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

• Conditions: Short Bowel Syndrome; Cholestasis

• Registration Number: NCT01297933
• Lead Sponsor: Children's Hospital of Philadelphia

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.

Study Timeline

• Study First Submitted: 2011-02-15
• Study First Submitted QC: 2011-02-15
• Study First Posted: 2011-02-17
• Status Verified: 2019-04
• Last Update Submitted: 2019-04-10
• Last Update Posted: 2019-04-12

Recruitment & Eligibility

• Status: APPROVED_FOR_MARKETING
• Sex: All
• Target Recruitment: Not specified

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)

Study & Design

• Study Type: EXPANDED_ACCESS
• Study Design: Not specified

Trial Locations

Locations (1)

Children's Hospital of Philadelphia; 🇺🇸 Philadelphia, Pennsylvania, United States