Clinical Trial: Compassionate Use of Omegaven in Children

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Fish Oil Lipid Emulsion for the Treatment of Parenteral-Nutrition-Associated Liver Disease in Infants

Brief Summary: This is a single-assignment study to evaluate whether Omegaven (IV fish oil) is effective at treating liver disease in children on long-term IV nutrition.

Detailed Summary:

Children up to 18 years of age, on parenteral nutrition, with a direct bilirubin level of 2 mg/dL or greater will be eligible to receive Omegaven at a maximum dose of 1 g/kg/day.

Up to 200 children will be eligible for enrollment.

Direct bilirubin levels and other labs will be monitored as well as growth parameters.


Sponsor: University of Oklahoma

Current Primary Outcome: Resolution of cholestasis [ Time Frame: Within the first 3 months of sole Omegaven use ]

To determine if Omegaven results in the resolution of cholestasis (DB <2 for 2 consecutive weeks)


Original Primary Outcome:

  • Peak direct bilirubin levels [ Time Frame: Within the first year of life ]
    Comparison of the highest direct bilirubin level for those receiving prophylactic Omegaven vs Intralipid
  • Resolution of cholestasis [ Time Frame: Within the first year of life ]
    Comparison of treatment with Omegaven vs Intralipid for the length of time for cholestasis to resolve (DB <2 for 2 consecutive weeks)
  • Dose-effect on peak bilirubin, rate of rise, and rate of resolution [ Time Frame: Within the first year of life ]
    Comparison of 1 g/kg/day vs 1.5 g/kg/day of Omegaven and Intralipid on peak direct bilirubin levels, rate of rise, and/or rate of cholestasis resolution


Current Secondary Outcome:

  • Safety issues [ Time Frame: Within the first year of use ]
    To determine if Omegaven results in safety issues such as increased bleeding, essential fatty acid deficiency, elevated triglyceride levels, elevated liver function tests, increased liver and/or intestinal transplant rates, or death
  • Essential fatty acid deficiency [ Time Frame: Within the first three months of combined use ]
    To determine if the combination of Omegaven and Intralipid results in normalized lipid profiles.


Original Secondary Outcome:

  • Neurodevelopmental outcomes [ Time Frame: The first 2 years of life ]
    Comparison of monotherapy on Bayley III scores (total, cognitive, language, and motor scaled scores) at 18 +/- 6 months for those on monotherapy
  • Retinopathy of prematurity [ Time Frame: The first 6 months of life ]
    Comparison of monotherapy on the incidence and severity of retinopathy
  • Sepsis [ Time Frame: The first year of life ]
    Comparison of the rate of blood, line, or urosepsis while on parenteral nutrition
  • Growth and fat mass accretion [ Time Frame: The first 2 years of life ]
    Comparison of weight, length, and head circumference growth from birth to neurodevelopmental follow-up as well as fat mass accretion as measured by air displacement plethysmography
  • Steatosis (fatty liver) [ Time Frame: The first year of life ]
    Comparison of the incidence and severity of steatosis
  • Liver transplant and/or death [ Time Frame: The first 2 years of life ]
    Comparison of the rate of liver transplant and/or death
  • Endothelial injury markers [ Time Frame: The first six months of life ]
    Endothelial injury markers in relation to the direct bilirubin levels


Information By: University of Oklahoma

Dates:
Date Received: February 17, 2015
Date Started: June 2015
Date Completion: April 2021
Last Updated: November 18, 2016
Last Verified: November 2016