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Safer Parenteral Nutrition in Pediatric Short Bowel Syndrome to Decrease Liver Damage

Expanded access is currently available for this treatment.
Verified January 2014 by Georgetown University
Sponsor:
Information provided by (Responsible Party):
Georgetown University
ClinicalTrials.gov Identifier:
NCT01861834
First received: May 22, 2013
Last updated: August 26, 2014
Last verified: January 2014
  Purpose

To provide children dependent on total parenteral nutrition with Omegaven®, a fish oil-based intravenous lipid emulsion that may be less hepatotoxic than conventional, vegetable oil-based intravenous lipid emulsions, and that may therefore reduce the need for liver transplantation.


Condition Intervention
Cholestasis of Parenteral Nutrition
Drug: Omegaven 10%

Study Type: Expanded Access     What is Expanded Access?
Official Title: A Safer Approach to Total Parenteral Nutrition in Pediatric Short Bowel Syndrome Intended to Decrease the Frequency and Severity of Liver Damage

Resource links provided by NLM:


Further study details as provided by Georgetown University:

Intervention Details:
    Drug: Omegaven 10%

    Patients with a sustained TPN requirement due to short bowel syndrome and TPN-associated liver disease that threatens progression to liver failure and death, for which the only available means of prevention at present is timely liver and/or intestinal transplant.

    Omegaven 10%, 1 gram/kg, IV, every 12 hours until transplantation, or stopping TPN

  Eligibility

Ages Eligible for Study:   2 Months to 18 Years
Genders Eligible for Study:   Both
Criteria

Inclusion Criteria:

  • The targeted population for enrollment is the cohort of patients with TPN-dependent short bowel syndrome, defined as any pediatric patient who, following abdominal surgery, has a residual small bowel length less than 25% of that predicted for gestational age or requires postoperative TPN for more than 42 days because of gastrointestinal intolerance and who has developed TPN-associated liver disease sufficient to pose a significant risk for progression to liver failure based on the following criteria:

    • Total serum bilirubin concentration greater than 3 mg/dL after a total duration of TPN greater than 2 months in the absence of a proven episode of bacteremia within the preceding 3 weeks.

PLUS EITHER:

• Platelet count less than 200,000/μL after a total duration of TPN greater than 2 months in the absence of a proven episode of bacteremia within the preceding 3 weeks.

OR

• Serum albumin concentration less than 3.2 mg/dL after a total duration of TPN greater than 2 months in the absence of a proven episode of bacteremia within the preceding 3 weeks.

Patients with coagulopathy due to parenteral nutrition-associated liver disease (INR > 1.2) will be potential candidates for enrollment, because patients with an elevated INR exceeding 2 have demonstrated resolution of coagulopathy after treatment with Omegaven®.11 Similarly, patients with hyperlipidemia will be potential candidates for enrollment.

Alternatively, patients currently receiving Omegaven that was initiated at another center because of intestinal failure with liver disease they do not need to meet the lab criteria listed above. The subject may continue Omegaven under this protocol at the discretion of the Principle Investigator.

Exclusion Criteria:

Patients with a history of the following will be excluded from enrollment in this protocol:

  • Allergy to fish or egg protein.
  • Liver disease proven or suspected to be caused by a process other than TPN-dependent short bowel syndrome, including but not limited to hepatitis C, hepatitis B, cystic fibrosis, biliary atresia, Alagille syndrome, familial intrahepatic cholestasis, and alpha-1-antitrypsin deficiency.11
  • Refusal of third party providers to reimburse hospital for the cost of Omegaven®.
  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: NCT01861834

Contacts
Contact: Stuart S. Kaufman, MD 202-444-0906

Locations
United States, District of Columbia
MedStar Georgetown Transplant Institute
Washington, District of Columbia, United States, 20007
Principal Investigator: Stuart Kaufman, MD         
Sponsors and Collaborators
Georgetown University
  More Information

No publications provided

Responsible Party: Georgetown University
ClinicalTrials.gov Identifier: NCT01861834     History of Changes
Other Study ID Numbers: IND107300
Study First Received: May 22, 2013
Last Updated: August 26, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Georgetown University:
Parenteral nutrition
TPN
Intestinal Failure
Short Bowel Syndrome
Cholestasis

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

ClinicalTrials.gov processed this record on November 27, 2014