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

Expanded access is currently available for this treatment.
Verified April 2017 by Maria Mascarenhas, Children's Hospital of Philadelphia
Sponsor:
ClinicalTrials.gov Identifier:
NCT01297933
First Posted: February 17, 2011
Last Update Posted: April 14, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Information provided by (Responsible Party):
Maria Mascarenhas, Children's Hospital of Philadelphia
  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
Cholestasis Short Bowel Syndrome Drug: Omegaven

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:


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

Study Start Date: January 2011
Intervention Details:
    Drug: Omegaven
    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

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
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 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)
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01297933


Contacts
Contact: Maria Mascarenhas, MBBS 215-590-2997 mascarenhas@email.chop.edu
Contact: Susan A. Becker, RN, BSN, CCRC 267-426-0269 beckers@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. doi: 10.1097/MPG.0b013e318182c8f6.
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. doi: 10.1542/peds.2007-2248.
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.
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
First Submitted: February 15, 2011
First Posted: February 17, 2011
Last Update Posted: April 14, 2017
Last Verified: April 2017

Keywords provided by Maria Mascarenhas, Children's Hospital of Philadelphia:
parenteral nutrition associated liver disease
cholestasis
short bowel syndrome
Omegaven

Additional relevant MeSH terms:
Liver Diseases
Cholestasis
Short Bowel Syndrome
Digestive System Diseases
Bile Duct Diseases
Biliary Tract Diseases
Malabsorption Syndromes
Intestinal Diseases
Gastrointestinal Diseases
Postoperative Complications
Pathologic Processes


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