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

Primary Purpose

Cholestasis of Parenteral Nutrition

Status
Approved for marketing
Phase
Locations
United States
Study Type
Expanded Access
Intervention
Omegaven 10%
Sponsored by
Georgetown University
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an expanded access trial for Cholestasis of Parenteral Nutrition focused on measuring Total parenteral nutrition, TPN, Intestinal Failure, Short Bowel Syndrome, Cholestasis, Intestinal failure associated liver disease, Parenteral nutrition associated liver disease

Eligibility Criteria

2 Months - 18 Years (Child, Adult)All Sexes

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®. 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 that 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.
  • Refusal of third party providers to reimburse hospital for the cost of Omegaven®.

Sites / Locations

  • MedStar Georgetown Transplant Institute
  • MedStar Georgetown University Hospital

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

First Posted
May 22, 2013
Last Updated
May 5, 2019
Sponsor
Georgetown University
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1. Study Identification

Unique Protocol Identification Number
NCT01861834
Brief Title
Safer Parenteral Nutrition in Pediatric Short Bowel Syndrome to Decrease Liver Damage
Official Title
A Safer Approach to Total Parenteral Nutrition in Pediatric Short Bowel Syndrome Intended to Decrease the Frequency and Severity of Liver Damage
Study Type
Expanded Access

2. Study Status

Record Verification Date
May 2019
Overall Recruitment Status
Approved for marketing
Study Start Date
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Primary Completion Date
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Study Completion Date
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3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Georgetown University

4. Oversight

5. Study Description

Brief Summary
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cholestasis of Parenteral Nutrition
Keywords
Total parenteral nutrition, TPN, Intestinal Failure, Short Bowel Syndrome, Cholestasis, Intestinal failure associated liver disease, Parenteral nutrition associated liver disease

7. Study Design

8. Arms, Groups, and Interventions

Intervention Type
Drug
Intervention Name(s)
Omegaven 10%
Intervention Description
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

10. Eligibility

Sex
All
Minimum Age & Unit of Time
2 Months
Maximum Age & Unit of Time
18 Years
Eligibility 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®. 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 that 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. Refusal of third party providers to reimburse hospital for the cost of Omegaven®.
Facility Information:
Facility Name
MedStar Georgetown Transplant Institute
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20007
Country
United States
Facility Name
MedStar Georgetown University Hospital
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20007
Country
United States

12. IPD Sharing Statement

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

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