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

Expanded access is currently available for this treatment.
Verified July 2014 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: July 30, 2014
Last verified: July 2014
  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:


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

Study Start Date: January 2011
Estimated Study Completion Date: January 2017
Estimated Primary Completion Date: January 2017 (Final data collection date for primary outcome measure)
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

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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

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:

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: July 30, 2014
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 September 15, 2014