Omegaven as Alternative Parenteral Fat Nutrition

To provide Omegaven® as a compassionate treatment for critically ill infants with parenteral nutrition associated cholestasis

Study Overview

• Status: Completed
• Conditions: Cholestasis; Cholestasis of Parenteral Nutrition
• Intervention / Treatment: Drug: Omegaven

Detailed Description

An intermediate population, Investigational New Drug study, to provide an alternate intravenous fat nutrition, Omegaven®, to help improve liver function while providing adequate nutrition for critically ill infants with parenteral nutrition associated cholestasis

• Study Type: Interventional
• Enrollment (Actual): 2
• Phase: Phase 3

Contacts and Locations

Study Locations

• United States
  • Florida
    • Tampa, Florida, United States, 33606
      • USF Health

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 weeks and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Greater than 14 days old
• Has parenteral nutrition associated cholestasis defined as at least 2 consecutive direct bilirubin >2 mg/dL with anatomical or functional short gut (OR >4 mg/dL if intact intestine) obtained at least 1 week apart with a ratio of direct: total bilirubin > 0.4
• Patient is parenteral nutrition dependent (unable to meet nutritional needs solely by enteral nutrition) and are expected to require parenteral nutrition for at least 3 more weeks
• Patient has not responded to other therapeutic approaches for parenteral nutrition-associated liver disease such as : cycling of parenteral nutrition, avoiding overfeeding, reduction/removal of copper and manganese from parenteral nutrition, advancement of enteral feeding, and use of Ursodiol
• Patient has been on at least 2 weeks of SMOFLIPIDs with no improvement or worsening of direct bilirubin levels
• Signed patient informed consent
• The patient is expected to have a reasonable possibility of survival
• No other known etiology of cholestasis other than parenteral nutrition-associated liver disease at the time of Omegaven® initiation

Exclusion Criteria:

• Known causes of cholestasis other than parenteral nutrition-associated liver disease including but not limited to Hepatitis C, Cystic fibrosis, biliary atresia, and alpha 1 antitrypsin deficiency are present, prior to Omegaven® initiation
• Known fish or egg allergy

• Any of the contraindications to use of Omegaven®:

  1. Active new infection at the time of initiation of Omegaven®
  2. Hemodynamic instability
  3. Recent use of medications with associated risk of bleeding, including NSAIDs
  4. Active coagulopathy or bleeding
  5. Platelet counts persistently under 30,000 despite transfusions
  6. Unstable hyperglycemia
  7. Impaired lipid metabolism (triglycerides >1000 mg/dL) while on 1 g/kg/day or less of Intralipid
  8. History of severe hemorrhagic disorders (ie. hemophilia, Von Willebrand disease, etc.)
  9. Unstable diabetes mellitus
  10. Collapse and shock
  11. Stroke/ Embolism
  12. Cardiac infarction within the last 3 months
  13. Undefined coma status

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Drug - Omegaven®; Therapy with Omegaven® will be initiated at the goal dose of 0.5-1/kg/day. The default is over 24 hours, but shorter intervals may be considered if a program of total parenteral nutrition cycling is recommended. Omegaven® will be infused intravenously through either a central or peripheral catheter.	Drug: Omegaven; Omegaven® fat emulsion will be used as a compassionate use treatment for critically ill infants with parenteral nutrition associated liver injury

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participant With Decrease in Direct Bilirubin Level	Number of participant with decrease in direct bilirubin levels within 30 days of treatment	First month of treatment

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participant With Resolution of Direct Hyperbilirubinemia	Number of participant with Direct bilirubin level of <2mg/dL at discontinuation of treatment	2 months
Number of Participant With Preservation of Length	Monitoring of weekly length while the patient is receiving Omegaven® treatment	Weekly measurement during treatment. Evaluation every 30 days from treatment initiation until discontinuation of treatment, patient hospital discharge, whichever comes first, or up to 6 months following initiation of treatment
Number of Participant With Preservation of Head Circumference	Monitoring of weekly head circumference while the patient is receiving Omegaven® treatment	Weekly measurement during treatment. Evaluation every 30 days from treatment initiation until discontinuation of treatment, patient hospital discharge, whichever comes first, or up to 6 months following initiation of treatment
Number of Participant With Preservation of Normal Weight Gain	Monitoring of daily weight gain while the patient is on Omegaven®	Weekly measurement during treatment. Evaluation every 30 days from treatment initiation until discontinuation of treatment, patient hospital discharge, whichever comes first, or up to 6 months following initiation of treatment

Collaborators and Investigators

• Sponsor: University of South Florida
• Investigators: Principal Investigator: Thao Ho, DO, University of South Florida

Study record dates

Study Major Dates

• Study Start (Actual): October 23, 2018
• Primary Completion (Actual): January 7, 2019
• Study Completion (Actual): January 7, 2019

Study Registration Dates

• First Submitted: July 23, 2018
• First Submitted That Met QC Criteria: September 5, 2018
• First Posted (Actual): September 7, 2018

Study Record Updates

• Last Update Posted (Actual): July 22, 2020
• Last Update Submitted That Met QC Criteria: July 7, 2020
• Last Verified: July 1, 2019

More Information

Terms related to this study

• Keywords: Liver disease; Cholestasis; parenteral nutrition; Omegaven
• Additional Relevant MeSH Terms: Digestive System Diseases; Biliary Tract Diseases; Bile Duct Diseases; Cholestasis
• Other Study ID Numbers: 00033519

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No