Efficacy and Safety of Omega-3 Lipid Therapy in Pediatric Patients With Parenteral Nutrition-Associated Liver Disease

This study is currently recruiting participants. (see Contacts and Locations)
Verified November 2012 by Amarnath, Rathna, M.D.
Sponsor:
Information provided by (Responsible Party):
Amarnath, Rathna, M.D.
ClinicalTrials.gov Identifier:
NCT01739517
First received: November 28, 2012
Last updated: November 29, 2012
Last verified: November 2012
  Purpose

This pilot study seeks to demonstrate the efficacy of an intravenous lipid preparation high in omega-3 fatty acids (Omegaven) in the treatment of cholestasis in parenteral nutrition dependent patients with short gut syndrome.


Condition Intervention Phase
Short Bowel Syndrome
Cholestasis
Drug: Omegaven Therapy
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Efficacy and Safety of Omega-3 Lipid Therapy in Pediatric Patients With Parenteral Nutrition-Associated Liver Disease

Resource links provided by NLM:


Further study details as provided by Amarnath, Rathna, M.D.:

Primary Outcome Measures:
  • Improvement of liver dysfunction as measured by time to achieve 50 % decrease in direct bilirubin [ Time Frame: weekly then biweekly data collection ] [ Designated as safety issue: No ]
    Direct bilirubin will be collected at baseline, then weekly for 30 days, and then biweekly, thereafter, up to an expected average of 108 weeks


Secondary Outcome Measures:
  • a) Maintenance of nutritional status [ Time Frame: Labwork will be collected at baseline, then weekly for the first month. Thereafter, a lipid panel will be collected every 2 months, complete metabolic panel every 2 weeks, and essential fatty acid profile monthly, up to an expected average of 108 weeks ] [ Designated as safety issue: No ]
    Nutritional status will be monitored by reviewing complete metabolic panel, magnesium, weight, vitals, phosphorus, prealbumin, lipid panel, and essential free fatty acid profile. The essential fatty acid profile will be checked at baseline and then monthly for at least 6 months until the patient is determined to be receiving at least 2.7% of caloric intake from linoleic acid. If the patient's essential fatty acid profile indicates that the patient is absorbing adequate amounts of essential fatty acid, the essential fatty acid profile will be discontinued .

  • Occurrence of potential adverse side effects [ Time Frame: biweekly labwork up to an expected average of 108 weeks ] [ Designated as safety issue: Yes ]
    Adverse events may include but are not limited to prolonged prothrombin time, hypertriglyceridemia, and anaphylaxis in relation to the patient's therapy.

  • c) Resolution of liver dysfunction [ Time Frame: weekly complete metabolic panel for the first month and then biweekly thereafter, up to an expected average of 108 weeks ] [ Designated as safety issue: No ]
    Resolution of liver dysfunction will be defined by achievement of normal direct bilirubin, aspartate aminotransferase and alanine transaminase.


Estimated Enrollment: 20
Study Start Date: March 2009
Estimated Study Completion Date: December 2013
Estimated Primary Completion Date: December 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Omegaven Therapy
After baseline labs, which have been collected no earlier than seven days prior to the initiation of therapy are obtained, therapy with Omegaven will be initiated at a starting dose of 0.5 g/kg/day infused over 12 hours. If tolerated, the dose will be increased to 1 g/kg/day, the goal dose. Omegaven will be infused intravenously through either a central or peripheral catheter in conjunction with parenteral nutrition.
Drug: Omegaven Therapy
After baseline labs, which have been collected no earlier than seven days prior to the initiation of therapy are obtained, therapy with Omegaven will be initiated at a starting dose of 0.5 g/kg/day infused over 12 hours. If tolerated, the dose will be increased to 1 g/kg/day, the goal dose. Omegaven will be infused intravenously through either a central or peripheral catheter in conjunction with parenteral nutrition.

  Eligibility

Ages Eligible for Study:   up to 1 Year
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Patient will be dependent upon parenteral nutrition (PN)
  • Patient will have short gut syndrome (loss of >50% of small bowel)
  • Patient's guardian/caregiver provides informed consent for patient to receive therapy
  • Pediatric patient ≤ 1 year of age
  • Expected PN duration is greater than 30 days
  • Direct bilirubin >2.0 mg/dL measured on two occasions no more than one week apart

Exclusion Criteria:

  • Liver dysfunction secondary to cause other than PN verified by standard of care diagnostic procedures and lab work to rule out alternative causes of neonatal cholestasis.
  • Any patient in whom Omegaven therapy would be contraindicated, such as an allergy to any seafood product, egg protein, and/or previously established allergy to Omegaven
  • impaired lipid metabolism
  • severe hemorrhagic disorder
  • unstable diabetes mellitus
  • collapse and shock, stroke/embolism, recent cardiac infarction, or undefined coma status
  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: NCT01739517

Locations
United States, South Carolina
Palmetto Health Children's Hospital Recruiting
Columbia, South Carolina, United States, 29203
Contact: Terra R Varner, PharmD    803-434-3034    terra.varner@palmettohealth.org   
Principal Investigator: Terra R Varner, PharmD         
Sponsors and Collaborators
Amarnath, Rathna, M.D.
Investigators
Principal Investigator: Terra R Varner, PharmD Palmetto Health Children's Hospital
  More Information

Publications:

Responsible Party: Amarnath, Rathna, M.D.
ClinicalTrials.gov Identifier: NCT01739517     History of Changes
Other Study ID Numbers: IND104555
Study First Received: November 28, 2012
Last Updated: November 29, 2012
Health Authority: United States: Food and Drug Administration

Keywords provided by Amarnath, Rathna, M.D.:
Short bowel syndrome
Cholestasis
Omegaven
Parenteral nutrition-associated Liver Disease

Additional relevant MeSH terms:
Cholestasis
Liver Diseases
Short Bowel Syndrome
Bile Duct Diseases
Biliary Tract Diseases
Digestive System Diseases
Malabsorption Syndromes
Intestinal Diseases
Gastrointestinal Diseases
Postoperative Complications
Pathologic Processes

ClinicalTrials.gov processed this record on July 20, 2014