Omegaven as Alternative Parenteral Fat Nutrition

NCT03662282 | Completed Phase 3 Results FDA Drug

• 1 other identifier: 00033519
• Study Type: interventional
• Target: 2
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Cholestasis; Cholestasis of Parenteral Nutrition

Keywords

Omegaven; parenteral nutrition; Liver disease; Cholestasis

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (4)

• Number of Participant With Resolution of Direct Hyperbilirubinemia

  2 months

• Number of Participant With Preservation of Length

  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

  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

  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

Study Arms (1)

Drug - Omegaven®

EXPERIMENTAL

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

Interventions

Omegaven DRUG

Omegaven® fat emulsion will be used as a compassionate use treatment for critically ill infants with parenteral nutrition associated liver injury

Drug - Omegaven®

Eligibility Criteria

• Age: 14 Days+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

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

Sponsors & Collaborators

• University of South Florida: lead

Study Sites (1)

USF Health

Tampa, Florida, 33606, United States

Location

MeSH Terms

Conditions

Cholestasis; Hyperphagia; Liver Diseases

Interventions

fish oil triglycerides

Condition Hierarchy (Ancestors)

Bile Duct Diseases; Biliary Tract Diseases; Digestive System Diseases; Signs and Symptoms, Digestive; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Results Point of Contact

• Title: Thao Ho
• Organization: University of South Florida

tho1@usf.edu

813-844-3437

Study Officials

• Thao Ho, DO

  University of South Florida

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 23, 2018
• First Posted: September 7, 2018
• Study Start: October 23, 2018
• Primary Completion: January 7, 2019
• Study Completion: January 7, 2019
• Last Updated: July 22, 2020
• Results First Posted: July 22, 2020

Record last verified: 2019-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)