CholestasisOmegavenNCT01297933
Compassionate Use of Omegaven for Pediatric Patients With Parenteral Nutrition Associated Liver Disease
A study testing Omegaven in people with Cholestasis and Short Bowel Syndrome, run by Children's Hospital of Philadelphia at 1 site in United States.
Trial summary
A compassionate use protocol to provide Omegaven to pediatric patients with parenteral nutrition (PN) dependence and parenteral nutrition associated liver disease (PNALD). Read more about this study
- Who is it for?
- Ages Up to 21 years
- Conditions the study is for
- Cholestasis, Short Bowel Syndrome
- What is being tested?
- Omegaven
- 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 Up to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- PN dependence due to congenital or acquired gastrointestinal disease
- Predicted PN requirement for at least an additional 30 days
- Parenteral nutrition associated liver disease (PNALD), defined as two conjugated bilirubin levels >= 2 mg/dL at least one week apart, must be obtained to demonstrate persistence of PNALD
- Failure to respond to standard therapies which may include cycling PN, reduction in the dose of soybean derived IFE, attempts to advance enteral feeds, ursodiol, metronidazole, and avoidance of excessive caloric provision
- signed parent or legal guardian informed consent
Who cannot join
- Acute treatable infection (e.g. urinary tract infection, sepsis)
- Known allergy to egg or fish protein
- Contraindications to Omegaven
- Pregnancy
- Serum triglyceride level greater than 400 mg/dL at baseline
- History of severe hemolytic disorders or INR greater than 1.5 at baseline (INR cutoff of greater than 2 for infants less than 1 week of age)
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
About this study
Official title: Intravenous Omega-3 Fatty Acid (Omegaven) for Pediatric Patients With Total Parenteral Nutrition Associated Liver Dysfunction
A compassionate use protocol to provide Omegaven to pediatric patients with parenteral nutrition (PN) dependence and parenteral nutrition associated liver disease (PNALD).
Read the detailed description
Omegaven is an intravenous fat emulsion (IFE) comprised of omega-3 fatty acids derived from fish oil. It will be used in an open label compassionate use treatment protocol, as an alternative to soybean oil (omega-6), as the sole IFE source of parenteral nutrition in an effort to reduce and/or reverse parenteral nutrition associated liver disease. The study will evaluate the safety and efficacy of Omegaven use in pediatric patients with PN dependence and PNALD.
- Conditions
- Cholestasis, Short Bowel Syndrome
- Being tested
- Omegaven
- Keywords
- parenteral nutrition associated liver disease, cholestasis, short bowel syndrome, Omegaven
- Medical subject headings
- Cholestasis, Short Bowel Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
OmegavenDrug
10% Omegaven 1g/kg/day, Intravenous by continuous infusion in conjunction with parenteral nutrition, until the patient no longer requires parenteral nutrition or until participation in the study is terminated.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Feb 2011 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (6)Papers the sponsor has linked to this study.
- Other trials for CholestasisStudies of the same condition, open and under way.
- More trials run by Children's Hospital of PhiladelphiaThe 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
- Children's Hospital of Philadelphia
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Maria Mascarenhas, Children's Hospital of Philadelphia
- Investigators
- Maria Mascarenhas, MBBS, Principal investigator, Children's Hospital of Philadelphia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01297933
- Sponsor's protocol ID
- 10-007681
- Acronym
- Omegaven
- Status last verified
- Apr 2019
- First submitted
- 15 Feb 2011
- Last updated
- 12 Apr 2019
- Results posted
- No
Publications
Alwayn IP, Gura K, Nose V, Zausche B, Javid P, Garza J, Verbesey J, Voss S, Ollero M, Andersson C, Bistrian B, Folkman J, Puder M. Omega-3 fatty acid supplementation prevents hepatic steatosis in a murine model of nonalcoholic fatty liver disease. Pediatr Res. 2005 Mar;57(3):445-52. doi: 10.1203/01.PDR.0000153672.43030.75. Epub 2005 Jan 19.
PubMed 15659701Diamond IR, Sterescu A, Pencharz PB, Kim JH, Wales PW. Changing the paradigm: omegaven for the treatment of liver failure in pediatric short bowel syndrome. J Pediatr Gastroenterol Nutr. 2009 Feb;48(2):209-15. doi: 10.1097/MPG.0b013e318182c8f6.
PubMed 19179884Gura KM, Parsons SK, Bechard LJ, Henderson T, Dorsey M, Phipatanakul W, Duggan C, Puder M, Lenders C. Use of a fish oil-based lipid emulsion to treat essential fatty acid deficiency in a soy allergic patient receiving parenteral nutrition. Clin Nutr. 2005 Oct;24(5):839-47. doi: 10.1016/j.clnu.2005.05.020.
PubMed 16029913Gura KM, Lee S, Valim C, Zhou J, Kim S, Modi BP, Arsenault DA, Strijbosch RA, Lopes S, Duggan C, Puder M. Safety and efficacy of a fish-oil-based fat emulsion in the treatment of parenteral nutrition-associated liver disease. Pediatrics. 2008 Mar;121(3):e678-86. doi: 10.1542/peds.2007-2248.
PubMed 18310188Puder M, Valim C, Meisel JA, Le HD, de Meijer VE, Robinson EM, Zhou J, Duggan C, Gura KM. Parenteral fish oil improves outcomes in patients with parenteral nutrition-associated liver injury. Ann Surg. 2009 Sep;250(3):395-402. doi: 10.1097/SLA.0b013e3181b36657.
