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

Expanded access is currently available for this treatment.

Verified December 2012 by Children's Hospital of Philadelphia

Sponsor:

Information provided by (Responsible Party):

Maria Mascarenhas, Children's Hospital of Philadelphia

ClinicalTrials.gov Identifier:

NCT01297933

First received: February 15, 2011

Last updated: December 14, 2012

Last verified: December 2012

History of Changes

Purpose

A compassionate use protocol to provide Omegaven to pediatric patients with parenteral nutrition (PN) dependence and parenteral nutrition associated liver disease (PNALD).

Condition	Intervention	Phase
Cholestasis Short Bowel Syndrome	Drug: Omegaven	Phase 1

Study Type:	Expanded Access What is Expanded Access?
Official Title:	Intravenous Omega-3 Fatty Acid (Omegaven) for Pediatric Patients With Total Parenteral Nutrition Associated Liver Dysfunction

Resource links provided by NLM:

MedlinePlus related topics: Liver Diseases

Drug Information available for: Parenteral Nutrition, Total

U.S. FDA Resources

Further study details as provided by Children's Hospital of Philadelphia:

Study Start Date:	January 2011
Estimated Study Completion Date:	January 2014
Estimated Primary Completion Date:	January 2014 (Final data collection date for primary outcome measure)

Intervention Details:

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.

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.

Eligibility

Ages Eligible for Study:	up to 21 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

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 a conjugated bilirubin >= 2 mg/dL
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:

Other known causes of chronic liver disease (i.e. infectious hepatitis, metabolic disease)
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)

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT01297933

Contacts

Contact: Maria Mascarenhas, MBBS	215-590-2997	mascarenhas@email.chop.edu
Contact: Debbie Kawchak	267-426-7576	kawchak@email.chop.edu

Locations

United States, Pennsylvania
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Principal Investigator: Maria Mascarenhas, MBBS

Sponsors and Collaborators

Children's Hospital of Philadelphia

Investigators

Principal Investigator:

Maria Mascarenhas, MBBS

Children's Hospital of Philadelphia

More Information

Publications:

Alwayn IP, Gura K, Nosé 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. Epub 2005 Jan 19.

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.

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.

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.

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.

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.

Responsible Party:	Maria Mascarenhas, Section Chief, Clinical Nutrition, Children's Hospital of Philadelphia
ClinicalTrials.gov Identifier:	NCT01297933 History of Changes
Other Study ID Numbers:	10-007681
Study First Received:	February 15, 2011
Last Updated:	December 14, 2012
Health Authority:	United States: Food and Drug Administration

Keywords provided by Children's Hospital of Philadelphia:

parenteral nutrition associated liver disease
cholestasis
short bowel syndrome
Omegaven

Additional relevant MeSH terms:

Cholestasis
Liver Diseases
Short Bowel Syndrome
Bile Duct Diseases
Biliary Tract Diseases
Digestive System Diseases

Malabsorption Syndromes
Intestinal Diseases
Gastrointestinal Diseases
Postoperative Complications
Pathologic Processes

ClinicalTrials.gov processed this record on May 21, 2013

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