CholestasisNCT02477345
Compassionate Use of Omegaven IV Fat Emulsion
A study testing Omegaven IV in people with Cholestasis and Liver Disease, run by Northwell Health at 1 site in United States.
Trial 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). Read more about this study
- Who is it for?
- Ages 1 day to 5 years
- Conditions the study is for
- Cholestasis, Liver Disease
- What is being tested?
- Omegaven IV
- Type of treatment
- Injection or infusion
- How many people
- Not given
- Where
- 1 siteUnited States
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 day to 5 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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
Show all 15Show fewer
- 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)
Trial locations and contacts
1 site in 1 country.
United States1 site
Staten Island University Hospital
Staten Island, New York, 10305
About this study
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).
Read the 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.
- Conditions
- Cholestasis, Liver Disease
- Being tested
- Omegaven IV
- Keywords
- Cholestasis, Parenteral Nutrition, Liver disease
- Medical subject headings
- Cholestasis, Liver Diseases, Hyperphagia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Omegaven IVDrug
Also called Fat Emulsion
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.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jun 2015 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for CholestasisStudies of the same condition, open and under way.
- More trials run by Northwell HealthThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- Northwell Health
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Jonathan Blau, Northwell Health
- Investigators
- Jonathan Blau, MD, Principal investigator, Staten Island University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02477345
- Sponsor's protocol ID
- SIUH12-025
- Status last verified
- Dec 2017
- First submitted
- 27 Nov 2013
- Last updated
- 8 Dec 2017
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Dec 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Compassionate Use of Omegaven IV Fat Emulsion
- ClinicalTrials.gov ID
- NCT02477345
- Acronym / sponsor ID
- SIUH12-025
- Conditions
- Cholestasis, Liver Disease
- Keywords
- Cholestasis, Parenteral Nutrition, Liver disease
- Study type
- Expanded access
- Recruitment status
- No Longer Available
- Lead sponsor
- Northwell HealthUniversity, hospital or other
- First posted
- 22 Jun 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
Who may qualify
- Ages
- 1 day to 5 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (15 listed)
- 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.
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Omegaven IVAlso: Fat EmulsionOmegaven 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. | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Dec 2017.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jonathan Blau, MD principal investigator
Staten Island University Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Omegaven IV in people with Cholestasis and Liver Disease, run by Northwell Health at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 8 Dec 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Dec 2017.