PubMed 19661785Van Aerde JE, Duerksen DR, Gramlich L, Meddings JB, Chan G, Thomson AB, Clandinin MT. Intravenous fish oil emulsion attenuates total parenteral nutrition-induced cholestasis in newborn piglets. Pediatr Res. 1999 Feb;45(2):202-8. doi: 10.1203/00006450-199902000-00008.
PubMed 10022591
Study at a glance
From the registry record, last updated by the sponsor on 12 Apr 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Intravenous Omega-3 Fatty Acid (Omegaven) for Pediatric Patients With Total Parenteral Nutrition Associated Liver Dysfunction
- ClinicalTrials.gov ID
- NCT01297933
- Acronym / sponsor ID
- Omegaven / 10-007681
- Conditions
- Cholestasis, Short Bowel Syndrome
- Keywords
- parenteral nutrition associated liver disease, cholestasis, short bowel syndrome, Omegaven
- Study type
- Expanded access
- Recruitment status
- Approved For Marketing
- Lead sponsor
- Children's Hospital of PhiladelphiaUniversity, hospital or other
- First posted
- 17 Feb 2011 (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.
A compassionate use protocol to provide Omegaven to pediatric patients with parenteral nutrition (PN) dependence and parenteral nutrition associated liver disease (PNALD).
Detailed description
Omegaven is an intravenous fat emulsion (IFE) comprised of omega-3 fatty acids derived from fish oil. It will be used in an open label compassionate use treatment protocol, as an alternative to soybean oil (omega-6), as the sole IFE source of parenteral nutrition in an effort to reduce and/or reverse parenteral nutrition associated liver disease. The study will evaluate the safety and efficacy of Omegaven use in pediatric patients with PN dependence and PNALD.
Study design
Who may qualify
- Ages
- Up to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- PN dependence due to congenital or acquired gastrointestinal disease
- Predicted PN requirement for at least an additional 30 days
- Parenteral nutrition associated liver disease (PNALD), defined as two conjugated bilirubin levels >= 2 mg/dL at least one week apart, must be obtained to demonstrate persistence of PNALD
- Failure to respond to standard therapies which may include cycling PN, reduction in the dose of soybean derived IFE, attempts to advance enteral feeds, ursodiol, metronidazole, and avoidance of excessive caloric provision
- signed parent or legal guardian informed consent
Key exclusion criteria (6 listed)
- Acute treatable infection (e.g. urinary tract infection, sepsis)
- Known allergy to egg or fish protein
- Contraindications to Omegaven
- Pregnancy
- Serum triglyceride level greater than 400 mg/dL at baseline
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 |
|---|---|---|
| Omegaven10% Omegaven 1g/kg/day, Intravenous by continuous infusion in conjunction with parenteral nutrition, until the patient no longer requires parenteral nutrition or until participation in the study is terminated. | 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 12 Apr 2019.
- Background: PubMed 15659701Alwayn IP, Gura K, Nose V, Zausche B, Javid P, Garza J, Verbesey J, Voss S, Ollero M, Andersson C, Bistrian B, Folkman J, Puder M. Omega-3 fatty acid supplementation prevents hepatic steatosis in a murine model of nonalcoholic fatty liver disease. Pediatr Res. 2005 Mar;57(3):445-52. doi: 10.1203/01.PDR.0000153672.43030.75. Epub 2005 Jan 19.
- Background: PubMed 19179884Diamond IR, Sterescu A, Pencharz PB, Kim JH, Wales PW. Changing the paradigm: omegaven for the treatment of liver failure in pediatric short bowel syndrome. J Pediatr Gastroenterol Nutr. 2009 Feb;48(2):209-15. doi: 10.1097/MPG.0b013e318182c8f6.
- Background: PubMed 16029913Gura KM, Parsons SK, Bechard LJ, Henderson T, Dorsey M, Phipatanakul W, Duggan C, Puder M, Lenders C. Use of a fish oil-based lipid emulsion to treat essential fatty acid deficiency in a soy allergic patient receiving parenteral nutrition. Clin Nutr. 2005 Oct;24(5):839-47. doi: 10.1016/j.clnu.2005.05.020.
- Background: PubMed 18310188Gura KM, Lee S, Valim C, Zhou J, Kim S, Modi BP, Arsenault DA, Strijbosch RA, Lopes S, Duggan C, Puder M. Safety and efficacy of a fish-oil-based fat emulsion in the treatment of parenteral nutrition-associated liver disease. Pediatrics. 2008 Mar;121(3):e678-86. doi: 10.1542/peds.2007-2248.
- Background: PubMed 19661785Puder M, Valim C, Meisel JA, Le HD, de Meijer VE, Robinson EM, Zhou J, Duggan C, Gura KM. Parenteral fish oil improves outcomes in patients with parenteral nutrition-associated liver injury. Ann Surg. 2009 Sep;250(3):395-402. doi: 10.1097/SLA.0b013e3181b36657.
- Background: PubMed 10022591Van Aerde JE, Duerksen DR, Gramlich L, Meddings JB, Chan G, Thomson AB, Clandinin MT. Intravenous fish oil emulsion attenuates total parenteral nutrition-induced cholestasis in newborn piglets. Pediatr Res. 1999 Feb;45(2):202-8. doi: 10.1203/00006450-199902000-00008.
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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.
Maria Mascarenhas, MBBS principal investigator
Children's Hospital of Philadelphia
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 in people with Cholestasis and Short Bowel Syndrome, run by Children's Hospital of Philadelphia 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 5 inclusion and 6 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 12 Apr 2019. 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?
On the ClinicalTrials.gov record for NCT01297933, which also lists 6 linked publications.
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 12 Apr 2019.