cholestasis, Liver Disease
Status
No longer available
Phase
Locations
United States
Study Type
Expanded Access
Intervention
Omegaven IV
Sponsored by
About this trial
This is an expanded access trial for Cholestasis focused on measuring Cholestasis, Parenteral Nutrition, Liver disease
Eligibility Criteria
Inclusion Criteria:
- subjects will be infants and children from birth to 5 years of age
- diagnosis of parenteral nutrition associated liver disease (PNALD) (defined as two consecutive direct bilirubin levels of 2 mg/dl or more) in a parenteral nutrition-dependent infant or child.
- subject must have utilized standard therapies to prevent the progression of the cholestasis including reduction/removal of copper and manganese from daily PN, trial of enteral feedings if possible, and the use of ursodiol and/or phenobarbital.
Exclusion Criteria:
- patients are excluded if they have other documented causes of chronic liver disease (i.e.: Hepatitis C, cystic fibrosis, biliary atresia, alpha-1-anti-trypsin deficiency),
- or already have signs of proven severe advanced liver disease including cirrhosis on biopsy, varices, ascites.
Additional exclusion criteria:
- an 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
- severe hyperlipidemia with or without pancreatitis
- unstable diabetes mellitus
- hyperglycemia
- stroke, embolism
- collapse and shock
- recent myocardial infarction (MI)
- cholestasis due to any reason other than parenteral nutrition associated cholestasis (PNAC)
- active new infection at time of initiation of Omegaven
- hemodynamic instability
- patient cannot be enrolled in any other clinical trial involving an investigational agent (unless approved by the designated physicians on the multidisciplinary team)
Sites / Locations
- Staten Island University Hospital
Outcomes
Primary Outcome Measures
Secondary Outcome Measures
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT02477345
Brief Title
Compassionate Use of Omegaven IV Fat Emulsion
Official Title
Compassionate Use of Omegaven IV Fat Emulsion
Study Type
Expanded Access
2. Study Status
Record Verification Date
December 2017
Overall Recruitment Status
No longer available
Study Start Date
undefined (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
undefined (undefined)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Northwell Health
4. Oversight
5. Study Description
Brief Summary
This protocol involves the compassionate use of intravenous fish oil infusion, Omegaven. The protocol involves infants and children with parenteral nutrition-associated liver disease to enable the reversal of elevated serum liver enzymes and direct bilirubin (cholestasis).
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. Intravenous lipid emulsions have been implicated in predisposing patients to PN associated liver disease. Phytosterols such as those contained in soybean oils are thought to have a deleterious effect on biliary secretion. Accumulation of lipids in the hepatic Kupffer cells may further impair liver function.
Omega 6 fatty acid emulsions prevent fatty acid emulsions prevent fatty acid deficiency, it is thought that they are not cleared in a manner similar to enteral chylomicrons and therefore accumulate in the liver and resulting in steatotic liver injury (neonatal cholestasis). It is hypothesized that a fat emulsion comprised of omega 3 fatty acids (i.e.: fish oil), such as de novo lipogenesis, the reduction of arachidonic acid-derived inflammatory mediators, prevention of essential fatty acid deficiency through the presence of small amounts of arachidonic acid, and improved clearance of lipids from the serum. Animal studies have shown that IV fat emulsions such as fish oil that are high in eicosapentaenic and docosahexaenoic acid reduce impairment of bile flow which is seen in cholestasis caused by conventional fat emulsions. Furthermore, intravenous omega-e fatty acids are well tolerated and might reduce the hepatic dysfunction. By administering Omegaven in place of conventional phytosterol/soybean fat emulsion, the progression of PN-associated cholestasis can be prevented or reversed.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cholestasis, Liver Disease
Keywords
Cholestasis, Parenteral Nutrition, Liver disease
7. Study Design
8. Arms, Groups, and Interventions
Intervention Type
Drug
Intervention Name(s)
Omegaven IV
Other Intervention Name(s)
Fat Emulsion
Intervention Description
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.
10. Eligibility
Sex
All
Minimum Age & Unit of Time
1 Day
Maximum Age & Unit of Time
5 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
subjects will be infants and children from birth to 5 years of age
diagnosis of parenteral nutrition associated liver disease (PNALD) (defined as two consecutive direct bilirubin levels of 2 mg/dl or more) in a parenteral nutrition-dependent infant or child.
subject must have utilized standard therapies to prevent the progression of the cholestasis including reduction/removal of copper and manganese from daily PN, trial of enteral feedings if possible, and the use of ursodiol and/or phenobarbital.
Exclusion Criteria:
patients are excluded if they have other documented causes of chronic liver disease (i.e.: Hepatitis C, cystic fibrosis, biliary atresia, alpha-1-anti-trypsin deficiency),
or already have signs of proven severe advanced liver disease including cirrhosis on biopsy, varices, ascites.
Additional exclusion criteria:
an 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
severe hyperlipidemia with or without pancreatitis
unstable diabetes mellitus
hyperglycemia
stroke, embolism
collapse and shock
recent myocardial infarction (MI)
cholestasis due to any reason other than parenteral nutrition associated cholestasis (PNAC)
active new infection at time of initiation of Omegaven
hemodynamic instability
patient cannot be enrolled in any other clinical trial involving an investigational agent (unless approved by the designated physicians on the multidisciplinary team)
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Jonathan Blau, MD
Organizational Affiliation
Staten Island University Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
Staten Island University Hospital
City
Staten Island
State/Province
New York
ZIP/Postal Code
10305
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Compassionate Use of Omegaven IV Fat Emulsion
We'll reach out to this number within 24 hrs