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Compassionate Use of Omegaven for Pediatric Patients With Parenteral Nutrition Associated Liver Disease (Omegaven)

Primary Purpose

Cholestasis, Short Bowel Syndrome

Status
Approved for marketing
Phase
Locations
United States
Study Type
Expanded Access
Intervention
Omegaven
Sponsored by
Children's Hospital of Philadelphia
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an expanded access trial for Cholestasis focused on measuring parenteral nutrition associated liver disease, cholestasis, short bowel syndrome, Omegaven

Eligibility Criteria

undefined - 21 Years (Child, Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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)

Sites / Locations

  • Children's Hospital of Philadelphia

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

First Posted
February 15, 2011
Last Updated
April 10, 2019
Sponsor
Children's Hospital of Philadelphia
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1. Study Identification

Unique Protocol Identification Number
NCT01297933
Brief Title
Compassionate Use of Omegaven for Pediatric Patients With Parenteral Nutrition Associated Liver Disease
Acronym
Omegaven
Official Title
Intravenous Omega-3 Fatty Acid (Omegaven) for Pediatric Patients With Total Parenteral Nutrition Associated Liver Dysfunction
Study Type
Expanded Access

2. Study Status

Record Verification Date
April 2019
Overall Recruitment Status
Approved for marketing
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
Children's Hospital of Philadelphia

4. Oversight

5. Study Description

Brief Summary
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cholestasis, Short Bowel Syndrome
Keywords
parenteral nutrition associated liver disease, cholestasis, short bowel syndrome, Omegaven

7. Study Design

8. Arms, Groups, and Interventions

Intervention Type
Drug
Intervention Name(s)
Omegaven
Intervention Description
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.

10. Eligibility

Sex
All
Maximum Age & Unit of Time
21 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: 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 Exclusion Criteria: 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)
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Maria Mascarenhas, MBBS
Organizational Affiliation
Children's Hospital of Philadelphia
Official's Role
Principal Investigator
Facility Information:
Facility Name
Children's Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States

12. IPD Sharing Statement

Citations:
PubMed Identifier
15659701
Citation
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.
Results Reference
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PubMed Identifier
19179884
Citation
Diamond 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.
Results Reference
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PubMed Identifier
16029913
Citation
Gura 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.
Results Reference
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PubMed Identifier
18310188
Citation
Gura 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.
Results Reference
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PubMed Identifier
19661785
Citation
Puder 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.
Results Reference
background
PubMed Identifier
10022591
Citation
Van 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.
Results Reference
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Compassionate Use of Omegaven for Pediatric Patients With Parenteral Nutrition Associated Liver Disease

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