Compassionate Use of Omegaven IV Fat Emulsion

Compassionate Use of Omegaven IV Fat Emulsion in Infants With Life Threatening Parenteral Nutrition-associated Liver Disease (PNALD).

The purpose of the study is to provide use of Omegaven in children with life threatening Total Parenteral Nutrition (TPN) associated cholestasis when all other medical and surgical treatments/therapies have been either ineffective or not feasible in the treatment of this serious condition. This is a compassionate use protocol.

Study Overview

• Status: Withdrawn
• Conditions: Total Parenteral Nutrition-induced Cholestasis
• Intervention / Treatment: Drug: Omegaven IV lipid emulsion

• Study Type: Interventional
• Phase: Not Applicable

Contacts and Locations

Study Locations

• United States
  • New York
    • New York, New York, United States, 10032
      • Columbia Presbyterian Medical Center-Children's Hospital of NY

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 months to 18 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Diagnosis of life threatening PNALD
• Dependent on Parenteral Nutrition for caloric needs
• Expected to require PN for at least another 30 days
• Other causes of liver disease have been excluded
• GI/Liver service is involved in patient care
• Standard therapies for the treatment of PNALD must have been utilized prior to initiating omegaven.
• Inpatient at Morgan Stanley Children's Hospital of NewYork Presbyterian Hospital
• Must be at least 2 months of age

Exclusion Criteria:

• Not inpatient
• Younger than 2 months of age
• Expected to be weaned off of parenteral nutrition within 30 days
• have other documented causes of liver disease
• have signs of proven severe advanced liver disease
• Allergy to seafood, egg protein and/or previous allergy to Omegaven
• active coagulopathy, impaired lipid metabolism or severe hyperlipidemia with or without pancreatitis, stoke, embolism, hemodynamic collapse or shock, recent MI, cholestasis due to any reason other than PNALD, active new infection at time of initiation of Omegaven, hemodynamic instability or if unable to tolerate the necessary laboratory monitoring
• must not be enrolled in another clinical trial involving an investigational agent (unless approved by the designated physicians).
• Parent or legal guardian must be willing to provide consent.

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: Omegaven; Omegaven IV lipid emulsion administration for infants with life threatening parenteral nutrition associated liver disease

Drug: Omegaven IV lipid emulsion; For infants meeting inclusion criteria and whose guardians consent to participation, Omegaven will be initiated at the dose of 0.5gram/kg/day and is infused over 24 hours for 1-2 days and then advanced to 1 gram/kg/day. Omegaven will be infused intravenously through a central or peripheral catheter alone or in conjunction with parenteral nutrition.; Other Names: Omega-3 Fatty Acids

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Reduction of direct serum bilirubin level (mg/dL)	For effectiveness endpoint, the allowable minimum time interval between serial direct bilirubin measurements that will determine reversal of cholestasis is 2 consecutive measurements of direct bilirubin < or = to 2mg/dL obtained at least 2 weeks apart.	Up to 6 months from the completion of the study.

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Essential fatty acid profile	Previous studies have found no association between Omegaven administration and essential fatty acid deficiency. Due to theoretical concern for developing fatty acid deficiency with low dose Omegaven administration, these levels will be followed during Omegaven therapy.	Up to 30 days from therapy completion

Collaborators and Investigators

• Sponsor: Sivan Kinberg
• Investigators: Principal Investigator: Sivan Kinberg, MD, MS, Columbia University

Study record dates

Study Major Dates

• Study Start (Anticipated): January 1, 2011
• Primary Completion (Actual): February 13, 2019
• Study Completion (Actual): February 13, 2019

Study Registration Dates

• First Submitted: March 16, 2011
• First Submitted That Met QC Criteria: March 24, 2011
• First Posted (Estimate): March 28, 2011

Study Record Updates

• Last Update Posted (Actual): February 6, 2020
• Last Update Submitted That Met QC Criteria: February 4, 2020
• Last Verified: February 1, 2020

More Information

Terms related to this study

• Keywords: liver disease; Cholestasis; Omegaven; Total Parenteral Nutrition-induced Cholestasis; Fat emulsions
• Additional Relevant MeSH Terms: Digestive System Diseases; Biliary Tract Diseases; Bile Duct Diseases; Cholestasis
• Other Study ID Numbers: AAAF2546