Intravenous Fat Emulsion Comprised of Fish Oil in the Treatment of Parenteral Nutrition Induced Liver Injury in Infants

Compassionate Use of an Intravenous Fat Emulsion Comprised of Fish Oil in the Treatment of Parenteral Nutrition Induced Liver Injury in Infants

To provide a mechanism for critically ill infants with parenteral nutrition (PN) associated cholestasis to receive Omegaven for compassionate use situations for which there are no satisfactory alternative treatments.

Study Overview

• Status: Approved for marketing
• Conditions: Total Parenteral Nutrition-induced Cholestasis
• Intervention / Treatment: Drug: Omegaven; Drug: Omegaven; Dietary supplement: Omegaven

Detailed Description

Compassionate Use

• Study Type: Expanded Access
• Phase: Phase 3

Contacts and Locations

Study Locations

• United States
  • Texas
    • San Antonio, Texas, United States, 78207
      • Children's Hospital of San Antonio

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 weeks to 2 years (Child)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Critically ill infants with either a known anatomic short gut (greater than 50% of the bowel removed) or known severe dysmotility of the gut reflecting non-functional gut similar to anatomic short gut will be offered Omegaven® when their direct bilirubin reaches 2 mg/dL. Infants who do not meet the above criteria for anatomic or dysfunctional short gut will be allowed to receive Omegaven® when their direct bilirubin reaches 4 mg/dL. The qualifying measurements of 2 mg/dL or 4 mg/dL direct bilirubin must be consecutive and obtained at least 24 hours apart.
2. Be expected to require intravenous nutrition for at least an additional 28 days

3. Patient must have documented failure of or ineligibility for the following therapies to prevent progression of PNALD:

   • Reduction of Intralipid® to 1 g/kg/day
   • Limiting trace minerals including copper and manganese
   • Initiation and use of Ursodiol
   • Cycling of parenteral nutrition
   • Advancement of enteral feedings
4. Parental informed consent must be signed.

Exclusion Criteria:

1. Have a congenitally lethal condition (e.g. Trisomy 13).
2. Have clinically severe bleeding not able to be managed with routine measures.
3. Have evidence of a viral hepatitis or primary liver disease as the primary etiology of their cholestasis.
4. Have other health problems such that survival is extremely unlikely even if the infant's cholestasis improves.
5. Has culture positive sepsis

Study Plan

How is the study designed?

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: 1; Drug: Omegaven

Drug: Omegaven; Therapy with Omegaven will be provided at a dose of 1 gm/kg/day (by continuous infusion). Omegaven will be infused intravenously through either a central or peripheral catheter in conjunction with parenteral nutrition.; Other Names: omega-3 enriched fat emulsion; Drug: Omegaven; Treatment will be given for as long as the child needs any TPN AND has a conjugated bilirubin greater than 2 mg/dL for a maximum of 2 years.; Drug: Omegaven; If the bilirubin is less than 2 mg/dL but the child still requires TPN, then the Omegaven will be continued until the infant no longer requires TPN.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Death associated with liver disease	Those patients with death only associated with liver diseasepre-treatment period and the expected rate of growth.	Maximum 2 years

Secondary Outcome Measures

Outcome Measure	Time Frame
Liver transplantation	Maximum 2 years
Growth over time as determined by weight in grams.	Maximum 2 years
Growth over time as measured by length in cm.	Maximum 2 years
Growth over time as measured by head circumference in cm.	Maximum 2 years
Development of essential fatty acid (EFA) deficiency as measured by triene:tetraene ratio in those with prolonged NPO status.	Maximum 2 years
Hyperlipidemia as measured by triglyceride level	Maximum 2 years

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Culture positive bloodstream infection	Calculate the number (total and daily rate) of bloodstream infections prior to therapy, in each baby receiving Omegaven and compare with the previous rate.	Maximum 2 years

Collaborators and Investigators

• Sponsor: Pediatrix
• Investigators: Principal Investigator: Kaashif Ahmad, MD, Pediatrix

Study record dates

Study Major Dates

• Study Start: August 1, 2015
• Primary Completion: April 1, 2019
• Study Completion: April 1, 2019

Study Registration Dates

• First Submitted: July 27, 2015
• First Submitted That Met QC Criteria: August 24, 2015
• First Posted (Estimated): August 27, 2015

Study Record Updates

• Last Update Posted (Actual): July 26, 2024
• Last Update Submitted That Met QC Criteria: July 23, 2024
• Last Verified: April 1, 2019

More Information

Terms related to this study

• Keywords: cholestasis; parenteral nutrition associated liver disease; omegaven
• Additional Relevant MeSH Terms: Digestive System Diseases; Biliary Tract Diseases; Bile Duct Diseases; Cholestasis
• Other Study ID Numbers: WIRB Study# 1155515