Compassionate Use of Omegaven IV Fat Emulsion

This study is enrolling participants by invitation only.
Sponsor:
Information provided by (Responsible Party):
Christiana Farkouh-Karoleski, Columbia University
ClinicalTrials.gov Identifier:
NCT01323907
First received: March 16, 2011
Last updated: February 11, 2013
Last verified: February 2013
  Purpose

The purpose of the study is to provide use of Omegaven in infants 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.


Condition Intervention
Total Parenteral Nutrition-induced Cholestasis
Drug: Omegaven IV lipid emulsion

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Compassionate Use of Omegaven IV Fat Emulsion in Infants With Life Threatening Parenteral Nutrition-associated Liver Disease (PNALD).

Resource links provided by NLM:


Further study details as provided by Columbia University:

Primary Outcome Measures:
  • Reduction of direct serum bilirubin level (mg/dL) [ Time Frame: Up to 6 months from the completion of the study. ] [ Designated as safety issue: No ]
    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.


Secondary Outcome Measures:
  • Essential fatty acid profile [ Time Frame: Up to 30 days from therapy completion ] [ Designated as safety issue: Yes ]
    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.


Estimated Enrollment: 10
Study Start Date: January 2011
Estimated Study Completion Date: January 2015
Estimated Primary Completion Date: January 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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 Name: Omega-3 Fatty Acids

  Eligibility

Ages Eligible for Study:   2 Months to 12 Months
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.
  • Under the care of an attending Neonatologist in the NICU
  • Must be at least 2 months of age

Exclusion Criteria:

  • Not a NICU patient
  • 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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01323907

Locations
United States, New York
Columbia Presbyterian Medical Center-Children's Hospital of NY
New York, New York, United States, 10032
Sponsors and Collaborators
Christiana Farkouh-Karoleski
Investigators
Principal Investigator: Chrsitiana R Farkouh, MD, MPH Columbia University
  More Information

No publications provided

Responsible Party: Christiana Farkouh-Karoleski, Assistant Clinical Professor of Pediatrics, Columbia University
ClinicalTrials.gov Identifier: NCT01323907     History of Changes
Other Study ID Numbers: AAAF2546
Study First Received: March 16, 2011
Last Updated: February 11, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by Columbia University:
Total Parenteral Nutrition-induced Cholestasis
Omegaven
liver disease
Cholestasis
Fat emulsions

Additional relevant MeSH terms:
Cholestasis
Bile Duct Diseases
Biliary Tract Diseases
Digestive System Diseases

ClinicalTrials.gov processed this record on October 23, 2014